StemCell Therapy

Were seeing a lot of bivalent molecules in drug discovery these days, especially with the popularity of bifunctional protein degrader ligands. The general structure of such thing is (ligand)-linker-(ligand), with the two ligands chosen (in the case of targeted protein degradation) to bring a ubiquitin ligase complex up close to some protein youve marked for destruction. But there are a lot of other bifunctional species that fit the same pattern. Heres a look back at the history of G-protein-coupled-receptor ligands set up that same way.

It goes back to 1982 work from the lab of Phil Portoghese (a name that medicinal chemists will definitely be familiar with!) That took a naltrexone derivative (naltrexamine) and put molecules of it on each end of a series of polyethyleneglycol (PEG) linkers, to try to bind simultaneously to adjacent opioid receptors. 1982 was not a time when you did this with membrane preps of cloned receptors that first paper is done with guinea pig ileum and mouse vas deferens tissue preps, and Im pretty sure the present authors (NIDA-NIH( are putting that detail in there just to raise the eyebrows of the later generations.

I was an undergraduate chemistry student when this work appeared, and was not a regular J. Med. Chem. reader at the time, although I do remember seeing some of the follow-up papers as the years went on. Its worth remembering that when this research started, no one had any idea that GPCR receptor dimers or oligomers might be defined functional units this was an effort to see how far apart they might be, just for starters. That first paper did conclude that they were in fact getting more binding than the sum of the parts and that this did depend on the length of the linker. Those two conclusions, in fact, continue to hold up for the general bifunctional-molecule field, and with many of the same qualifications and complications that Portoghese encountered during the 1980s.

For example, see this 2007 paper from the Whitesides group, looking at effective molarity and linker length. In that case, theyre tying a known carbonic anhydrase ligand to the protein covalently at a known distance from the binding site and giving it various tether lengths to find its way. The fundamental lesson is that too long a linker can do you some harm (lots of floppy entropy to overcome), but too short a linker is deadly, because youre just not going to reach the binding site at all. Targeted protein degradation projects have seen similar effects.

But there are others laid on top of those. It would be easy to imagine such linkers as just inert spacers, thingies that hold the business end of these molecules apart. But that would be a mistake, too, because the linkers can participate in the binding events, too. The Sharpless groups first report in 2002 of a ligand formed by in situ click chemistry is an example. Two ligands for the far ends of acetylcholinesterases roomy binding site did their own azide/alkyne cycloaddition when brought into proximity in there, and formed a femtomolar inhibitor. The X-ray crystal structure showed that the triazole linker itself participated in the binding, which is where some of that impressive affinity came from (its worth noting that that crystal structure is in itself an interesting and complicated story).

The spanning-two-adjacent-receptors work described in the first link of this post shows some of these effects, too the NIDA authors have done a lot of work on bifunctional dopamine receptor ligands, and theyve found that the linker length, functionality, and flexibility have to be considered as key variables. The targeted protein degradation field can tell you a lot about that, too length, as mentioned before, is the first consideration, but there are all sorts of cases where what seem to be similar linkers (in length and flexibility) that give very different effects in cellular degradation assays. Add that to the variable effects of the ligands, and youre in for a real fiesta: for example, you cant just optimize your bifunctional head groups based on potency to the target protein, because the eventual degradation efficiency does not have to follow that order at all. Nor does the selectivity against related proteins have to translate to selective degradation; you can get surprised in both directions, and you probably will.

In fact, the TPD world is probably even crazier than the linked-receptor one, because in the latter case, youre (at least some of the time) spanning the distance between proteins that are already naturally in proximity. GPCRs form all sorts of dimers and oligomers on the cell surface, in important patterns that were still trying to figure out. But targeted protein degradation is all about bringing proteins together that normally have no business with each other at all. Youre forcing some hapless target protein into the proximity of a ubiquitin ligase complex that just assumes that hey, heres another protein next to me, lets do that voodoo that I do and ubiquitinate the crap out of it. I speak technically here, you understand. Normally this ligase complex wouldnt even be seeing your target protein, but youre trying to rewire that system for fun and profit.

That means that the ternary complex (target protein, bifunctional degrader, and ubiquitin ligase) is a wild frontier of molecular interaction. Some things are going to match up as these species are brought together, and some things are going to clash, and at the moment we really dont have a good way to anticipate whats going to happen. TPD remains a rather. . .empirical. . .field for now, which in practice means that youd better try this and try that and try that other thing over there, what the heck. It would make everyone feel better if that werent the case, and everyone would be far more efficient steely-eyed protein degradation masters sitting in mission control and pointing out targets, but that is a vision for the future. For now, its similar to the traditional ag-chem development model of spray and pray.

One last note on the whole bifunctional idea: when I used to see papers with such linked molecules in them back in the early 1990s, in my first years in med-chem, I would (being honest here) just roll my eyes. The whole idea seemed too simplistic, too academic, and too odd. You couldnt just go around sticking two molecules together with a little connecting chain and expect that to work, right? And if it worked in an in vitro assay, well OK, nice paper, but you couldnt just go around trying to turn such weirdo molecules into drugs, right? Wrong, wrong, and wrong. As discussed above, its actually a complicated thing to get to work, but it can be a really good idea if you have a good reason for sticking two proteins together. Its quite possible that drug-discovery progress in this area was delayed by the rule-of-five years when people were terrified of high molecular weights, but since that has been breaking down under the weight of evidence, people are a lot more willing to explore the larger species needed for linked-bifunctionals. Dont overlook, though, that a less specific thing that slowed these ideas down was that people thought that these molecules just looked strange.

Read more:
Linked-Up Molecules Through the Years - Science Magazine

Inflammatory bowel disease (IBD) is an umbrella term for two conditions, Crohns disease and ulcerative colitis, that are characterized by chronic inflammation of the gastrointestinal (GI) tract. This chronic and prolonged inflammation results in damage to the GI tract.

Historically IBD has been predominantly seen in industrialized countries, with the highest reported prevalence values in Europe and North America. In the United States alone, approximately 1.6 million people currently have Crohns disease or ulcerative colitis, and as many as 70,000 new cases of IBD are diagnosed each year. Although the incidence of IBD in North America and Europe is currently reported to be stabilizing or decreasing, the burden remains high as prevalence exceeds 03%. Over the last 30 years, the predominance of IBD has accelerated in newly industrialized countries including Africa, Asia and South America. Reports of IBD appear to be higher in urban areas than in rural areas, as well as in higher socio-economic classes. Individuals who immigrate to industrial urbanized developed nations before adolescence and those immigrants who initially belonged to a low-incidence population show a significantly higher incidence of IBD. This rise has been attributed to the rapid modernization and westernization of the population.

The reported rise in the number of people living with inflammatory bowel disease reflects a need for more research to find a cure. This article explores current therapies, drugs in the pipeline and disease outlook for patients and their caregivers living with IBD.

Overview

Inflammatory Bowel Disease is a broad term that describes conditions characterized by chronic inflammation of the gastrointestinal tract. The GI tract is responsible for the digestion of food, absorption of nutrients and elimination of waste. Inflammation impairs the ability of affected GI organs to function properly, leading to symptoms such as persistent diarrhea, abdominal pain, rectal bleeding, weight loss and fatigue. The two most common inflammatory bowel diseases are Crohns disease and ulcerative colitis.

Causes:

The exact cause of IBD is not entirely understood, however, it is known to involve an interaction between the immune system, genes and environmental factors.

In people with IBD, the immune system mounts an inappropriate response to the intestinal tract, resulting in inflammation. This abnormal immune system reaction occurs in people who have inherited genes that make them susceptible to IBD. Unidentified environmental factors serve as the trigger that initiates the harmful immune response in the intestines.

Studies have shown that 5 to 20% of affected individuals have a first-degree relative (parent, child or sibling) with one of the diseases. Numerous genes and genetic mutations connected to IBD have been identified including a mutation in the NOD2/CARD15 gene. Up to 20% of IBD patients in North America and Europe may have a mutation in the NOD2/CARD15 gene. While genetic testing is possible, it is not currently a part of the diagnostic process for IBD. Genetic testing can identify a potential risk for IBD in an individual but cannot predict whether IBD will develop.

The environmental factors that trigger IBD are not known, but several potential risk factors have been studied. These include smoking, use of antibiotics, use of Nonsteroidal anti-inflammatory drugs, diet and geographic location (more prevalent in industrialized countries).

Life expectancy: People with Inflammatory Bowel Disease have a relatively normal life expectancy compared to the general population. However, people with Crohns disease have a slightly higher overall mortality rate than the general healthy population. The increase in deaths is largely due to conditions such as cancer (particularly lung cancer), chronic obstructive pulmonary disease, gastrointestinal diseases, (excluding Crohns disease), and diseases of the genital and urinary tracts. In addition, patients with extensive inflammation in the colon due to ulcerative colitis are at higher risk than the general population for dying from gastrointestinal and lung diseases.

Annual Cost: There are both direct and indirect costs associated with IBD. Direct medical costs include expenses for hospitalizations, physician services, prescription drugs, over-the-counter drugs, skilled nursing care, diagnostic procedures and other healthcare services. Indirect costs are the value of lost earnings or productivity. Indirect costs also include the value of leisure time lost.

Direct Costs: The annual direct cost of Crohns disease is estimated to be from $8,265 per patient to $18,963 per patient and the annual direct cost of ulcerative colitis is estimated to be from $5,066 per patient to $15,020 per patient. Extrapolating from this data to the current prevalence estimates of IBD (780,000 cases of Crohns disease and 907,000 cases of ulcerative colitis), the total annual direct costs for all patients with IBD in the United States is estimated to be between $11 billion to $28 billion.

Indirect Costs: Based on a national health survey in 1999, nearly 32% of symptomatic IBD patients reported being out of the workforce in a one-year period, incurring an indirect cost of an estimated $5,228 per patient, bringing the total indirect cost of IBD in 1999 to $3.6 billion.

Diagnostic Strategies

One or more of the following tests or procedures may be used to help confirm a diagnosis of IBD.

Blood tests to check for anemia or infection from bacteria or viruses. A fecal occult blood test may also be used to test for hidden blood in the stool. Although a blood test cannot confirm the presence of IBD, it can help rule out conditions that cause similar symptoms.

Endoscopic procedures such as colonoscopy, upper endoscopy, sigmoidoscopy, and capsule endoscopyare also used to diagnose IBD. These procedures provide clear and detailed views of the gastrointestinal tract and can help to differentiate between Crohns disease and ulcerative colitis.

Imaging testssuch as X-rays, CT scans, and MRI scans are often used in conjunction with endoscopic procedures. Imaging data can reveal signs of IBD in the lining of the intestines, such as tears, bleeding, inflammation, or an obstruction.

Current Therapies

There is no cure for IBD. The goal of treatment is to reduce the inflammation that triggers the signs and symptoms, thus providing relief as well as long-term remission and reduced risks of complications. IBD treatment usually involves either drug therapy or surgery. Classes of medications used to treat IBD include anti-inflammatories, immunosuppressants, biologics and antibiotics.

Anti-inflammatory drugs are often the first step in the treatment of inflammatory bowel disease. Anti-inflammatories include corticosteroids and aminosalicylates (5-ASA), such as Allergans mesalamine (Asacol HD, Delzicol, others), Salix Pharmas Colazal (balsalazide) and Uceris (budesonide) and AstraZeneca's Entocort EC.

Immune system suppressor medications are used to stem the immune response that releases inflammation-inducing chemicals in the intestinal lining. Some examples of immunosuppressant drugs include azathioprine (Azasan, Imuran), mercaptopurine (Purinethol, Purixan), cyclosporine (Gengraf, Neoral, Sandimmune) and methotrexate (Otrexup, Rasuvo, Rheumatrex Dose Pack, Trexall, Xatmep).

Tumor necrosis factor (TNF)-alpha inhibitors, or biologics, are a group of medicines that suppress the body's natural response to tumor necrosis factor (TNF), a protein produced by white blood cells that is involved in early inflammatory events. Examples include AbbVie's Humira (adalimumab), Janssen's Simponi (golimumab), UCB's Cimzia and infliximab (Remicade, Inflectra). Other commonly used biologic therapies are Tysabri (natalizumab), Entyvio (vedolizumab) and Stelara (ustekinumab).

Antibiotics may be used in addition to other medications or when infection is a concern. Frequently prescribed antibiotics include ciprofloxacin (Cipro) and metronidazole (Flagyl).

Drug Pipeline

A variety of targeted therapies are currently being explored through clinical trials. In addition to TNF-alpha inhibitors, aminosalicylates and glucocorticosteroids, common targets and mechanisms include:

As of October 2019, there are 730 clinical trials (not yet recruiting/active/active, no longer recruiting) listed on clinicaltrials.gov. Of these trials, 268 are in the United States and the remainder are across global sites. When broken down by phase, there are 63 trials in phase I, phase II has 132 trials, 101 phase III trials and the remainder are in phase IV or are other types of studies, including behavioral modification or observational studies, those using dietary supplements and various devices.

The IBD market contains a mixture of big and smaller sized pharma and biotech companies. The market share is dominated by Janssen with 5 drugs in all phases (I-III) and Pfizerwith 5 drugs in phase I and II. The remaining companies include Roche in collaboration with Genentechwith three drugs in phases I-III and AbbVie with three drugs in phase II, II/III and III.

The following analysis of selected drugs in the pipeline will briefly discuss how each drug works and where it is in clinical trials. This information was up to date as of October 2019. Any text in italics represents failed or terminated trials.

Note: This section is not meant to be completely comprehensive and may unintentionally exclude some drugs in development or clinical trials, especially those trials outside of the United States.

Janssen has five drugs in development for IBD: golimumab, ustekinumab, JNJ-64304500 (JNJ-4500/ IPH-2301/NN-8555), JNJ-67864238, guselkumab monotherapy and guselkumab combined with golimumab.

Pfizer also has five drugs in development for both Crohns disease and ulcerative colitis.

This trial was suspended in July due to an interruption in the supply of the radiolabeled material.

A phase II trial is underway to determine if PF-06687234 is effective and safe as add-on therapy to infliximab in subjects with active ulcerative colitis who are not in remission.

Genentech/Roche has three treatments in development for IBD.

A phase I trial is evaluating etrolizumab in pediatric patients of 4 to <18 years of age with moderate to severe ulcerative colitis (UC) or with moderate to severe Crohn's disease (CD).

Genentechs Phase III Etro Studies program is also underway, assessing its safety and efficacy in both UC and Crohns. During this trial, etrolizumab will be compared to other currently the U.S. FDA-approved drugs, namelyRemicade (infliximab) and Humira (adalimumab), and to placebo. There are five planned trials for patients with UC, and two for thosewith CD, to be carried out in the U.S., as well as one non-U.S. based trial in UC patients. All these studies arecurrently recruiting participants.

AbbVie has three drugs in development for inflammatory bowel disease.

Phase I

Aevi Genomics has partnered with Kyowa Kirin for AEVI-002, a human monoclonal antibody that binds an inflammatory protein found in intestinal tissue called LIGHT. The trial is evaluating AEVI-002 for severe pediatric-onset Crohns disease. Initial data are expected in the second half of 2019.

Assembly Biosciences is conducting a phase IB clinical trial of its lead investigational live biotherapeutic product (LBP) candidate, ABI-M201, in patients with mildly to moderately active UC. ABI-M201 is comprised of a dened consortium of gut commensal bacterial strains, specically selected based on their functional attributes to target key aspects of disease biology. Assembly and Allergan have entered into a collaboration to jointly develop LBP compounds for Ulcerative colitis (UC), Crohns Disease and Irritable Bowel Syndromes. ABI-M201 is the first LBP candidate under this collaboration.

Enteromeis partnered with Takeda for the co-development and co-commercialization of EB8018 (TAK018), a first-in-class, non-systemic, orally administered small molecule. EB8018 is specifically designed to remain gut-restricted and to block bacteria expressing the bacterial virulence factor, FimH, a key inducer of the inflammatory cascade in the intestine, thereby decreasing intestinal inflammation in patients with Crohns disease. Preliminary data from a Phase Ib clinical trial of EB8018 for the treatment of Crohns disease is expected in 2019. Takeda is planning to commence a phase II trial in November of 2019.

Gossamer Bio is developing GB004, an oral HIF-1 stabilizer. Patient enrollment in a Phase Ib study of active mild-to-moderate ulcerative colitis (UC) began during the second quarter, and the Company expects topline results from the study in the first half of 2020.

Intralytix, Inc.'s EcoActive bacteriophage therapy targeting adhesive invasive E. coli (AIEC) in Crohn's disease patients has entered a Phase I/IIa clinical trial at the Icahn School of Medicine at the Mount Sinai Hospital in New York, NY. The presence of AIEC in the intestines is associated with worsening inflammation in this disease. The trial will measure the effect of oral phage administration on the AIEC (CFU/g) in stools of patients receiving phages versus patients receiving a placebo.

Nutrition Science Partners Limited, a 50/50 joint venture between Chi-Med and Nestl Health Science SA, is assessing HMPL004-6599, a proprietary botanical (Andrographis paniculate) with in vitro inhibitory activity against TNF-, IL-1 and NF-B. A pilot study of A. paniculata extract (HMPL-004) indicated similar efficacy to mesalamine for ulcerative colitis. HMPL004-6599 is an enriched/purified re-formulation of HMPL-004, an anti-inflammatory with anti-TNF properties. A phase 3 trial of the original formulation was evaluating its efficacy in ulcerative colitis. However, the trial was terminated in 2014 following an interim analysis showing the endpoint was unlikely to be reached.

OSE Immunotherapeutics has a pipeline of drugs aimed at immune-oncology and autoimmune diseases, one of which is OSE-127. OSE-127 is a monoclonal immunomodulatory antibody targeting the CD127 receptor, the alpha chain of the interleukin-7 receptor (IL-7R) that induces a powerful antagonist effect on effector T lymphocytes. The blockage of IL-7R prevents the migration of pathogenic T lymphocytes while preserving regulator T lymphocytes (1,2,3,4) which have a positive impact in autoimmune diseases. The first patients were dosed in a phase I trial in December of 2018.

Phase II

AbGenomicsis evaluating neihulizumab/AbGn-168H, a humanized therapeutic antibody with a unique mechanism of action, which preferentially induces apoptosis of late-stage activated T cells. This activated-T cell apoptosis-inducing antibody effectively eliminates chronic pathogenic T cells while fully maintaining host defense. A phase II trial is currently enrolling up to 40 patients with moderate to severe active ulcerative colitis and who have failed or are intolerant to anti-TNF and/or anti-integrin therapy.

Allergan's brazikumab is an anti-inflammatory with the potential to curb inflammation by blocking the proinflammatory molecule interleukin-23. In a double-blind, placebo-controlled study of 119 adults with moderate to severe CD who had failed treatment with tumor necrosis factor antagonists, 8 and 24 weeks of treatment were associated with clinical improvement. Higher baseline serum concentrations of IL22 were associated with a greater likelihood of response to treatment compared withplacebo.

Two trials are currently underway, and both are utilizing a personalized approach to evaluate the role of biomarkers, such as IL22, in predicting treatment response to brazikumab.

The phase II EXPEDITION trial is comparing brazikumab to placebo or Entyvio (vedolizumab) in approximately 375 patients with moderately to severely active ulcerative colitis.

The phase IIb/III INTREPID trial is currently enrolling up to 1,140 patients with moderate to severe Crohns disease to evaluate brazikumab versus placebo and versus an active comparator, Humira (adalimumab).

Boehringer Ingelheimis developing Spesolimab (BI 655130) is a monoclonal antibody that blocks the action of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system that may play a role in many inflammatory diseases. Proof of concept was demonstrated in a phase I study in patients with generalized pustular psoriasis, an IL36-mediated skin disease.

Four clinical trials are currently underway for both Crohns disease and ulcerative colitis.

Bridge Biotherapeutics is evaluating BBT-401, a GI-tract restricted small molecule inhibitor of Pellino-1. Pellino-1 is a ligase acting as a critical mediator for a variety of immune receptor signaling pathways, including Toll-like receptors, interleukin-1 receptor and T-cell receptors. BBT-401 was proved to be well-tolerated and safe in a phase I study in 80 healthy volunteers. In addition, the PK data demonstrated its key feature of no or minimal systemic exposure. A randomized, placebo-controlled, dose-escalation phase II trial is recruiting 48 patients with active ulcerative colitis.

Bristol-Myers Squibbs BMS-986165 is an oral, selective tyrosine kinase 2 (TYK2) inhibitor. TYK2, an intracellular signaling kinase, mediates cytokine-driven immune and pro-inflammatory signaling pathways that are critical in the cycle of chronic inflammation central to immune-mediated diseases. A phase II trial in patients with psoriasis was successfully completed in 2018. Two phase II trials are currently underway, one each in Crohns disease and ulcerative colitis.

Eli Lillyis progressing with mirikizumab (LY 3074828), a humanized IgG4 monoclonal antibody that binds and inhibits the p19 subunit of interleukin 23. Positive results have been reported from the phase II SERENITY trial in patients with Crohns disease and from a phase II trial for ulcerative colitis. Both trials showed that patients treated with mirikizumab achieved significantly greater rates of clinical and endoscopic remission at 12 weeks compared to placebo. Lilly is currently conducting six clinical trials in phase II and phase III, for both Crohns disease and ulcerative colitis.

Gilead, in global collaboration with Galapagos NV, is studying filgotinib, a highly selective JAK1 inhibitor. Results from a phase II study, FITZROY, were published in The Lancet in December of 2017. The study examined the efficacy and safety of filgotinib for the treatment of active moderate-to-severe Crohn's disease. Data showed filgotinib induced clinical remission in significantly more patients compared with placebo and had an acceptable safety profile. There are currently seven active phase II and III trials underway, including MANTA, DIVERSITY, DIVERGENCE2 and SELECTION 1.

Immunic Therapeutics is developing IMU-838, an oral tablet formulation of a small molecule drug (vidofludimus calcium), which inhibits dihydroorotate dehydrogenase (DHODH). Immunic completed two phase I studies in 2017, which evaluated single or repeated once-daily doses of IMU-838 in healthy volunteers. Results supported the tolerability of repeated daily dosing of up to 50 mg of IMU-838. A phase II trial, CALDOSE 1, is underway for ulcerative colitis.

Incyteis evaluating itacitinib, an orally administered small molecule Janus kinase 1 (JAK1) inhibitor. A double-blind, dose-ranging, placebo-controlled phase II trial is currently recruiting patients with moderate to severe ulcerative colitis.

Landos Biopharma's lead program is BT-11, an orally active, locally acting small molecule therapeutic that binds to LANCL2. In preclinical studies, it was shown to exert potent anti-inflammatory effects and was safe and well-tolerated with no dose-limiting toxicities in a phase I study. A phase II trial began in August of 2019 in patients with mild to moderate ulcerative colitis and is underway in Europe and the U.S. A second phase II study is anticipated to begin shortly in patients with moderate to severe Crohns disease, also in Europe and the United States.

Reistone Biopharma is developing SHR-0302, an orally administered selective JAK1 inhibitor. Two trials are underway: a phase II trial evaluating SHR0302 compared to placebo in patients with moderate to severe active ulcerative colitis and a phase II trial evaluating SHR-0302 in patients with moderate to severe active Crohn's Disease.

Seres Therapeutics is advancing with SER-287 is an oral capsule developed using Seres proprietary microbiome therapeutics platform. It is biologically-sourced and contains a consortium of live and diverse bacterial spores. SER-287 was designed to reduce the triggers of immune activation rather than suppress the immune system. Results from a phase Ib trial in patients with ulcerative colitis showed SER-287 microbiome treatment resulted in a dose-dependent benefit in clinical remission rates, and an improvement in endoscopic scores. A phase II trial, ECO-RESET, is currently recruiting 200 adults, age 18-80, with active mild-to-moderate ulcerative colitis.

Sublimity Therapeutics has developed an oral formulation of cyclosporine, referred to as ST-0529, using Sublimitys proprietary SmPill delivery system. Unlike conventional oral or intravenous cyclosporine, SmPill technology enables precise delivery of cyclosporine directly into diseased tissue in the colon, thus minimizing systemic exposure and unwanted side effects. In a phase IIa study ST-0529 was safe, well-tolerated and showed a numerically higher difference in remission rates in patients with mild-to-moderate ulcerative colitis compared to placebo after only four weeks of treatment. A phase IIb study, AURORA (CYC-202), is currently recruiting 280 subjects with moderately to severely active ulcerative colitis.

Theravanceand Janssen Biotech are collaborating on the development of TD-1473, an orally administered and intestinally restricted pan-Janus kinase (JAK) inhibitor. Data from a phase Ib trial demonstrated that four weeks of TD-1473 treatment produced signals of clinical, histologic, and biomarker activity in patients with moderately-to-severely active ulcerative colitis. A phase II trial, DIONE, is evaluating TD-1473 in subjects with moderately-to-severely active Crohn's Disease with up to 42 weeks of treatment. A phase II/III trial, RHEA, is evaluating the efficacy and safety of induction and maintenance therapy with TD-1473 in subjects with moderately-to-severely active ulcerative colitis with up to 60 weeks of treatment.

Phase III

Arena Pharmaceuticalsis developing etrasimod, a next-generation, once-daily, oral, highly selective sphingosine 1-phosphate (S1P) receptor modulator designed for optimized pharmacology and engagement of S1P receptor 1, 4 and 5, which may lead to an improved efficacy and safety profile. The phase II OASIS trial met primary and all secondary endpoints with statistical significance for patients with ulcerative colitis receiving 2 mg dose of etrasimod for 12 weeks. Based on these results, Arena initiated the global phase III ELEVATE UC program, which consists of two worldwide clinical trials and an open-label extension study. In addition, planning is underway for a phase II/III trial in patients with Crohns disease.

Celgene's ozanimod targets the sphingosine-1-phosphate (S1P)-1 and -5 receptors. Results from phase II trials were reported in October of 2017. In the phase II STEPSTONE open-label study, ozanimod demonstrated meaningful clinical and endoscopic improvements in patients with moderately to severely active Crohn's disease at week 12. In the phase II TOUCHSTONE open-label extension study, ozanimod continued to demonstrate clinically meaningful results in moderately to severely active ulcerative colitis across multiple measures of disease activity through week 92. Celgene currently has nine active studies underway for both forms of IBD.

EA Pharma, a joint venture between Eisai Groups gastrointestinal disease business and Ajinomoto Group, is evaluating AJM300 (carotegrast methyl) an orally-active small molecule that antagonizes the 4 integrin receptor. A phase III trial was initiated in June of 2018 to evaluate AJM300 in patients with active ulcerative colitis.

RedHill Biopharma is evaluating RHB-104, a formulation of the generic antibiotics clarithromycin, rifabutin and clofazimine that is designed to treat Crohns disease by wiping out Mycobacterium avium paratuberculosis (MAP) infection.Some studies have linked infection with the bacterium to Crohns disease. In July of 2018, positive results were reported from the MAP US study, which evaluated RHB-104 for Crohns disease. The primary endpoint was successfully achieved, with superior remission rate at week 26 in patients treated with RHB-104 versus placebo.

Shire, a Takedacompany, has moved into phase III development with ontamalimab (SHP 647), a fully human IgG2 monoclonal antibody that targets mucosal addressin cell adhesion molecule (MADCAM1). Shire licensed the drug from Pfizer in 2016. Results from a phase II trial in patients with ulcerative colitis were published in the journal The Lancet in 2017 and demonstrated the treatment was safe and well-tolerated in this patient population, and better than placebo for induction of remission. Seven phase III trials are currently recruiting patients with both ulcerative colitis and Crohns disease.

Preclinical Candidates

The following is a sampling of companies that are preparing to develop and investigate drugs and other therapeutics in the preclinical laboratory setting. Data gathered from these preclinical trials will be used to determine whether the drug/therapy will move forward with clinical testing in humans.

AntaraLife Science's lead human health product is referred to as GaRP (Gastrointestinal ReProgramming). The GaRP product is a microbiome-targeted multi-component dietary supplement that has been designed to address the primary underlying factors associated with IBD and IBS. It is being positioned as an adjunct to existing therapies and not to replace existing prescription medications. Antara has completed the preclinical program, which provided strong scientific evidence that GaRP can combat the underlying causes of chronic bowel conditions, including inflammation and dysbiosis of the microbiome. Antara anticipates submitting regulator applications to move into clinical trials by the end of 2019.

Prometheus Bioscienceshas partnered with Takeda to discover, develop, and commercialize targeted therapies for inflammatory bowel disease. The collaboration combines the proprietary bioinformatics discovery platform and companion diagnostic tools developed by Prometheus Biosciences with Takeda's expertise in gastroenterology and drug development, in order to discover and advance up to three targeted IBD therapeutics and companion diagnostics.

Read this article:
Inflammatory Bowel Disease Insight Report: Current Therapies, Drug Pipeline and Outlook - BioSpace

BRUNSWICK Bradford Thomas Martin, 33, passed away on Nov. 8, 2019 due to complications from a rare genetic disease, Dyskeratosis Congenita.

Brad was born August 15, 1986 in Newark, Del. He and his family moved to Maine in January of 1991. He attended the Yarmouth school system from kindergarten through his high school graduation in 2005. He was a graduate of Keene State College, Class of 2009, with a degree in Occupational Health and Safety Studies.

He was a dedicated volunteer, Badge No. 356, for the Yarmouth Fire Department for over nine years.

He had been a Sugarloafer since 1997 where he participated in group ski lessons, the Junior Professional Program, and was an instructor for several years. He recently became a member of the Sugarloaf Ski Club. Brad was a very accomplished skier and loved it more than anything. He spent every winter at Sugarloaf where he and his friends regularly camped out at our house. Sugarloaf was home for Brad.

He was a passionate sports fan of multiple sports, too many to list, but especially his beloved Boston Bruins, Boston Red Sox, and New England Patriots. He enjoyed playing golf and traveling. In 2015 he traveled alone extensively throughout southeast Asia and Europe for more than three months. He was a foodie and enjoyed trying new foods. He also loved swimming and boating at Thompson Lake, in Poland, MaIne, where he spent most of his childhood summers.

Along with Brads passion for sports, he was an avid reader including several daily newspapers.

Brad was his own person. He had strong principles and opinions and lived his life on his terms. Brad positively touched so many lives in so short a time. His curiosity about the world, his intolerance for bullshit, and his willingness to always stick up for the underdog is why he had so many friends across all ages.

Brad is survived by his parents Donna and Bruce of Brunswick and Port St. Lucie, Fla.; his brother Garrett of South Portland; his girlfriend, Cyndee DAgostini of Portland and Orange County, Calif.; as well as many aunts, uncles, and cousins. He is predeceased by his grandparents Claudette and Normand Allard, formerly of Port St. Lucie, Fla. and Central Falls, R.I.; Dorothy and Norman Martin, formerly of Cumberland, R.I.; and uncle, John Martin, formerly of Harrisville, R.I.

In honor of Brads journey through life, there will be a celebration of his life at The Rack in Carrabassett Valley, ME in Spring 2020.

In memory of Brad, donations may be made to:

Childrens Medical Center Corporation

A Massachusetts

Charitable Corporation

300 Longwood Ave.

Boston, MA 02115

Tax ID: 04-1174680

To Be Used For

Dyskeratosis Congenita Research

In The Stem Cell

Transplant Center

Bone Marrow Study of The PARN Mutation

or; Camp Sunshine

35 Acadia Rd.

Casco, ME 04015

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Obituary: Bradford Thomas Martin - Press Herald

Florida Poly assistant professor Dr. Ajeet Kaushik has received the 2019 USERN Prize in biological sciences, an international award recognizing his work in the field of nanomaterials for the detection and treatment of diseases.

LAKELAND, Fla., Nov. 18, 2019 /PRNewswire-PRWeb/ -- Dr. Ajeet Kaushik is determined to make detecting and treating diseases easy, accessible, and precise through the use of nanomaterials for biosensing and medicine.

His extensive work and resolute desire to improve the delivery of healthcare has earned Kaushik the prestigious Universal Scientific Education Research Network (USERN) Prize. He was named a laureate in the field of biological sciences during the group's fourth annual congress on Nov. 8 in Budapest, Hungary.

USERN, a non-governmental, non-profit organization and network dedicated to non-military scientific advances, is committed to exploring science beyond international borders.

"I was speechless for a while," said Kaushik, who is an assistant professor of chemistry at Florida Polytechnic University.

Kaushik did not attend the awards ceremony in person but did submit a video to be played at the event. He was among hundreds vying for the prize and one of five people who were recognized in different areas of study.

His submitted project, Nano-Bio-Technology for Personalized Health Care, focuses on using nanomaterials to create biosensors that will detect the markers of a disease at very low levels.

"Biosensing is not a new concept, but now we are making devices that are smarter and more capable," Kaushik said.

He cited the recent zika virus epidemic that affected pregnant women and their fetuses, leading to significant health complications upon birth. "There was a demand to have a system that could detect the virus protein at a very low level, but there was no device. There was no diagnostic system," he said.

Kaushik worked on the development of a smart zika sensor that could detect the disease at these low levels. "The kind of systems I'm focusing on can be customized in a way that we carry like a cell phone and do the tests wherever we need to do them," he said.

In addition to using nanotechnology for the detection of diseases like zika, his research on nanoparticles is advancing efforts to precisely deliver medicine to a specific part of the body without affecting surrounding tissue or other parts of the body.

"The drugs we use now do not go only where they need to go, or sometimes they have side effects. We are treating one disease but creating other symptoms," Kaushik said. "I'm exploring nanotechnology that can carry a drug, selectively go to a place, and release the drug so we avoid using excessive drugs."

This nanomedicine could be used to precisely target brain tumors or other difficult-to-treat conditions. He has published papers in scientific journals about this work and also holds multiple patents.

"My whole approach is using smart material science for better health for everybody, which is accessible to everybody everywhere," Kaushik said.

In addition to his USERN prize, Kaushik was named a USERN junior ambassador for 2020 and will work to advance the organization's mission in the United States.

For the most recent university news, visit Florida Poly News.

About Florida Polytechnic University:

Florida Polytechnic University is accredited by the Southern Association of Colleges and Schools Commission on Colleges and is a member of the State University System of Florida. It is the only state university dedicated exclusively to STEM and offers ABET accredited degrees. Florida Poly is a powerful economic engine within the state of Florida, blending applied research with industry partnerships to give students an academically rigorous education with real-world relevance. Connect with Florida Poly online at http://www.floridapoly.edu.

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Nanotechnology for disease diagnosis and treatment earns Florida Poly professor international award - Yahoo Finance

Nes-Ziona, Israel, Nov. 12, 2019 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (ENLV), a clinical-stage immunotherapy company, today announced that company management will make a corporate presentation and host 1x1 meetings at the 10th Annual Jefferies 2019 London Healthcare Conference, being held in London on November 20 & 21. The presentation is scheduled for November 20 2019 at 8:00am local time.

ALLOCETRATMby Enlivex was designed toprovide a novel immunotherapy mechanism of actionthat targets life-threatening clinical indications that are defined as unmet medical needs, includingprevention or treatment of complications associated with bone marrow transplantations (BMT) and/or hematopoietic stem cell transplantations (HSCT); organ dysfunction and acute multiple organ failure associated with sepsis; and enablement of an effective treatment of solid tumors via immune checkpoint rebalancing.

Enlivex announced on November 4, 2019 positive interim safety and efficacy data from an ongoing trial of off-the-shelf universal Allocetra in patients with severe sepsis. The interim analysis comparing Allocetra-treated patients with 37 severe sepsis patients with equivalent source of infection and disease severity who were hospitalized at the same hospital, demonstrated the potential of Allocetra as therapy for prevention of sepsis-associated organ failure and mortality.

For more information about the 10th Annual Jefferies 2019 London Healthcare Conference, visit http:// https://www.jefferies.com/IdeasAndPerspectives/Conferences/325/112019.

ABOUT ENLIVEXEnlivex is a clinical stage immunotherapy company, developing an allogeneic drug pipeline for immune system rebalancing. Immune system rebalancing is critical for the treatment of life-threatening immune and inflammatory conditions which involve an out of control immune system (e.g. Cytokine Release Syndrome) and for which there are no approved treatments (unmet medical needs), as well as solid tumors immune-checkpoint rebalancing. For more information, visit http://www.enlivex.com.

ENLIVEX CONTACT:Shachar Shlosberger, CFOEnlivex Therapeutics, Ltd.shachar@enlivex-pharm.com

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Enlivex Therapeutics To Present At The 10th Annual Jefferies 2019 London Healthcare Conference - Yahoo Finance

Dr Alexandra Butler/Principal Investigator at Qatar Biomedical Research Institute, part of Hamad Bin Khalifa University, talks about efforts to tackle

What is diabetes?Diabetes is a disease that occurs when there is an inability to produce adequate amounts of insulin (a hormone produced by beta cells in the pancreas) to maintain blood glucose levels within normal limits, and it represents a major medical, social, and economic burden worldwide.The prevalence of diabetes has reached epidemic proportions globally, with 424.9mn affected adults aged 20-79, representing 8.8% of the global adult population.This figure is predicted to rise to 628.6mn by 2045, affecting almost 10% of the worldwide adult population. Type 2 diabetes accounts for 90 to 95% of diabetes cases, with type 1 diabetes accounting for about 5%. However, half of those with type 2 diabetes have not been diagnosed.In Qatar, 20% of the population is affected by diabetes (type 2 accounting for 90% of those) with a projected rise to 24% in the next two decades. Diabetes in Qatar affects all segments of society, from the young to the elderly, and places a significant social and economic burden on Qatari society.What is the difference between type 1 and type 2 diabetes, and are there other types?The reasons underlying the inability to produce adequate amounts of insulin depend upon the type of diabetes.Type 1 is essentially a total insulin deficiency due to the destruction of beta cells by the bodys immune system. Type 2 is a complex disease characterised by insulin resistance and insulin deficiency. The degrees of resistance and deficiency vary, but insulin deficiency is key to developing diabetes.Gestational diabetes is a form of diabetes that can develop during pregnancy as pregnancy is a state of increased insulin resistance and these women have a higher risk of developing type 2 diabetes later in life.There are also genetic defects of beta cell function and insulin action, such as Maturity Onset Diabetes of the Young (MODY).Some conditions, such as diseases of the exocrine pancreas, can cause secondary diabetes as can certain drugs, corticosteroids being a prime example.

What are the symptoms of diabetes?Those with type 1 diabetes may experience thirst, a frequent need to urinate, weight loss, fatigue, blurred vision, ketoacidosis (a life-threatening conditions where the body starts breaking down fat at a rapid rate), and infections.Type 2 diabetes can lead to thirst, frequent urination, and malaise, but symptoms are usually much milder and many individuals with type 2 diabetes do not know they have the disease until they are tested.In certain circumstances, it can be difficult to distinguish between type 1 and type 2 diabetes (see Table 1). Distinguishing the type of diabetes can be particularly difficult in younger patients who are treated with insulin, but clinically appear to have type 2 diabetes, and older patients with late onset of diabetes who require insulin and share characteristics of patients with type 1 diabetes where Latent Autoimmune Diabetes of Adulthood (LADA) should be considered.What role is Qatar Biomedical Research Institute (QBRI) playing in relation to diabetes research?The genetic risks for type 2 diabetes are likely to be different for Qataris compared to other Asians and Europeans. Therefore, identification of factors that are associated with the high prevalence of type 2 diabetes in the Qatari population is an important focus of ongoing research at the Diabetes Research Center (DRC) at Qatar Biomedical Research Institute (QBRI), part of Hamad Bin Khalifa University.Diabetic complications are costly, extremely difficult to manage, and lead to increased mortality rates. Diabetic patients are also at increased risk of neurological impairment and certain forms of cancer, so identifying biomarkers that predict the future development of diabetes and/or its related complications is imperative.Accordingly, biomarker discovery is a major focus of the ongoing research at the DRC. With the advent of stem cells, the concept of mimicking nature to generate new beta-cells to reverse diabetes has emerged, although more work needs to be done to allow for safe and effective therapy.In collaboration with the Harvard Stem Cell Institute, the Stem Cell group at DRC is working towards developing safe and effective stem cell-derived therapy to meet the needs of diabetic patients in Qatar.The underlying causes of insulin resistance are not addressed by any current therapy.The DRC is also investigating the mechanisms of insulin resistance to identify new targets for drug discovery. The mechanisms underlying the susceptibility of kidney, retina, peripheral nerve and arteries to damage in diabetes are yet to be proven, but the DRC has made advances towards understanding these. The centre is paving the way towards improved biomarkers and focused treatments, or precision medicine for diabetes, in order to tackle this globally devastating disease.

Can we prevent or reverse diabetes if we follow a healthy lifestyle?In the Gulf region, the diabetes epidemic is largely fuelled by obesity and sedentary lifestyles. Type 1 diabetes is not reversible but type 2 is largely preventable and can be reversed in its earlier stages by adopting a healthy lifestyle, the mainstays of which include regular exercise, a healthy diet and maintaining a healthy weight.Anyone can suffer from diabetes and those with affected family members are at an increased risk. It is therefore a good idea to follow your doctors advice as to the frequency of testing for diabetes.

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Diabetes has reached epidemic proportions, yet many don't know they suffer from it - Gulf Times

Get your news fix on happenings outside the pool with the latest Beyond the Lane Lines. With each edition, we collect personal stories, little known facts and general items of interest from around the world. Read on and learn something new this week.

#1 Joseph Schooling Launches Skincare Line

Olympian Joseph Schooling took gold in the mens 100m fly at the 2016 Games in Rio and has been a superstar in his home nation ever since. The 24-year-old former Texas Longhorns latest accomplishment is taking place outside the pool, where the Singaporean just launched his own line of skincare products.

Teaming up with aesthetic doctor Dr. SM Yuen from Atlas Medical Laser & Aesthetics Clinic, Schooling has helped create a range of unisex beauty products that he hopes will help educate consumers on the importance of taking care of their skin.

My teammates used to laugh at me because I was the only one on the team who moisturized. We used to have our training [sessions] at the Toa Payoh pool where the chlorine level was super high. When I got out of the pool, my skin looked ashy and flaky. Our hair used to be bleached blonde and I had white patches all over my body and neck, said Schooling. So at age 11, taking care of my skin became a daily routine. It was something I had to do, otherwise, Id feel uncomfortable.

The result of Schoolings self-care experience isJS Orphic, a four-pronged skincare range made up of his daily grooming essentials: Pore Care Splash-Away cleanser, My Favourite Skin Hydrator, UV Shield + Skin Protector, and Stem Cell + Snail Waterfall Serum.

Quotes courtesy of CNA Lifestyle.

#2 Ellie Challis &Katie Shanahan Shortlisted for One-to-Watch Award

15-year-old British swimmerKatie Shanahan has been making a name for herself as of late, with the City of Glasgow swimmer reaping half of her nations gold medals at this years European Youth Olympic Festival in Baku, Azerbaijan.

For her efforts, as well as her future promise, teen Shanahan has been shortlisted out of approximately 1,000 candidates for the annual SportsAid One-to-Watch Award

Ive achieved so much over the last year, but the highlight has to be being nominated for awards like this one with SportsAid, said Shanahan of her nomination.

It was such an amazing opportunity to compete at the European Youth Olympic Festival. I didnt even expect to medal, let alone break any records, especially because it was such a high-level meet.

I was also lucky to race at Mare Nostrum in Barcelona, but I knew that one was going to be completely different. I was coming up against Olympians, Commonwealth, and European champions and I was so pleased to be almost as fast as some of the swimmers I have always looked up to.

As for Ellie Challis, she, too, has been nominated for the award, following a record-breaking 2019 in the pool. The 15-year-old busted out the SB2 class 50m breast World Record at the British Para Swimming International Meet in Glasgow.

Its lovely to be recognised for the One-to-Watch Award and for all my achievements. Its really nice and its that recognition at such a young age that makes it so special for me.

The winner of the One-to-Watch Award will be revealed at the charitys Celebrate the Next event on Tuesday, November 19th.

Quotes courtesy of Scottish Swimming.

#3 Cate Campbell Joins Melanoma Awareness Campaign

Olympic championCate Campbell averted a melanoma scare in November 2018 when the Aussie had a mole removed, only to find out it was indeed cancerous. Since then, C1 has been on the awareness train, helping fellow Australian citizens be more skin-conscious and educated when it comes to the effects of the sun.

Most recently, Campbell has been named as teh face of Melanoma Institute Australias Game On Mole campaign.

Says C1 of her ambassadorship, Im actually quite a private person, but I only went and got a skin check after running into someone who had their own melanoma story to tell.

If I hadnt met them on the off chance, would I have gone for that skin check?

Id like to think so, but I cant be sure and I may still be walking around with melanoma in my arm possibly heading into my bloodstream, which could have caused more complications than just a scar.

So I felt like I had an obligation to share my story and to be a part of the Game On Mole campaign.

If by sharing you can save one person, or if one person hears the story and goes and gets their skin checked, then I think that thats a win.

Quotes courtesy of Perth Now.

#4 Katie Ledecky Joins Team Visa

Worldwide Payment Technology Partner of the Olympic and Paralympic Games, Visa, announced its roster of athletes on Team Visa Tokyo 2020. The more than 70 athletes span 30 sports and represent 43 nations, the most in Team Visa history.

As one of the longest-standing partners of the Olympic and Paralympic Games, Visa looks forward to Tokyo 2020. Set to be the largest Games yet, Tokyo 2020 will feature 7,000 hours of broadcast programming and three billion minutes of streamed content, said Chris Curtin, chief brand and innovation marketing officer, Visa.

Our Team Visa Tokyo 2020 roster reflects The Games themselves: globally diverse, representative of new emerging sports and larger than ever. We are proud to reveal this years Team Visa roster and support these athletes as go for Gold in Tokyo.

Founded in 2000, Team Visa has championed nearly 500 athletes. Each has been selected based on athletic achievements, character and personal journey, and is representative of many of Visas brand values and priorities.

American Olympic icon Katie Ledecky has been named to the Visa roster, along with Adam Peaty of Great Britain, Florian Wellbrock of Germany, Daiya Seto and Kanako Watanabe of Japan, Dmitriy Baladin of Kazakhstan, Tatjana Schoenmaker of South Africa, Gregorio Paltrinieri of Italy, Daniel Dias of Brazil, Aurlie Rivard of Canada,Teresa Perales of Spain andYelyzaveta Mereshko of Ukraine.

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Beyond The Lane Lines: Schooling Launches Skincare, Ledecky Joins A New Team - SwimSwam

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

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The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

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Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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Myelofibrosis Treatment Market To Witness an Outstanding Growth During 2016-2022 - Zebvo

Aplastic anemia is a medical condition that damages stem cells in a person's bone marrow. These cells are responsible for making red blood cells, white blood cells, and platelets, which are vital to human health.

Doctors believe various conditions can cause aplastic anemia, while the disease itself ranges in severity from mild to life threatening.

Medical advancements mean that aplastic anemia is more treatable than ever. In this article, learn more about this rare medical disorder.

When a person has aplastic anemia, their bone marrow does not create the blood cells it needs. This causes them to feel ill and increases their risk of getting infections.

Doctors also call aplastic anemia bone marrow failure.

Doctors do not know exactly how many people in the United States have aplastic anemia.

According to the National Organization for Rare Disorders (NORD), doctors diagnose approximately 500 to 1,000 cases every year. It is most common in older children, teenagers, and young adults.

Researchers believe that most cases of aplastic anemia are due to the immune system attacking healthy bone marrow cells, according to NORD.

Doctors have also identified some of the possible causes of this immune system response, including:

However, doctors usually cannot pinpoint the underlying cause in most aplastic anemia cases.

When the cause is unknown, doctors refer to the condition as idiopathic aplastic anemia.

Symptoms of aplastic anemia include:

These symptoms may be severe. Some people may have heart-related symptoms, such as chest pain.

A doctor will start by asking about a person's symptoms and their medical history.

They will usually use a blood test known as a complete blood count (CBC) to evaluate a person's red blood cells, white blood cells, and platelets. If all three of these components are low, a person has pancytopenia.

A doctor may also recommend taking a sample of bone marrow, which comes from a person's pelvis or hip.

A laboratory technician will examine the bone marrow. If a person has aplastic anemia, the bone marrow will not have typical stem cells.

Aplastic anemia can also have similar symptoms as other medical conditions, such as myelodysplastic syndrome and paroxysmal nocturnal hemoglobinuria. A doctor will want to rule out these conditions.

Sometimes, a person with other medical conditions can develop aplastic anemia. These conditions include:

If a person has these conditions, a doctor will recognize that they are more likely to get aplastic anemia.

Doctors usually have two goals when treating aplastic anemia. The first is to reduce the person's symptoms, and the second is to stimulate the bone marrow to create new blood cells.

People with aplastic anemia can receive blood and platelet transfusions to correct low blood counts.

A doctor may also prescribe antibiotics as a person needs white blood cells to fight infections. Ideally, these drugs will prevent infections until a person can build more new white blood cells.

Doctors usually recommend a bone marrow transplant to stimulate new cell growth in the long term.

For this, a doctor may first prescribe chemotherapy medications to kill off abnormal bone marrow cells that are affecting a person's overall bone marrow function.

Next, a doctor performs a bone marrow transplant by injecting the bone marrow into a patient's body.

Ideally, the individual will receive bone marrow from a close family member. However, even a sibling donor is only a match in 2030% of cases.

People can also receive bone marrow from someone who is not related to them if doctors can find a compatible donor.

Some people cannot tolerate bone marrow transplants, especially older adults, and those having difficulty recovering from chemotherapy. Others may not be able to find a donor that matches their bone marrow. In these instances, a doctor can prescribe immunosuppressive therapy.

Immunosuppressive medicines suppress the immune system, which ideally stops it from attacking healthy bone marrow cells. Examples of these medications include antithymocyte globulin (ATG) and cyclosporine.

According to NORD, an estimated one-third of people with aplastic anemia do not respond to immunosuppressive drugs.

If this is the case, doctors may consider other treatments, such as hematopoietic stem cell transplantation and a medication called eltrombopag (Promacta).

Those with aplastic anemia may face complications due to their disease as well as their treatment.

Sometimes, a person's body rejects a bone marrow transplant. Doctors call this graft-versus-host disease or GVHD.

GVHD can make a person feel extremely ill and can cause symptoms that include:

According to 2015 research, about 15% of aplastic anemia patients who receive immunosuppressive therapy will develop myelodysplastic syndromes or acute myeloid leukemia.

These conditions can develop years after a person's initial diagnosis.

Some people do not respond to aplastic anemia treatments. When this is the case, they are more vulnerable to infections that can be life threatening.

The outlook for a person with aplastic anemia depends on many factors, including:

A doctor will discuss a person's treatment outlook when considering the various therapies.

Aplastic anemia damages stem cells in a person's bone marrow. The bone marrow makes red blood cells, white blood cells, and platelets, which are all essential for the body.

A person with aplastic anemia may experience severe anemia symptoms. Treatment may include chemotherapy, stem cell transplants, and immunotherapy.

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What is aplastic anemia? Symptoms, causes, and treatment - Medical News Today

Coming to terms with my [end stage kidney failure] has been one of the hardest things I have had to accept. The mental turmoil and anxiety replay daily in my mind, over and over. Naomi Adams, a British-Caribbean mother of two, was diagnosed with the critical condition just before her birthday last year. As her health continues to deteriorate, the nurse in her thirties knows she needs an organ transplant sooner rather than later.

For Naomi, the risks are much higher if she fails to get a kidney transplant. One in five people who died last year while waiting for an organ transplant came from a black, Asian or ethnic minority background (BAME), according to 2018 statistics from NHS Blood and Transplant.

Naomi still remembers being told the news. As the doctor informed her of the diagnosis, her mind drifted back to the life she had. It was one of the most devastating events of my life The future lacked clarity, certainty. Hopes and dreams crushed, she says. I was devastated, in total shock. End-stage renal failure is a life-changing condition. The impact it had on my mental and physical well-being has been enormous for such a short amount of time.

She is just one of the hundreds of patients who belongs to an ethnic minority group in the UK. In total, more than a quarter of those on the transplant waiting list are from BAME communities, despite representing around 11 percent of the UK population.

Different blood and tissue types across racial groups means that finding a donor from a similar ethnic background could be a matter of life or death for those in need, especially since BAME patients are more vulnerable to developing particular illnesses that can lead to organ failure (such as high blood pressure and diabetes). That makes it much harder to match patients and donors who are genetically similar.

Given the circumstances, many have described the problem as a silent crisis that has reached a tipping point. In 2018, Labour MP Eleanor Smith led a review into the situation. The resulting report, titled Ending the Silent Crisis, published a series of recommendations to help tackle the low number of ethnic minority donors.

About her review, Smith wrote: Everyone has a role to play, whether that be the Government, NHS Blood and Transplant, the community itself, or MPs. We need to foster more superheroes in our community, who selflessly donate blood.

Without a doubt, greater community participation is urgently needed. According to the report, fewer than 5 percent of donors who gave blood in the last year, alongside 7 percent of deceased organ donors, were from BAME communities. Only 61 percent of BAME patients in need of a stem cell transplant find a suitably matched donor, in comparison with 96 percent of white patients.

For patients such as Naomi, a lack of dialogue surrounding donation within her community means that she not only had to adjust to a life-changing condition, but also needed to have difficult conversations with her family about the possibility of donating one kidney to help save her. She asked, but to no avail.

In my case, my family has not been forthcoming in being tested [and] a friend who stepped forward to be tested wasn't a blood match. It's quite difficult to put into words the emotions I felt. It's bittersweet.

But there is still a glimmer of hope. A new organ donation system comes into effect next year in England to help reduce the number of people waiting for a life-saving transplant. Currently, there is a voluntary opt-in system, but under the new legislation, known as Max and Keiras Law, consent will be presumed for adults unless they opt out. (Family members can still block the donation if relatives did not give explicit consent or if the donation will cause distress to their family.)

Orin Lewis OBE, the co-founder of the African Caribbean Leukaemia Trust, said the new opt-out scheme could break down the present status quo. Max and Keiras Law gives hope to patients [from minority groups] waiting for a transplant, he tells VICE. But in terms of turning that into more supply of donors, I'm hoping for a positive change [in the number of donors].

But hes fearful there will be an initial backlash of people opting out, due to an expected influx of damaging social media messages that will spread fear and misinformation. Over the last several years the number of BAME patients in need of blood, stem cell and organ transplants has risen, while the number of eligible donors from minority groups has remained alarmingly low. This disparity between patients and donors has no doubt led to BAME communities making up 35 percent of those waiting for a kidney transplant.

Among those waiting is Faizan Azwan, 33, who has suffered from renal complications since birth. As a newborn, he was fed by tube and tasted solid food for the first time at the age of three.

Faizan was saved by a transplant twice in the last three decades and is now facing the same fate for a third time. Five years ago, he began to feel unwell and was rushed to a hospital by his parents only to find out that the kidney his father donated had failed.

His five-year wait for a transplant reflects the small pool of donors Azwan has to choose from as a British-Pakistani patient. The average waiting time for a kidney transplant is two years for European patients, in comparison to two and a half for those from minority ethnic groups.

He said: [Renal failure] has affected me, as with most BAME patients waiting for a transplant. On average we have to wait approximately six months to a year longer than our European counterparts, and that is simply because our communities do not tend to donate.

Lewis sees the new opt-out scheme as an opportunity to remind those who come from communities that do not donate, that they are needed. We need those individuals to realise their ethnicity counts, and get the message across that we need everyone, but actually we need you the most.

He adds: If we don't step forward as donors, we are self discriminating against our own.

@Zahra_ZW

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Not White? You Might Struggle to Find an Organ Donor - VICE UK

Ewing sarcoma is a form of bone cancer that usually affects children and adolescents.

Ewing sarcoma can be very aggressive, but the cells tend to respond well to radiation therapy. Ideally, doctors will diagnose the cancer before it has spread.

According to the National Library of Medicine, an estimated 250 children in the United States receive a diagnosis of Ewing sarcoma each year.

In this article, learn more about Ewing sarcoma, including the symptoms, causes, and treatment options.

Ewing sarcoma is a rare type of cancer that usually starts in the bone typically in the pelvis, chest wall, or legs and occurs mostly in children and teenagers.

Dr. James Ewing first described Ewing sarcoma in 1921. He identified cancer cells that looked different than the cells in osteosarcoma, another type of bone tumor.

Doctors may also refer to this cancer type as the Ewing family of tumors. These tumors have distinct cells that usually respond well to radiation treatments.

This rare cancer type accounts for just 1.5% of all childhood cancers and is the second most common bone cancer type in childhood, after osteosarcoma.

Although researchers are unsure why some people develop Ewing sarcoma, they have identified mutations in certain genes in the tumor cells that cause this cancer.

These include the EWSR1 gene on chromosome 22 and the FLI1 gene on chromosome 11.

These genetic mutations occur spontaneously during a person's lifetime. The individual does not inherit them from a family member.

There are no known risk factors for Ewing sarcoma that make one person more likely than another to develop this cancer.

Ewing sarcoma can cause the following symptoms:

An estimated 87% of Ewing sarcomas are sarcoma of the bone. The other types form in the soft tissues, such as cartilage, that surround the bones.

Ewing sarcoma can spread to other areas of the body. Doctors call this process metastasis.

Areas that the cancer can spread to include other bones, bone marrow, and the lungs.

Doctors categorize Ewing sarcoma as one of three types according to its extent:

Before diagnosing Ewing sarcoma, a doctor will take a person's full medical history and ask them what symptoms they are having, when they noticed them, and what makes them better or worse. They will also perform a thorough physical exam, focusing on the area of concern.

A doctor will usually recommend an imaging study to view the bone or bones. These tests include:

If it looks as though a tumor may be present, a doctor will perform a biopsy, which involves taking a sample of bone tissue. They will send this tissue to a laboratory, where a specialist called a pathologist will check it for the presence of cancerous cells.

A doctor may also order blood tests, a bone marrow biopsy, and other scans when necessary. These tests can help determine whether the cancer has spread to other locations.

A doctor will work with a team of cancer specialists and surgeons to recommend and implement particular treatments.

Possible treatments for Ewing sarcoma include:

Doctors may use a combination of treatments depending on how far the cancer has spread and a person's overall health.

Research into new treatments for Ewing sarcoma is ongoing. Some doctors may inform their patients about clinical trials, which help test new treatments.

Possible complications of Ewing sarcoma include:

If Ewing sarcoma has spread to other areas of the body, it can be life threatening. For this reason, it is vital for a doctor to evaluate any symptoms as quickly as possible.

According to the American Academy of Orthopaedic Surgeons, an estimated two-thirds of people in whom cancer has not spread to other areas of the body survive at least 5 years after their diagnosis.

People who are more likely to have positive outcomes include those who have:

The likelihood of successful treatment is different for every individual, so people should speak to a doctor about their or their child's expected outlook.

Ewing sarcoma is a rare type of cancer that mostly affects young people.

When doctors detect it early enough, the condition usually responds well to treatment.

Anyone who notices signs or symptoms of Ewing sarcoma, such as a bone that breaks for no apparent reason or a painful lump or swelling, should speak to a doctor.

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Ewing sarcoma: Causes, symptoms, and treatment - Medical News Today

DUBLIN--(BUSINESS WIRE)--The "Wound Care Market - Global Outlook and Forecast 2019-2024" report has been added to ResearchAndMarkets.com's offering.

The global wound care market is growing at a CAGR of over 5% during the forecast period 2018-2024

The adoption of wound care biologics is augmenting the growth of the global wound care market. The commercial availability of a wide array of wound biologics is likely to encourage many end-users to use them in treating wounds as they are clinically proven, safe, and effective than other products.

The growing incidence of infections caused in lesions is another factor accelerating the growth of anti-microbial dressings market segment. Anti-microbial agents such as chlorhexidine, maggots, silver, iodine, and honey are increasingly becoming important in the global wound care market. Therefore, the incorporation of anti-microbial agents in wound dressing products is improving clinical outcomes for the treatment of wounds, thereby driving the global wound care market.

Key Vendor Analysis

The global wound care market is highly competitive and dynamic characterized by the presence of several global, regional, and local vendors, offering a diverse range of products and devices for treating various acute and chronic wounds. There are approximately more than 400 vendors providing a wide array of wound care products worldwide. Multiple product launches, strategic acquisitions, and differentiated products have fueled the growth in recent years.

New product launches and strategic acquisitions, collaborations will be crucial for companies to maintain revenue growth in the coming years. Large and diversified companies account for nearly 48% of the market share. Also, the majority of key players have demonstrated consistent growth in the last three years. Moderate to the high growth of major players will continue to boost the market growth. In addition, both established and emerging players are developing or commercializing wound care products and devices.

Major players are focusing on technological innovations, thereby investing substantial amounts on R&D activities for offering advanced wound care products such as advanced wound dressings, skin grafts, and wound biologics.

Key Topics Covered:

1 Research Methodology

2 Research Objectives

3 Research Process

4 Scope & Coverage

4.1 Market Definition

4.2 Base Year

4.3 Scope of the study

4.4 Market Segments

5 Report Assumptions & Caveats

5.1 Key Caveats

5.2 Currency Conversion

5.3 Market Derivation

6 Market at a Glance

7 Introduction

7.1 Wound Care: An Overview

7.1.1 Background

7.1.2 Wound Care for Acute & Chronic Wounds

7.1.3 Wound Care: Market Snapshot

8 Market Dynamics

8.1 Market Growth Enablers

8.1.1 Increasing Prevalence of Acute & Chronic Wounds

8.1.2 New Product Approvals/Launches

8.1.3 Increasing Number of Surgical Procedures

8.2 Market Growth Restraints

8.2.1 High Cost of Advanced Wound Care Products & Devices

8.2.2 Limitations & Complications with Wound Care Products & Devices

8.2.3 Intense Competition & Pricing Pressure

8.2.4 Shortage of Resources for Wound Care Treatments

8.3 Market Opportunities & Trends

8.3.1 Focus on Development & Commercialization of Wound Biologics

8.3.2 High Demand for Anti-microbial Wound Dressing Products

8.3.3 Growing Popularity of Natural Surgical Sealants

8.3.4 Emergence of Stem Cell Therapy For Wound Healing

9 Global Wound Care Market

9.1 Market Overview

9.2 Market Size & Forecast

9.3 Five Forces Analysis

10 By Product Type

10.1 Market Snapshot & Growth Engine

10.2 Market Overview

11 Advanced Wound Care Products

11.1 Market Snapshot & Growth Engine

11.2 Market Overview

11.3 Advanced Wound Care Segmentation by Product Type

11.4 Advanced Wound Dressings

11.5 Wound Therapy Devices

11.6 Wound Care Biologics

12 Traditional Wound Care Products

12.1 Market Overview

12.2 Market Size & Forecast

13 Sutures & Stapling Devices

13.1 Market Snapshot & Growth Engine

13.2 Market Overview

13.3 Market Size & Forecast

13.4 Segmentation by Product Type

13.5 Sutures

13.6 Stapling Devices

14 Hemostats & Surgical Sealants

14.1 Market Snapshot & Growth Engine

14.2 Market Overview

14.3 Market Size & Forecast

14.4 Segmentation by Product Type

14.5 Hemostats

14.6 Surgical Sealants

15 By Wound Type

15.1 Market Snapshot & Growth Engine

15.2 Market Overview

15.3 Acute Wounds

15.4 Chronic Wounds

16 By End Users

16.1 Market Snapshot & Growth Engine

16.2 Market Overview

16.3 Hospitals & Specialty Wound Care Clinics

16.4 Long-term Care Facilities

16.5 Home Healthcare

16.6 Others

17 By Geography

17.1 Market Snapshot & Growth Engine

17.2 Market Overview

Key Company Profiles

Other Prominent Vendors

For more information about this report visit https://www.researchandmarkets.com/r/gxho7k

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Global Wound Care Market Outlook and Forecasts, 2018 & 2019-2024 - ResearchAndMarkets.com - Business Wire

The ELECTRONIC HEALTH RECORDS (EHR) market research report added by Report Ocean, is an in-depth analysis of the latest trends, market size, status, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players. The research study provides market introduction, ELECTRONIC HEALTH RECORDS (EHR) market definition, regional market scope, sales and revenue by region, manufacturing cost analysis, Industrial Chain, market effect factors analysis, ELECTRONIC HEALTH RECORDS (EHR) market size forecast, 100+ market data, Tables, Pie Chart, Graphs and Figures, and many more for business intelligence.

The global EHR market was valued at $23,592 million in 2016, and is expected to reach $33,294 million by 2023, growing at a CAGR of 5.0% from 2017 to 2023.

UPTO 30% OFF ON SINGLE USER PDF: https://www.reportocean.com/industry-verticals/sample-request?report_id=27174

ELECTRONIC HEALTH RECORDS (EHR) Market: Competitive Analysis by Key Players:

AdvancedMD, Inc.Allscripts Healthcare Solutions, Inc.Cerner CorporationComputer Programs and Systems, Inc.CureMD CorporationeClinicalWorksEpic Systems CorporationGeneral Electric CompanyGreenway Health, LLCQuality Systems, Inc.

The Global ELECTRONIC HEALTH RECORDS (EHR) Market also explains the competitive landscape among the major key players of the market, based on various parameters, such as:

ELECTRONIC HEALTH RECORDS (EHR) Market Segments:

By Product (Cloud-Based Software and Server-Based/On-Premise Software), Type (Inpatient EHR and Ambulatory EHR), Application (Clinical Application, Administrative Application, Reporting in Healthcare System, Healthcare Financing, and Clinical Research Application), and End User (Hospitals, Clinics, Specialty Centers, and Other End Users)

ELECTRONIC HEALTH RECORDS (EHR) Market: Insights

The global EHR market was valued at $23,592 million in 2016, and is expected to reach $33,294 million by 2023, growing at a CAGR of 5.0% from 2017 to 2023.

An electronic health record (EHR) is a digital version of the paper chart of a patient. It contains all the data related to a patients medical history including medications, diagnosis, immunization dates, treatment plans, radiology images, allergies, and test results from laboratories. These also allow access to evidence-based tools used by healthcare providers to make decisions about a patients treatment. EHRs have also helped to streamline and automate the workflow in a healthcare setting.

Rise in adoption of EHR, increased use of cloud based EHR software, and rapid surge in aging population and subsequent rise in the number of chronic diseases drive the market growth. However, high cost of EHR and increase in concerns regarding the patient data safety & security are expected to impede the market growth. Moreover, huge market potential in the developing regions are expected to offer further opportunities for market growth during the forecast period.

The report segments the market based on product, type, application, end user, and region. Based on product, the market is bifurcated into cloud-based software and server-based/on-premise software. The type segment is categorized into inpatient EHR and ambulatory EHR. Based on application, the market is segmented into clinical application, administrative application, reporting in healthcare system, healthcare financing, and clinical research application. EHRs may be used in hospitals, clinics, specialty centers, and other medical settings. Based on region, the market is analyzed across North America (U.S., Canada, and Mexico), Europe (Germany, France, UK, Spain, and rest of Europe), Asia-Pacific (China, Japan, Australia, India, and rest of Asia-Pacific), and LAMEA (Brazil, South Africa, Saudi Arabia, and rest of LAMEA).

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Furthermore, the years considered for the study are as follows:

Historical year 2013-2017

Base year 2018

Forecast period** 2019 to 2025 [** unless otherwise stated]

Regional Analysis:

The market research study offers in-depth regional analysis along with the current market scenarios. The major regions analyzed in the study are:

Key highlights and important features of the Report:

Overview and highlights of product and application segments of the global ELECTRONIC HEALTH RECORDS (EHR) Market are provided. Highlights of the segmentation study include price, revenue, sales, sales growth rate, and market share by product.

Explore about Sales data of key players of the global ELECTRONIC HEALTH RECORDS (EHR) Market as well as some useful information on their business. It talks about the gross margin, price, revenue, products, and their specifications, type, applications, competitors, manufacturing base, and the main business of key players operating in the ELECTRONIC HEALTH RECORDS (EHR) Market.

Explore about gross margin, sales, revenue, production, market share, CAGR, and market size by region.

Describe ELECTRONIC HEALTH RECORDS (EHR) Market Findings and Conclusion, Appendix, methodology and data source;

Research Methodology:

The market research was done by adopting various tools under the category of primary and secondary research. For primary research, experts and major sources of information have been interviewed from suppliers side and industries, to obtain and verify the data related to the study of the Global ELECTRONIC HEALTH RECORDS (EHR) Market. In secondary research methodology, various secondary sources were referred to collect and identify extensive piece of information, such as paid databases, directories and annual reports and databases for commercial study of the Global ELECTRONIC HEALTH RECORDS (EHR) Market. Moreover, other secondary sources include studying technical papers, news releases, government websites, product literatures, white papers, and other literatures to research the market in detail.

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There are 15 Chapters to display the Global ELECTRONIC HEALTH RECORDS (EHR) Market:

Chapter 1, to describe Definition, Specifications and Classification of Global ELECTRONIC HEALTH RECORDS (EHR), Applications of, Market Segment by Regions;Chapter 2, to analyze the Manufacturing Cost Structure, Raw Material and Suppliers, Manufacturing Process, Industry Chain Structure;Chapter 3, to display the Technical Data and Manufacturing Plants Analysis of , Capacity and Commercial Production Date, Manufacturing Plants Distribution, Export & Import, R&D Status and Technology Source, Raw Materials Sources Analysis;Chapter 4, to show the Overall Market Analysis, Capacity Analysis (Company Segment), Sales Analysis (Company Segment), Sales Price Analysis (Company Segment);Chapter 5 and 6, to show the Regional Market Analysis that includes United States, EU, Japan, China, India & Southeast Asia, Segment Market Analysis (by Type);Chapter 7 and 8, to explore the Market Analysis by Application Major Manufacturers Analysis;Chapter 9, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type, Market Trend by Application;Chapter 10, Regional Marketing Type Analysis, International Trade Type Analysis, Supply Chain Analysis;Chapter 11, to analyze the Consumers Analysis of Global ELECTRONIC HEALTH RECORDS (EHR) by region, type and application;Chapter 12, to describe ELECTRONIC HEALTH RECORDS (EHR) Research Findings and Conclusion, Appendix, methodology and data source;Chapter 13, 14 and 15, to describe ELECTRONIC HEALTH RECORDS (EHR) sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

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Molecular Diagnostics Market is Anticipated to Expand at a CAGR of 11.6% from 2016 to 2025 - Health News Office

DUBLIN, Oct. 22, 2019 /PRNewswire/ -- The "Wound Care Market - Global Outlook and Forecast 2019-2024" report has been added to ResearchAndMarkets.com's offering.

The global wound care market is growing at a CAGR of over 5% during the forecast period 2018-2024.

The adoption of wound care biologics is augmenting the growth of the global wound care market. The commercial availability of a wide array of wound biologics is likely to encourage many end-users to use them in treating wounds as they are clinically proven, safe, and effective than other products.

The growing incidence of infections caused in lesions is another factor accelerating the growth of anti-microbial dressings market segment. Anti-microbial agents such as chlorhexidine, maggots, silver, iodine, and honey are increasingly becoming important in the global wound care market. Therefore, the incorporation of anti-microbial agents in wound dressing products is improving clinical outcomes for the treatment of wounds, thereby driving the global wound care market.

This research report includes detailed market segmentation by products, wound type, end-users, and geography. The increase in the geriatric population is a major contributing factor for the growth of the advanced wound care segment as the prevalence of diabetes and other diseases is more common in the elderly age group than youth.

The advanced segment is also growing as the majority of market players are offering innovative products to meet the demand for wound care worldwide. The rising incidence of diabetes and associated diabetic foot ulcers in the elderly population globally is fueling steady growth for traditional products.

The market is also growing steadily as products such as gauze bandages and adhesive bandages witness sustainable demand for small cuts, bruises as well as for chronic wounds and burns, especially in developing countries. Developing regions such as Africa, Asia, and Latin America are the largest contributors to the traditional products.

The acute wound market is growing mainly due to the rise in surgical site infections (SSI) and the increase in the number of burn cases worldwide. Chronic wounds do not heal through the normal healing process. The segment is growing due to the growing burden of diabetic foot ulcers, venous leg ulcers, pressure ulcers, and some surgical site infections that do not heal naturally or with medicines.

The shift from traditional lower technology wound care treatments to the adoption of advanced treatments is a major factor for the high share of the hospitals and specialty wound clinic segment. Long-term care facilities segment is growing at a steady pace because of the growing incidence of chronic wounds due to the increase in chronic diseases such as diabetes. The growing elderly population is contributing to the growth of the segment as they are more prone to chronic diseases.

Key Topics Covered:

1 Research Methodology

2 Research Objectives

3 Research Process

4 Scope & Coverage4.1 Market Definition4.2 Base Year4.3 Scope of the study4.4 Market Segments

5 Report Assumptions & Caveats5.1 Key Caveats5.2 Currency Conversion5.3 Market Derivation

6 Market at a Glance

7 Introduction7.1 Wound Care: An Overview7.1.1 Background7.1.2 Wound Care for Acute & Chronic Wounds7.1.3 Wound Care: Market Snapshot

8 Market Dynamics8.1 Market Growth Enablers8.1.1 Increasing Prevalence of Acute & Chronic Wounds8.1.2 New Product Approvals/Launches8.1.3 Increasing Number of Surgical Procedures8.2 Market Growth Restraints8.2.1 High Cost of Advanced Wound Care Products & Devices8.2.2 Limitations & Complications with Wound Care Products & Devices8.2.3 Intense Competition & Pricing Pressure8.2.4 Shortage of Resources for Wound Care Treatments8.3 Market Opportunities & Trends8.3.1 Focus on Development & Commercialization of Wound Biologics8.3.2 High Demand for Anti-microbial Wound Dressing Products8.3.3 Growing Popularity of Natural Surgical Sealants8.3.4 Emergence of Stem Cell Therapy For Wound Healing

9 Global Wound Care Market9.1 Market Overview9.2 Market Size & Forecast9.3 Five Forces Analysis

10 By Product Type10.1 Market Snapshot & Growth Engine10.2 Market Overview

11 Advanced Wound Care Products11.1 Market Snapshot & Growth Engine11.2 Market Overview11.3 Advanced Wound Care Segmentation by Product Type11.4 Advanced Wound Dressings11.5 Wound Therapy Devices11.6 Wound Care Biologics

12 Traditional Wound Care Products12.1 Market Overview12.2 Market Size & Forecast

13 Sutures & Stapling Devices13.1 Market Snapshot & Growth Engine13.2 Market Overview13.3 Market Size & Forecast13.4 Segmentation by Product Type13.5 Sutures13.6 Stapling Devices

14 Hemostats & Surgical Sealants14.1 Market Snapshot & Growth Engine14.2 Market Overview14.3 Market Size & Forecast14.4 Segmentation by Product Type14.5 Hemostats14.6 Surgical Sealants

15 By Wound Type15.1 Market Snapshot & Growth Engine15.2 Market Overview15.3 Acute Wounds15.4 Chronic Wounds

16 By End Users16.1 Market Snapshot & Growth Engine16.2 Market Overview16.3 Hospitals & Specialty Wound Care Clinics16.4 Long-term Care Facilities16.5 Home Healthcare16.6 Others

17 By Geography17.1 Market Snapshot & Growth Engine17.2 Market Overview

Key Company Profiles

Other Prominent Vendors

For more information about this report visit https://www.researchandmarkets.com/r/iiu23r

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global Wound Care Market Outlook to 2024: New Product Approvals/Launches, Emergence of Stem Cell Therapy For Wound Healing - P&T Community

Cell-based therapy for type 1 diabetes is a new treatment strategy that is showing promising results. Type 1 diabetes is a chronic disease that can develop early in life. The disease involves the destruction of pancreatic beta cells by the bodys auto-immune response resulting in insufficient insulin production to regulate blood glucose. If left untreated, this condition can lead to serious long-term effects such as neuropathy, retinopathy, and renal failure. Insulin therapy is the current standard of care treatment of type 1 diabetes. However, insulin therapy cannot fully prevent the long-term complications associated with type 1 diabetes.

Organoids are tiny, three-dimensional tissue cultures that are derived from stem cells. Organoids can be created to replicate the complexity of an organ or they can be crafted to express selected aspects of an organ such as producing only certain types of cells.

Researchers have created organoids that differentiate into insulin-producing pancreatic cells. These modified insulin-producing cells successfully regulated blood glucose levels when implanted in diabetic mice.

The cluster of cells in the pancreas that produce insulin is known as islets. Islet cell transplantation is a powerful tool to treat type 1 diabetes. Many studies have shown how this cell therapy could be effective in the treatment of diabetes if long-term control of glucose levels can be achieved.

Researchers have faced challenges in this treatment strategy due to loss of islet cells after the transplantation. The loss of cells occurs mainly because of inflammation of the transplant site and revascularization of cells that disrupts blood and oxygen supply leading to cell death. Scientists are looking for new strategies that can prevent the loss of islet cells and improve clinical islet transplantation outcomes.

Amniotic epithelial cells are stem cells that have a high proliferative capacity, self-renewal ability, multilineage differentiation, ease of access, and are safe for transplantation. During the last few years, human amniotic epithelial cells have been of great interest to researchers working on regenerative medicine.

In a new study recently published in Nature Communications, researchers from Geneva, Switzerland, engineered viable and functional insulin-producing organoids by combining human amniotic epithelial cells and dissociated islet cells. The researchers tested if inclusion of human amniotic epithelial cells enhanced the engraftment and viability of islet cells and determined if this cell-therapy for type 1 diabetes was successful in mouse models. Various tests such as insulin expression, insulin secretion, and stability under hypoxic conditions were used to test the viability of the organoids.

The organoids composed of human amniotic epithelial cells and islet cells did not experience any islet loss post transplantation. The researchers observed a clear protective effect of amniotic epithelial cells on islet cells in conditions of hypoxia. In addition, the organoids maintained responsiveness to glucose and showed significant protection from cell death.

The researchers found that islet organoids enriched with human amniotic epithelial cells resulted in mass engraftment of insulin producing beta cells thus improving function of these cells. Compared with islet cell organoids not combined with amniotic epithelial cells, the organoids with islet cells and amniotic epithelial cell combination normalized the blood glucose levels in diabetic mice. This suggests that there was adequate blood glucose regulation achieved by these engineered organoids.

These findings show that combining islet cells with human amniotic epithelial cells markedly improves their functionality and viability of islet cells. It also helps in the successful engraftment of islet cells. This cell therapy for type 1 diabetes has the potential to be the next treatment strategy for this condition.

The researchers express the need to further explore the use of these organoids to include more favorable implantation sites and expanding to the use of stem cells that are an unlimited source of insulin.

Written by Preeti Paul, M.Sc.

Reference: Fanny Lebreton et al., Insulin producing organoids engineered from islet and amniotic epithelial cells to treat diabetes. Nature Communications 10, Article number: 4491 (2019)

Image bySteve BuissinnefromPixabay

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Engineered cell-based therapy as a new treatment strategy for type 1 diabetes - Medical News Bulletin

KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending approval of two regimens of KEYTRUDA, Mercks anti-PD-1 therapy, for the first-line treatment of metastatic or unresectable recurrent head and neck squamous cell carcinoma (HNSCC). KEYTRUDA, as monotherapy or in combination with platinum and 5-fluorouracil (5-FU) chemotherapy, is recommended in patients whose tumors express PD-L1 (combined positive score [CPS] 1). This recommendation is based on data from the pivotal Phase 3 KEYNOTE-048 trial, in which KEYTRUDA, as monotherapy and in combination with chemotherapy, demonstrated a significant improvement in overall survival, compared with standard treatment (cetuximab with carboplatin or cisplatin plus 5-FU), in these patient populations.

Head and neck cancer remains a devastating disease with poor long-term outcomes and advances in survival have been difficult to achieve for more than 10 years said Dr. Jonathan Cheng, vice president, clinical research, Merck Research Laboratories. The positive EU CHMP opinion further validates the potential of KEYTRUDA, as monotherapy and in combination with chemotherapy, to help patients and address the high unmet need in this aggressive form of head and neck cancer.

Merck currently has the largest immuno-oncology clinical development program in HNSCC and is continuing to advance multiple registration-enabling studies investigating KEYTRUDA as monotherapy and in combination with other cancer treatmentsincluding, KEYNOTE-412 and KEYNOTE-689. The CHMPs recommendation will now be reviewed by the European Commission for marketing authorization in the EU, and a final decision is expected in the fourth quarter of 2019.

About Head and Neck CancerHead and neck cancer describes a number of different tumors that develop in or around the throat, larynx, nose, sinuses and mouth. Most head and neck cancers are squamous cell carcinomas that begin in the flat, squamous cells that make up the thin surface layer of the structures in the head and neck. Two substances that greatly increase the risk of developing head and neck cancer are tobacco and alcohol. It is estimated that there were more than 705,000 new cases of head and neck cancer diagnosed and over 358,000 deaths from the disease worldwide in 2018. In Europe, it is estimated that there were more than 146,000 newly diagnosed cases of head and neck cancer and around 66,000 deaths from the disease in 2018.

About KEYTRUDA (pembrolizumab) InjectionKEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,000 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patients likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

About KEYTRUDA (pembrolizumab) InjectionKEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,000 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patients likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) IndicationsMelanomaKEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung CancerKEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung CancerKEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck CancerKEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin LymphomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell LymphomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for the treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial CarcinomaKEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [CPS 10] as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Microsatellite Instability-High (MSI-H) CancerKEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric CancerKEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal CancerKEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical CancerKEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular CarcinomaKEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell CarcinomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell CarcinomaKEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Selected Important Safety Information for KEYTRUDA

Immune-Mediated PneumonitisKEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grade 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated ColitisKEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination with Axitinib)Immune-Mediated HepatitisKEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination with AxitinibKEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated EndocrinopathiesKEYTRUDA can cause hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%), receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of hypophysitis (including hypopituitarism and adrenal insufficiency), thyroid function (prior to and periodically during treatment), and hyperglycemia. For hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 and withhold or discontinue for Grade 3 or 4 hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal DysfunctionKEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin ReactionsImmune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse ReactionsImmune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including cHL, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related ReactionsKEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple MyelomaIn trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal ToxicityBased on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse ReactionsIn KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC; the most common was pneumonitis (1.8%). The most common adverse reactions (20%) were decreased appetite (25%), fatigue (25%), dyspnea (23%), and nausea (20%).

Adverse reactions occurring in patients with SCLC were similar to those occurring in patients with other solid tumors who received KEYTRUDA as a single agent.

In KEYNOTE-048, KEYTRUDA monotherapy was discontinued due to adverse events in 12% of 300 patients with HNSCC; the most common adverse reactions leading to permanent discontinuation were sepsis (1.7%) and pneumonia (1.3%). The most common adverse reactions (20%) were fatigue (33%), constipation (20%), and rash (20%).

In KEYNOTE-048, when KEYTRUDA was administered in combination with platinum (cisplatin or carboplatin) and FU chemotherapy, KEYTRUDA was discontinued due to adverse reactions in 16% of 276 patients with HNSCC. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonia (2.5%), pneumonitis (1.8%), and septic shock (1.4%). The most common adverse reactions (20%) were nausea (51%), fatigue (49%), constipation (37%), vomiting (32%), mucosal inflammation (31%), diarrhea (29%), decreased appetite (29%), stomatitis (26%), and cough (22%).

In KEYNOTE-012, KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (20%) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of facial edema and new or worsening hypothyroidism.

In KEYNOTE-087, KEYTRUDA was discontinued due to adverse reactions in 5% of 210 patients with cHL. Serious adverse reactions occurred in 16% of patients; those 1% included pneumonia, pneumonitis, pyrexia, dyspnea, GVHD, and herpes zoster. Two patients died from causes other than disease progression; 1 from GVHD after subsequent allogeneic HSCT and 1 from septic shock. The most common adverse reactions (20%) were fatigue (26%), pyrexia (24%), cough (24%), musculoskeletal pain (21%), diarrhea (20%), and rash (20%).

In KEYNOTE-170, KEYTRUDA was discontinued due to adverse reactions in 8% of 53 patients with PMBCL. Serious adverse reactions occurred in 26% of patients and included arrhythmia (4%), cardiac tamponade (2%), myocardial infarction (2%), pericardial effusion (2%), and pericarditis (2%). Six (11%) patients died within 30 days of start of treatment. The most common adverse reactions (20%) were musculoskeletal pain (30%), upper respiratory tract infection and pyrexia (28% each), cough (26%), fatigue (23%), and dyspnea (21%).

In KEYNOTE-052, KEYTRUDA was discontinued due to adverse reactions in 11% of 370 patients with locally advanced or metastatic urothelial carcinoma. Serious adverse reactions occurred in 42% of patients; those 2% were urinary tract infection, hematuria, acute kidney injury, pneumonia, and urosepsis. The most common adverse reactions (20%) were fatigue (38%), musculoskeletal pain (24%), decreased appetite (22%), constipation (21%), rash (21%), and diarrhea (20%).

In KEYNOTE-045, KEYTRUDA was discontinued due to adverse reactions in 8% of 266 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reaction resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.9%). Serious adverse reactions occurred in 39% of KEYTRUDA-treated patients; those 2% were urinary tract infection, pneumonia, anemia, and pneumonitis. The most common adverse reactions (20%) in patients who received KEYTRUDA were fatigue (38%), musculoskeletal pain (32%), pruritus (23%), decreased appetite (21%), nausea (21%), and rash (20%).

Adverse reactions occurring in patients with gastric cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

Adverse reactions occurring in patients with esophageal cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

In KEYNOTE-158, KEYTRUDA was discontinued due to adverse reactions in 8% of 98 patients with recurrent or metastatic cervical cancer. Serious adverse reactions occurred in 39% of patients receiving KEYTRUDA; the most frequent included anemia (7%), fistula, hemorrhage, and infections [except urinary tract infections] (4.1% each). The most common adverse reactions (20%) were fatigue (43%), musculoskeletal pain (27%), diarrhea (23%), pain and abdominal pain (22% each), and decreased appetite (21%).

Adverse reactions occurring in patients with HCC were generally similar to those in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of ascites (8% Grades 3-4) and immune-mediated hepatitis (2.9%). Laboratory abnormalities (Grades 3-4) that occurred at a higher incidence were elevated AST (20%), ALT (9%), and hyperbilirubinemia (10%).

Among the 50 patients with MCC enrolled in study KEYNOTE-017, adverse reactions occurring in patients with MCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy. Laboratory abnormalities (Grades 3-4) that occurred at a higher incidence were elevated AST (11%) and hyperglycemia (19%).

In KEYNOTE-426, when KEYTRUDA was administered in combination with axitinib, fatal adverse reactions occurred in 3.3% of 429 patients. Serious adverse reactions occurred in 40% of patients, the most frequent of which (1%) included hepatotoxicity (7%), diarrhea (4.2%), acute kidney injury (2.3%), dehydration (1%), and pneumonitis (1%). Permanent discontinuation due to an adverse reaction occurred in 31% of patients; KEYTRUDA only (13%), axitinib only (13%), and the combination (8%). The most common adverse reactions (>1%) resulting in permanent discontinuation of KEYTRUDA, axitinib or the combination were hepatotoxicity (13%), diarrhea/colitis (1.9%), acute kidney injury (1.6%), and cerebrovascular accident (1.2%). When KEYTRUDA was used in combination with axitinib, the most common adverse reactions (20%) were diarrhea (56%), fatigue/asthenia (52%), hypertension (48%), hepatotoxicity (39%), hypothyroidism (35%), decreased appetite (30%), palmar-plantar erythrodysesthesia (28%), nausea (28%), stomatitis/mucosal inflammation (27%), dysphonia (25%), rash (25%), cough (21%), and constipation (21%).

LactationBecause of the potential for serious adverse reactions in breastfed children, advise women not to breastfeed during treatment and for 4 months after the final dose.

Pediatric UseThere is limited experience in pediatric patients. In a trial, 40 pediatric patients (16 children aged 2 years to younger than 12 years and 24 adolescents aged 12 years to 18 years) with various cancers, including unapproved usages, were administered KEYTRUDA 2 mg/kg every 3 weeks. Patients received KEYTRUDA for a median of 3 doses (range 117 doses), with 34 patients (85%) receiving 2 doses or more. The safety profile in these pediatric patients was similar to that seen in adults; adverse reactions that occurred at a higher rate (15% difference) in these patients when compared to adults under 65 years of age were fatigue (45%), vomiting (38%), abdominal pain (28%), increased transaminases (28%), and hyponatremia (18%).

Mercks Focus on CancerOur goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck, the potential to bring new hope to people with cancer drives our purpose and supporting accessibility to our cancer medicines is our commitment. As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology with one of the largest development programs in the industry across more than 30 tumor types. We also continue to strengthen our portfolio through strategic acquisitions and are prioritizing the development of several promising oncology candidates with the potential to improve the treatment of advanced cancers. For more information about our oncology clinical trials, visit http://www.merck.com/clinicaltrials.

About MerckFor more than a century, Merck, a leading global biopharmaceutical company known as MSD outside of the United States and Canada, has been inventing for life, bringing forward medicines and vaccines for many of the worlds most challenging diseases. Through our prescription medicines, vaccines, biologic therapies and animal health products, we work with customers and operate in more than 140 countries to deliver innovative health solutions. We also demonstrate our commitment to increasing access to health care through far-reaching policies, programs and partnerships. Today, Merck continues to be at the forefront of research to advance the prevention and treatment of diseases that threaten people and communities around the world - including cancer, cardio-metabolic diseases, emerging animal diseases, Alzheimers disease and infectious diseases including HIV and Ebola. For more information, visit http://www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking Statement of Merck & Co., Inc., Kenilworth, N.J., USAThis news release of Merck & Co., Inc., Kenilworth, N.J., USA (the company) includes forward-looking statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the companys management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline products that the products will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the companys ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the companys patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the companys 2018 Annual Report on Form 10-K and the companys other filings with the Securities and Exchange Commission (SEC) available at the SECs Internet site (www.sec.gov).

Please see Prescribing Information for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf andMedication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.

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Merck Receives Positive EU CHMP Opinion for Two New Regimens of KEYTRUDA (pembrolizumab) as First-Line Treatment for Metastatic or Unresectable...

Grant will support researchers from Kings College London and Kings College Hospital to test a personalised treatment approach for Aplastic Anaemia patients who have not responded to available therapies

21 October 2019 - LifeArc, a UK-based medical research charity, and the Aplastic Anaemia Trust (AAT) have jointly awarded a 1.15m research grant to Kings College London and Kings College Hospital to investigate the potential of a novel type of personalised cellular therapy to reverse the ultra-rare condition aplastic anaemia (AA). The results of this research could give new hope to people living with a severe, life-limiting form of this condition.

The grant will fund a clinical trial to investigate the safety and efficacy of using a patients own T-reg cells to restore the blood-making function of the bone marrow. This follows laboratory-based research from the team of scientists where T-reg cells from a patients own blood were collected, selected for activity and multiplied. In a test tube, these cells prevented the immune system from attacking the patients bone marrow stem cells.[i]

Professor Ghulam Mufti, Department of Haematological Medicine at Kings College London and Kings College Hospital, and lead study investigator said: For patients with this ultra-rare disease, were looking for the first time at a personalised medicine approach where their own immune cells could be used to alter their disease. In AA there is a reduction in the number of T-regs and most of the ones that the AA patients do have are non-functional. Weve seen success in the laboratory by selecting and bolstering the number of functional T-reg cells. Now, with funding from LifeArc and the AAT, we can investigate the potential of this approach in treating AA patients who currently have very limited treatment options.

AA is an ultra-rare life-threatening illness caused by the bone marrow failing to make enough of all three types of blood cellsred blood cells, white blood cells and platelets. Only around 100150 people in UK are diagnosed per year, affecting all ages but most commonly people between the ages of 10 to 20 years old and those over the age of 60 years.

People with the illness are at greater risk of infections, bleeding, and can experience extreme fatigue, which leaves them unable to carry out simple daily tasks that most people take for granted. Around one in three patients with severe AA fail to respond to existing drug treatments and the other option a bone marrow transplant is reliant on finding a suitable donor, requires life-long treatment with immunosuppression therapy and is unsuccessful in one in three people.

Dr Catriona Crombie, LifeArcs Head of Philanthropic Fund explained why the charity had approved the funding: LifeArc set up the Philanthropic fund to support translational research into rare diseases, where there is less interest from commercial organisations. Patients with AA can have limited treatment options; this opportunity with Kings College London, Kings College Hospital and the AAT has the potential to transform the lives of patients living with a severe form of the disease.

The trial at Kings College London and Kings College Hospital will run for a duration of three years and aims to recruit nine patients. A blood sample of the patients T-reg cells will be extracted, purified and grown in the lab before being given back to the patient in a higher concentration. As patients with AA are more susceptible to infection, this personalised treatment approach is more likely to avoid the risk of severe infection and inflammation.

Grazina Berry, CEO of the AAT said: AA can severely impact a persons quality of life. Through AATs close work with Kings College London and Kings College Hospital as a specialist centre of clinical care and research in AA, we identified the project with the most potential to directly benefit patients who are currently at a loss for solutions. We are delighted to have partnered with LifeArc and Kings College London and Kings College Hospital to progress this ground-breaking work, which could potentially enable people living with severe AA to once again lead a normal life.

[i] Kordasti S, Costantini B, Seidl T, Perez-Abellan P, Llordella MM, McLornan D, Diggins KE, Kulasekararaj A, Benfatto C, Feng X, Smith A, Mian SA, Melchiotti R, de Rinadis E, Heck S, Ellis R, Petrov N, Povoleri GAM, Chung SS, Thomas NSB, FarzanehF, Irish JM, Young NS, Marsh JCW, Mufti GJ. Deep-phenotyping of Tregs identifies an immune signature for idiopathic aplastic anemia and predicts response to treatment. Blood. 2016 Sep 1;128(9):1193-20

About Aplastic Anaemia:

Aplastic Anaemia (AA) is a rare andlife-threatening illnesscaused by the bone marrow failing to make enough of all three types of blood cells - red blood cells, white blood cells and platelets.It is estimated that between 100 and 150 people will be diagnosed in UK alone every year. That is around 2 people for every 1,000,000 of population, making AA a very rare disease. In most cases of AA, the immune system attacks the bone marrow, thinking it is faulty. This makesthebone marrow function slow down, leading to the under-production ofall types ofblood cells.

The most common symptoms of AA are anaemia - caused by a lack of red blood cells - with the associated feeling of fatigue, shortness of breath, headaches and, occasionally, chest pains, and increased risk of infections caused by low white blood cells. Low platelets cause people to bleed easily. Being more susceptible to severe and life-threatening infection and bleeding complications makes AA a highly dangerous disease. Patients will visit hospital regularly to receive blood transfusions and treatments for infection; many will be admitted to hospital for weeks at a time while they receive treatment, and those who recover must take a lot of time regaining their strength, avoiding places where they could easily pick up infections.

There are currently two standard first-line treatments for AA:immune-suppressive therapy (IST), which uses medicines (antithymocyteglobulin (ATG) and ciclosporin) to dampen down abnormal immune responses, or bone marrowstem cell transplantation, the only known curative AA treatment to date, but suitable onlyfor a proportion of patients. A bone marrow transplant is an intense procedure that requires an immunological matched donor and may require lifelong immunosuppression therapy, which can lead to further debilitating side effects. Both bone marrow transplants and the combination of ATG and ciclosporin work in only around 2 in 3 of AA patients.

About the Aplastic Anaemia Trust

The Aplastic Anaemia Trust is the only charity in the UK dedicated to research into aplastic anaemia and alliedrare bone marrow failures and supportingeveryone affected nationally. We have builtproductive working partnershipswith major research and treatment centres of excellence in England, providing us with direct access to world-class experts, state-of-the-art labs and excellent patient care.Access to this medical and scientific expertise puts us in a strong position to identify areas of need, raisefunds for research, and engage with the expertcommunity in the UK and internationally. Our vision isa world free from aplastic anaemia and alliedrare bone marrow failures. Our mission is to enable vital research into the causes of aplastic anaemia and other rare bone marrow failures that ultimately leads to finding a cure, and to support everyone affected by them, so they can lead healthy and fulfilling lives.

Our strategic objectives are

Find out more about our work on http://www.theaat.org.uk

About LifeArc

LifeArc is a self-funded medical research charity. Our mission is to advance translation of early science into health care treatments or diagnostics that can be taken through to full development and made available to patients. We have been doing this for more than 25 years and our work has resulted in a diagnostic for antibiotic resistance and four licensed medicines.

Our success allows us to explore new approaches to stimulate and fund translation. We have our own drug discovery and diagnostics development facilities, supported by experts in technology transfer and intellectual property who also provide services to other organisations. Our model is built on collaboration, and we partner with a broad range of groups including medical research charities, research organisations, industry and academic scientists. We are motivated by patient need and scientific opportunity.

Two funds help us to invest in external projects for the benefit of patients: our Philanthropic Fund provides grants to support medical research projects focused on the translation of rare diseases research and our Seed Fund is aimed at start-up companies focussed on developing new therapeutics and biological modalities.

Find out more about our work on http://www.lifearc.org or follow us on LinkedIn or Twitter.

Kings College London

King's College Londonis one of the top 10 UK universities in the world (QS World University Rankings, 2018/19) and among the oldest in England. Kings has more than 31,000 students (including more than 12,800 postgraduates) from some 150 countries worldwide, and some 8,500 staff.

King's has an outstanding reputation for world-class teaching and cutting-edge research. In the 2014 Research Excellence Framework (REF), eighty-four per cent of research at Kings was deemed world-leading or internationally excellent (3* and 4*).

Since our foundation, Kings students and staff have dedicated themselves in the service of society. Kings will continue to focus on world-leading education, research and service, and will have an increasingly proactive role to play in a more interconnected, complex world.Visit our websiteto find out more about Vision 2029, Kings strategic vision for the next 12 years to 2029, which will be the 200th anniversary of the founding of the university. World-changing ideas. Life-changing impact:https://www.kcl.ac.uk/news/headlines.aspx

About Kings College HospitalKings College Hospital is a leading national and international centre for the diagnosis and treatment of blood cancers.

The hospital is home to the largest bone marrow transplant programme in the UK and performs more than 160 transplants a year. Kings College Hospital is also an international centre for research into and the treatment of myeloid leukaemias, lymphomas and myeloma, and have the first immune gene therapy programme for leukaemia approved by the Gene Therapy Advisory Committee (GTAC). It is the first hospital in the UK to treat adult lymphoma patients with CAR T therapy, and it has an active CAR programme in lymphoma, leukaemia and myeloma. It is also a centre of excellence for myelodysplasia and expertise in matched and unrelated transplants for the treatment of myelodysplastic syndrome (MDS).

The haemato-oncology service:

For more information about the haematology service at Kings College Hospital visit https://www.kch.nhs.uk/service/a-z/haemato-oncology

Read more from the original source:
Patients with ultra-rare bone marrow disease set to benefit from 1.15m grant from LifeArc and The Aplastic Anaemia Trust - PharmiWeb.com

This article, written byKatharine Sedivy-Haley, University of British Columbia, originally appeared on The Conversation and is republished here with permission:

When I was applying to graduate school in 2012, it felt like stem cells were about to revolutionize medicine.

Stem cells have the ability to renew themselves, and mature into specialized cells like heart or brain cells. This allows them to multiply and repair damage.

If stem cell genes are edited to fix defects causing diseases like anemia or immune deficiency, healthy cells can theoretically be reintroduced into a patient, thereby eliminating or preventing a disease. If these stem cells are taken or made from the patient themselves, they are a perfect genetic match for that individual, which means their body will not reject the tissue transplant.

Because of this potential, I was excited that my PhD project at the University of British Columbia gave me the opportunity to work with stem cells.

However, stem cell hype has led some to pay thousands of dollars on advertised stem cell treatments that promise to cure ailments from arthritis to Parkinsons disease. These treatments often dont help and may harm patients.

Despite the potential for stem cells to improve medicine, there are many challenges as they move from lab to clinic. In general, stem cell treatment requires we have a good understanding of stem cell types and how they mature. We also need stem cell culturing methods that will reliably produce large quantities of pure cells. And we need to figure out the correct cell dose and deliver it to the right part of the body.

Embryonic, 'induced and pluripotent

Stem cells come in multiple types. Embryonic stem cells come from embryos which makes them controversial to obtain.

A newly discovered stem cell type is the induced pluripotent stem cell. These cells are created by collecting adult cells, such as skin cells, and reprogramming them by inserting control genes which activate or induce a state similar to embryonic stem cells. This embryo-like state of having the versatile potential to turn into any adult cell type, is called being pluripotent.

However, induced pluripotent and embryonic stem cells can form tumours. Induced pluripotent stem cells carry a particularly high risk of harmful mutation and cancer because of their genetic instability and changes introduced during reprogramming.

Genetic damage could be avoided by using younger tissues such as umbilical cord blood, avoiding tissues that might contain pre-existing mutations (like sun-damaged skin cells), and using better methods for reprogramming.

Stem cells used to test drugs

For now, safety concerns mean pluripotent cells have barely made it to the clinic, but they have been used to test drugs.

For drug research, it is valuable yet often difficult to get research samples with specific disease-causing mutations; for example, brain cells from people with amyotrophic lateral sclerosis (ALS).

Researchers can, however, take a skin cell sample from a patient, create an induced pluripotent stem-cell line with their mutation and then make neurons out of those stem cells. This provides a renewable source of cells affected by the disease.

This approach could also be used for personalized medicine, testing how a particular patient will respond to different drugs for conditions like heart disease.

Vision loss from fat stem cells

Stem cells can also be found in adults. While embryonic stem cells can turn into any cell in the body, aside from rare newly discovered exceptions, adult stem cells mostly turn into a subset of mature adult cells.

For example, hematopoietic stem cells in blood and bone marrow can turn into any blood cell and are widely used in treating certain cancers and blood disorders.

A major challenge with adult stem cells is getting the right kind of stem cell in useful quantities. This is particularly difficult with eye and nerve cells. Most research is done with accessible stem cell types, like stem cells from fat.

Fat stem cells are also used in stem cell clinics without proper oversight or safety testing. Three patients experienced severe vision loss after having these cells injected into their eyes. There is little evidence that fat stem cells can turn into retinal cells.

Clinical complications

Currently, stem cell based treatments are still mostly experimental, and while some results are encouraging, several clinical trials have failed.

In the brain, despite progress in developing treatment for genetic disorders and spinal cord injury, treatments for stroke have been unsuccessful. Results might depend on method of stem cell delivery, timing of treatment and age and health of the patient. Frustratingly, older and sicker tissues may be more resistant to treatment.

For eye conditions, a treatment using adult stem cells to treat corneal injuries has recently been approved. A treatment for macular degeneration using cells derived from induced pluripotent stem cells is in progress, though it had to be redesigned due to concerns about cancer-causing mutations.

A path of cautious optimism

While scientists have good reason to be interested in stem cells, miracle cures are not right around the corner. There are many questions about how to implement treatments to provide benefit safely.

In some cases, advertised stem cell treatments may not actually use stem cells. Recent research suggests mesenchymal stem cells, which are commonly isolated from fat, are really a mixture of cells. These cells have regenerative properties, but may or may not include actual stem cells. Calling something a stem cell treatment is great marketing, but without regulation patients dont know what theyre getting.

Members of the public (and grad students) are advised to moderate their excitement in favour of cautious optimism.

Katharine Sedivy-Haley, PhD Candidate in Microbiology and Immunology, University of British Columbia

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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BEYOND LOCAL: Expert recommends 'path of cautious optimism' about the future of stem cell treatment - TimminsToday

I grew up in Ohio and met my first real Westerner at age 27. Kate was from Durango, Colorado and lived next door to me for two months during a rural primary care rotation in medical school. They rolled up the sidewalks at night in Twin Falls, Idaho and so we had plenty of time to talk. Books always figured prominently in the conversation and she recommended her favorite book about the West

Reading that first iconic chapter, Time Spent, led to a 25-year Doig odyssey that eventually landed me in a Montana State University archive reading the final draft of that same chapter, marked up with Ivans own red pen.

It begins, That start of memorys gather: June 27, 1945. I have become 6 years old, my mothers life has drained out at 31 years. And in the first grey daylight, dully heading our horses around from that cabin of the past, my father and I rein away toward all that would come next.

The Doig archive at Montana State is a treasure trove for fans. Its extensively indexed and entirely on line and filled with pictures, original manuscripts and the collection of 3 x 5 and 5 x 8 cards on which Doig kept quotes and observations from his extensive research travels in Montana.

Ivan kept box after box of these cards, many of them with only a single type written sentence, sometimes annotated in longhand. He shuffled and reshuffled these bits of memory assembling them into temporary collections as material to flesh out a particular character or story line and then returned them to their boxes only to be reshuffled and reassembled for the next novel.

In summer 2019, Carol Doig met with a group of visitors and discussed her husband's journey in his last years. Joining them was her close friend, Betty Mayfield, who helped assemble Doig's edited manuscripts, diaries, correspondence and other documents that today are part of the MSU Doig collection. Those joining Carol in her living are, left to right, Betty Mayfield, Dr. Rob Patrick, Justin Shanks a post-doctoral fellow working on the Doig material to make it digitally accessible, and writer Todd Wilkinson of Mountain Journal. Photo by Kenning Arlitsch, dean of MSU Libraries

Sometimes this shuffling was frozen into a more permanent form when he collected them into 2-to-3 page novellas with titles like Scotchisms and Curses.Most assumptions arent conscious until they are shattered. Without realizing it, I had cast my favorite writer in his own movie that ran only in my head.

He woke up in the morning, hunted big game, slept with the world's most beautiful women, cavorted at the Algonquin round table, drank his weight in scotch and then, late at night, great work flowed forth from his pen in a tortured and inspired torrent. He threw himself down exhausted, only to arise and repeat the performance with the dawn. The truth that emerged from touching the physical remnants of his process was far different.

Ivan Doig was . . . a nerd . . .just like me. I clipped articles and collected them in folders, wrote down random thoughts and observations on cards, restacked, hoarded and recombined information. My stories were just about thrombocytopenia and clonal proliferation instead of resilient ranchers scraping out an existence under the Big Sky.

The champion of the lariat proletariat was a closet geek. How disappointing.But my biggest disappointment was yet to come.The archive contained an odd and alluring folder title medical journey that was irresistible to me as a physician. I hadnt realized that Ivan suffered from multiple myeloma for the last eight years of his life and had written four books after being diagnosed with a terminal disease.

Myeloma is a strange form of cancer as cancers go, it is both painstakingly slow to progress and inexorably fatal. Patients rack up complications from the treatment, not because treatment is so toxic, but because they live long enough to suffer from the cure as well as the disease.

The core of the pathology is something called a plasma cell which under normal circumstances produces the antibodies that help fight off invading viruses and bacteria. In myeloma, a single plasma cell mutates and grows uncontrollably crowding out everything else in a patients bone marrow and gumming up their organs with immunoglobulin. The mutant cells eventually cant be contained inside the bone marrow and invade the surrounding solid bone causing painful fractures in the spine and long bones of the skeleton.

As if that was not bad enough, the core of chemotherapy is high dose steroids, usually dexamethasone or prednisone. Steroids are the poster child for double edged swords in medicine. They are simultaneously incredibly useful for suppressing the immune system in autoimmune diseases, cancer and anything that involves inflammation, while at the same time having the most broad ranging side effect profile of almost any medication.

It was a love for wildlands in the West that led Rob Patrick, at right, down the trail of Ivan Doig's books and when he had an opportunity to dive deeper into Doig's final years he jumped at the chance. Another thing that brought him to Bozeman and Greater Yellowstone is his close friendship with Kenning Arlitsch, Dean of MSU Libraries. Here they are on an autumn trip into the Yellowstone backcountry.

Probably the most serious side effects for myeloma patients are immunosuppression leading to increased risk for infection, a softening of the bones accelerating the tendency of the disease to cause fractures and emotional lability. The last of these sounds trivial, but isnt.

My first patient who suffered from this particular side effect literally started a sentence laughing and ended it crying. The cognitive effects can be especially debilitating, because at its peak, the drug lulls one into a false sense of security. It can make patients feel super human and I had one multiple sclerosis patient tell me it was the most powerful antidepressant she had ever taken and she almost looked forward to her next flare so she could get it again. However, on the way up and the way down it can cause confusion and a loss of emotional control that is profoundly disturbing, especially to someone who depends on their brain to make a living. Truly a blessing and a curse of modern medicine.

One of my biggest losses of innocence after medical school was realizing that professors had pulled the wool over my eyes concerning one of the fundamental diagnostic tools in medicinethe patient history. During the pre-clinical years you seldom get to talk to an actual patient and instead hone your skills using case presentations which I later came to understand were carefully curated stories masquerading as actual patients in which the non-salient details were conveniently expunged and the salient ones amplified for teaching purposes.

My teachers smugly told me, If you dont know whats wrong by the time you finish taking the history, take the history again." This illusion is perpetuated during third year clerkships when cases are cherry picked for medical students so as not to dispel the myth. The gloves come off during internship when it is too late to turn back and you realize that most patients have a hard time telling you how they feel no matter how many creative ways you come up with to ask the same question. Its not their fault, they usually just have never felt like this before and dont have the words to describe it.

When you add intoxication, mental illness, dementia, etc. to the mix, taking a history often becomes an exercise in communication breakdown and frustration. So imagine my joy at finding a history written by a professional communicator whose livelihood depended on his ability to observe the world and record it. It was like winning the internal medicine lottery.

Doig observed of myeloma: The waiting room of hell, furnished with side effects.

Of those side effects, he observed, The dex makes me longitudinal - - concentrated on a single line of endeavor at a time, no latitude to speak of and I would go to blow my nose and find there was not a handkerchief within 50 of me. Pill bottle caps leapt for the floor. My ordinary thought process resembles a homesteader digging out a stump, when loaded with dex I plodded right past nuances of life in temporary fixations on getting to my desk and writing things down. Which, amazingly, produced pages of a novel faster than when I wasnt taking the stuff. Dex gave me a mental pop, off-the-chart energy upstairs while it played games with the rest of me. Writing proved to be therapy for therapy.

On the topic of mortality, he explained in a written passage, I am now in remission, that terra incognita but better than being off the map(oblivion). He would add, I have not come out of this as any cheerleader for Nietzche: thera are countless preferable ways to strengthen in life without having something trying to kill you.

Facing the reality, he noted, Invariably fatal. Damn. But then, so is life. Its probably not polite to laugh out loud at the writings of a dying man, but I couldnt help myself and I also couldnt help wondering what a pleasure it would have been to take care of him. There was plenty of correspondence in the archive between Ivan and his doctors, but the most striking examples would probably have been overlooked by the uninitiated. The age of electronic medical records and e-mail allow patients unprecedented access to providers.

Like most technology, this chart messaging is both a blessing and a curse. The blessing is that it doesnt take three phone calls to catch a patient at home and tell them about their lab results.

The curse is the dozens of chart messages to return at the end of a busy day. Consequently, as Doig chronicled, brevity is the rule: Everything normal on your labs today, see you in 6 months."

Doig, when in his prime, trying to instill the lessons of history into his work. Here he absorbs the vibe in an abandoned farm house where heart-felt dreams rose, fell part like a heartache and drifted away. Photo by Carol Doig, courtesy MSU Library Doig Archives

The messages from Ivans providers went on for paragraphs, like post cards from your grandmother, and often came close to open displays of affection. All patients are equal, but some are more equal than others.

My day in the Doig archive was followed by an evening at the annual trout lecture hosted by the MSU library and I happened to find the only other Doig nerd who had spent any time with the medical journey files. Todd Wilkinson, the editor of Mountain Journal, shared my fascination with this little known part of Ivan Doigs life and suggested we pursue an event centered around his medical journey.

I couldnt imagine who else would show up to hear about such a niche topic, but didnt want to spoil the glow of our mutual fandom and encouraged him to pursue it. Three months later I found myself sitting in Carol Doigs living room in Seattle.This would be a good time to disclose that I am not a casual Doig fan. Im not a religious person, but I have made two literary pilgrimages in my life. The first was to Arches National Park to find the location of Edward Abbeys trailer from Desert Solitaire and the second was to White Sulfur Springs, Montana to see the place that had figured so prominently in Doig'sThis House of Sky.

Something still haunted me about the archives. Aside from the few pithy quotes above, there wasnt much mention of how Ivan faced his own mortality. How does an author get up every day and write four more novels when he knows hes dying? More importantly, why does he do it?

My practice over the last 20 years was working as a hospitalist. All of my patients were sick enough to be in the hospital and these days you have to be pretty sick to make it through those doors. I had seen hundreds of patients die and typically had end of life conversations with patients and families several times a week. Indeed, I had been on a personal crusade in the last few years to get doctors to talk with their terminally ill patients about their goals for the end of life and had coached other providers about how to do it.

So here was my chance to salvage something of my shattered romantic ideal about writers. Ivan must have had some big epiphany, I thought, that just wasnt there in his writing and my task was to extract it from his widow. I was as if a literary anthropologist on a mission.

It led to having a wonderful day in Seattle, sunny and warm; the Doig living room had a commanding view of Puget Sound. The house was spare and elegant and warm and inviting all at the same time and I got to see Ivans personal desk with his typewriter and his book collection.

Carol was charming and intelligent and well educated and everything you would expect from the spouse of your literary hero. Todd Wilkinson was there and Kenning Arlitsch, Dean of Libraries at MSU, too, and the person responsible for securing the Doig archive. Our conversation flowed easily.

Todd had a flurry of journalistic questions for Carol about Ivan and his writing. I was the final interviewer and my experience told me that it was almost always the wife that was the keeper of the medical history. So I started with some easy logistical questions.

Doig's desk, where he completed five books in eight years, battling through pain, the effects of medicines and a bone marrow transplant. All this and yet critics say these final works contain passages that are among the most incisive and moving of his four-decade long career. Photo by Todd Wilkinson

No, she did not go to all of his medical appointments with him and she didnt even know about the folder where he had kept all of the materials about his illness until after his death. There goes my first assumption.

We walked through the chronology of his illness, his initial diagnosis, the stem cell transplant, chemotherapy, remission, relapse, second line chemotherapy. What was daily life like? How did they deal with telling friends and family since he was not obviously ill until the end? How long was he on hospice? What was it like at the end? I probed, I rephrased, I asked the same question a different way. But there was no profound epiphany.

What she described instead was a guy who got up every day, made breakfast, went to his study and pounded out his words for the day. If he finished a novel on Friday, he started the next on Monday. A literary proletarian if there ever was one.

I have watched plenty of people die in my career, some face it with grace and dignity and resolve and some fight it and raise their fist against the sky until the last breath. What separated those who faced their end well from those who didnt?

Regret. Regret for things they hadnt done or relationships that had soured, but it boiled down to not living life the way they wanted to. My epiphany was that there was no epiphany. Epiphanies are extraneous when you are already living your best life. Ivan Doig was a wonderful writer, husband, friend, and colleague. If it isnt broken, dont fix it.

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A Doctor Plumbs The Depths Of Ivan Doig's Illness And Asks: 'Did He Have An Epiphany?' - Mountain Journal

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (NASDAQ: AVRO) (the Company) today announced that the first patient has been dosed in the Companys AVR-RD-04 investigational gene therapy program for cystinosis, a devastating lysosomal storage disease, in an ongoing Phase 1/2 clinical trial sponsored by academic collaborators at the University of California San Diego. The gene therapy is derived from the patients own hematopoietic stem cells, which are genetically modified to produce functional cystinosin, a crucial protein that patients with cystinosis lack.

The trial will enroll up to six patients with cystinosis, a rare inherited disease caused by a defect in the gene that encodes for cystinosin. The cystinosin protein enables transport of the amino acid cystine out of lysosomes. When it is absent, cystine accumulates and crystalizes, causing progressive damage to the kidneys, liver, muscles, eyes and other organs and tissues. Cystinosis affects both children and adults; they face shortened life spans and often painful symptoms, including muscle wasting, difficulty breathing, blindness and kidney failure.

Cystinosis is a debilitating and progressive disease, and new treatment options are sorely needed. The current standard of care does not avert deterioration; at best, it can attenuate symptoms. Thats why gene therapy is particularly exciting: It has the potential to change the course of disease -- and the lives of patients -- by addressing the underlying cause of cystinosis, said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. We believe we can engineer patients own stem cells so they sustainably produce the functional protein that is needed to prevent a toxic buildup of cystine and halt progression of the disease. We are so pleased that this investigational gene therapy is now in the clinic in collaboration with Dr. Stephanie Cherqui at UC San Diego.

The single-arm trial will enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis. If started at an early age and taken on a strict dosing schedule, cysteamine can delay kidney failure. However, the treatment regimen is highly burdensome, with side effects that can be severe and unpleasant, and many patients find it difficult to adhere to this treatment regimen. Even if compliance is high, cysteamine therapy cannot prevent kidney failure or avert other complications.

For people with cystinosis, there are no healthy days. They must take dozens of pills a day, around the clock, just to stay alive. It is a relentless disease and we urgently need new treatments, said Nancy J. Stack, President of the Cystinosis Research Foundation, which supported development of the gene therapy with more than $5.4 million in grants to Dr. Cherquis lab at UC San Diego. We believe that we are now an important step closer to the potential cure that our community has been working toward for many years.

The trials primary endpoints are safety and tolerability, assessed for up to two years after treatment, as well as efficacy, as assessed by cystine levels in white blood cells. Secondary endpoints to assess efficacy include changes in cystine levels in the blood, intestinal mucosa and skin and cystine crystal counts in the eye and skin. Efficacy will also be evaluated through clinical tests of kidney function, vision, muscle strength, pulmonary function and neurological and psychometric function, as well as through assessments of participants quality of life after treatment. The trial is funded by grants to UC San Diego from the California Institute for Regenerative Medicine (CIRM) as well as the Cystinosis Research Foundation.

This investigational gene therapy starts with the patients own stem cells, which are genetically modified so that their daughter cells can produce and deliver functional cystinosin to cells throughout the body. With this approach we aim to prevent the abnormal accumulation of cystine that causes so many devastating complications, said Stephanie Cherqui, PhD, an Associate Professor of Pediatrics at UC San Diego School of Medicine, and consultant to AVROBIO. We have been working toward this trial for years and we are grateful for all the support that brought us to this moment.

About AVR-RD-04

AVR-RD-04 is a lentiviral-based gene therapy designed to potentially halt the progression of cystinosis with a single dose of the patients own hematopoietic stem cells. The stem cells are genetically modified so they can produce functional cystinosin with the aim of substantially reducing levels of cystine in cells throughout the patients body. Before the infusion of the cells, patients undergo personalized conditioning with busulfan to enable the cells to permanently engraft. The Phase 1/2 clinical trial is being conducted under the name CTNS-RD-04 by AVROBIOs academic collaborators at the University of California, San Diego.

About Cystinosis

Cystinosis is a rare, inherited lysosomal storage disorder characterized by the accumulation of cystine in all the cells of the body, resulting in serious and potentially fatal damage to multiple organs and tissues and the shortening of patients life spans. The kidneys and eyes are especially vulnerable; more than 90% of untreated patients require a kidney transplant before age 20. An estimated 1 in 170,000 people are diagnosed with cystinosis.

About AVROBIO, Inc.

AVROBIO, Inc. is a leading, Phase 2 gene therapy company focused on the development of its investigational gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Companys plato platform includes a proprietary vector system, automated cell manufacturing solution and a personalized conditioning regimen deploying state-of-the-art precision dosing. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit http://www.avrobio.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as aims, anticipates, believes, could, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, including the ongoing Phase 1/2 trial of the Companys AVR-RD-04 investigational gene therapy, the anticipated benefits of our gene therapy platform, the expected safety profile of our product candidates, timing and likelihood of success of our current or future product candidates, and the market opportunity for our product candidates. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy for Cystinosis - Business Wire