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Archive for the ‘Stem Cell Complications’ Category

1st CRISPR Gene Editing Trial Slated to Open in Severe SCD Patients – Sickle Cell Anemia News

Sunday, April 4th, 2021

The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease(SCD).

Both the therapy and the upcoming Phase 1/2 trial planned to start this summer are the result of a collaboration between the Innovative Genomics Institute (IGI) and the University of California (UC), Los Angeles (UCLA).

IGI, a joint research initiative between UC Berkeley and UC San Francisco (UCSF), was founded by Berkeleys Jennifer Doudna, PhD. Doudna,along with Emmanuelle Charpentier of France, was awarded the 2020 Nobel Prize in Chemistry for groundbreaking work on the CRISPR-Cas9 gene editing tool.

Similar to the editing system used by bacteria as a defense mechanism, CRISPR-Cas9 allows researchers to edit parts of the genome by adding, removing, or changing specific sections of DNA.

We are motivated to work towards a cure that can be accessible and affordable to patients worldwide, Doudna said in a press release.

The launch of this trial is an essential first step on that path, added Doudna, who first approached the team at UCSF Benioff Childrens Hospital Oakland with the idea of developing a CRISPR/Cas9-based cure for SCD in 2014.

CRISPR_SCD001 uses the power of the CRISPR-Cas9 gene editing system to replace the mutatedHBB gene in a patients hematopoietic stem cells with a healthy version. The HBB gene, whose mutation cause SCD, provides the instructions to produce the beta subunit of hemoglobin, a protein found in red blood cells that transports oxygen.

These stem cells, which can give rise to all types of blood cells, are collected from a patients bone marrow, genetically modified in the lab with the innovative tool, and then returned to the patient in the form of a stem cell transplant.

By restoring production of normal hemoglobin and preventing red blood cells from becoming damaged and acquiring a sickle shape, the therapy is expected to be a potential cure for SCD.

Mark Walters, MD, the trials principal investigator and a professor of pediatrics at UCSF, said the goal of this form of genome-editing therapy is to correct the mutation in enough stem cells so the resulting blood in circulation has corrected red blood cells.

Based on previous bone marrow transplants, we predict that correcting 20% of the genes should be sufficient to out-compete the native sickle cells and have a strong clinical benefit, added Walters, who is also director of the blood and marrow transplant program at Benioff Childrens Hospital Oakland.

Unlike other investigational gene editing approaches for sickle cell, CRISPR_SCD001 delivers the CRISPR-Cas9 machinery to cells without relying on a virus as a transport agent. Its method, called electroporation, uses electrical pulses to create temporary pores in cell membranes that allow for the gene-editing tool to enter.

As such, the upcoming Phase 1/2 trial (NCT04774536) will mark the first attempt to correct the faulty HBB gene in a patients own cells with non-viral delivery of CRISPR/Cas9 gene correction tools.

The four-year study will evaluate the safety and effectiveness of a single dose of CRISPR_SCD001 in up to nine patients with severe SCD, ages 1235, who will be recruited at UCLA and Benioff Childrens Hospital Oakland.

If the therapy is found to be safe in the first six treated patients, all adults, the trial will proceed to enroll three adolescents, ages 1218, to evaluate its safety in younger patients.

This therapy has the potential to transform sickle cell disease care by producing an accessible, curative treatment that is safer than the current therapy of stem cell transplant from a healthy bone marrow donor, Walters said.

If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease, he added.

Donald Kohn, MD, the principal investigator at the UCLA trial site and who will oversee all therapy manufacturing for the study, said that in theory, gene therapy and gene-editing approaches should be much safer than a transplant from another person and could become universally available because they eliminate the need to find the needle in a haystack that is a matched stem cell donor.

Kohn, who has also been involved in the development of several gene therapies for other diseases, is a distinguished professor of microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA and a member of the UCLA Broad Stem Cell Research Center.

It is noteworthy that this new trial comes from a consortium of not-for-profit academic institutions incentivized with a long-term vision to cure the disease with an affordable solution that can globally benefit everyone who needs it, said Fyodor Urnov, PhD, IGIs director of technology and translation, who will oversee the trials bioinformatics and genomics activities.

Marta Figueiredo holds a masters in evolutionary and developmental biology and a PhD in biomedical sciences from the University of Lisbon, Portugal. Her research is focused on the role of several signaling pathways in thymus and parathyroid glands embryonic development.

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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that made up the lining of blood vessels found in the umbilical cord of newborns.

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Transplant After CD19 CAR T-Cell Therapy Shows Durable Disease Control in Children, Young Adults With B-ALL – Cancer Network

Sunday, April 4th, 2021

In a long-term follow-up of an early-phase trial examining CD19.28 chimeric antigen receptor (CAR) T-cell therapy, the use of consolidative allogeneic hematopoietic stem cell transplant (alloHSCT) was associated with long-term and durable disease control in children and young adults with B-cell acute lymphoblastic leukemia (B-ALL).

These results were based on a cohort of 20 patients who were initially treated in a dose-escalation part of a phase 1 trial (NCT01593696) examining anti-CD19 CAR T-cell therapy in patients between 1 and 30 years who have not responded to standard treatment, plus an additional 30 patients who were treated in an expansion portion. The median follow-up for all patients examined was 4.8 years and represents the longest time period examined for the use of this therapy in children and young adults with B-ALL.

We demonstrate that CD19.28 CAR T cells followed by a consolidative alloHSCT can provide long-term durable disease control in [child and young adult patients] with relapsed or refractory B-ALL, wrote the study investigators who were led by Nirali N. Shah, MD. Following alloHSCT, we observed a significant long-term [event-free survival (EFS)] with an apparent plateau and a low relapse rate, providing support for this sequential approach for long-term cure.

The complete response rate (CR) was 62.0%, with 28 of the 31 patients achieving this end point also reaching minimal residual disease (MRD) negativity by flow cytometry. The rate of CR was higher in patients with primary refractory disease (P = .0035), fewer prior lines of therapy (P = .033), and an M1 marrow (P = .0007). Additionally, CR rates were better for patients who received fludarabine/cyclophosphamidebased lymphodepletion versus other regimens, at 69% and 25%, respectively (P = .041).

The median overall survival (OS) for the cohort was 10.5 months (95% CI, 6.3-29.2 months). The median EFS was 3.1 months (95% CI, 0.9-7.7), with rates at 3 and 6 months of 52.0% (95% CI, 37.4%-64.7%) and 38.0% (95% CI, 24.8%-51.1%), respectively. Notably, median EFS was not reached in patients treated with M1 marrow versus 0.9 months in those with M2 marrow (P .0001).

Of the patients achieving MRD-negative CR (n = 28), 21 (75.0%) went on to receive consolidative alloHSCT, with a median time to transplant of 54 days from infusion (range, 42-97). The median OS from transplant day 0 was 70.2 months (95% CI, 10.4 months-not estimable) and the median EFS was not reached. The rate of EFS at 5 years was 61.9% (95% CI, 38.1%-78.8%). There were 8 deaths between 0.8 and 71 months following alloHSCT, which included transplant-related complications and/or graft-versus-host disease or infection in 6 patients and 1 patient with a complication of secondary malignancy at 3 years post-transplant. Teo patients relapsed after alloHSCT, with a cumulative risk of relapse was 4.8% (95% CI, 0.3-20.3) and 9.5% (95% CI, 1.5%-26.8%) at 12 and 24 months, respectively, with death as a competing risk.

Of note, achieving a CR was associated with greater CAR T-cell expansion and grade 3/4 cytokine release syndrome (CRS). Overall, CRS occurred in 70.0% of patients, with 9 (18.0%) having a grade 3/4 event. Neurotoxicity occurred in 10 patients (20.0%), with 4 having severe neurotoxicity.

Central nervous system involvement was effectively treated with CAR T-cell therapy all patients with a marrow response and CRS, although 1 patients did have residual disease by flow cytometry at low levels.

The authors noted that given these findings, CD19-directed CAR T-cell therapy may be considered as a bridge to alloHSCT versus standard-of-care blinatumomab (Blincyto).

Despite its more ready availability, which is not dependent on manufacturing time or success thereof, the efficacy of blinatumomab in children is lower than in adults receiving blinatumomab and also lower than remission rates following CD19-CAR T cells, using any construct, particularly for those with high-burden disease, the study author wrote. Therefore, selection of CAR T cells over blinatumomab may be advantageous in patients with higher-burden disease and [extramedullary] disease or as a salvage for blinatumomab nonresponders.

References

Shah NN, Lee DW, Yates B, et al. Long-Term Follow-Up of CD19-CAR T-Cell Therapy in Children and Young Adults With B-ALL. J Clin Oncol. March 25, 2021. doi: 10.1200/JCO.20.02262

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Timely Bone Marrow Transplant by Fortis gives new lease of life to a patient with Multiple Myeloma – APN News

Sunday, April 4th, 2021

Published on April 2, 2021

Recently, a 43-year-old man was presented at Fortis Hospital, Noida, complaining of severe back pain. Upon investigation, it was found that he was suffering from a rare disease, multiple myeloma which is a type of cancer that forms in the white blood cells or plasma cells. Here cancerous plasma cells accumulate in the bone marrow and crowd around the healthy blood cell that help in fighting infections by building antibodies, this puts the patients life at high risk. He therefore urgently required a Bone Marrow Transplant (BMT). Dr Rahul Bhargava, Director and Head, Hematology and Bone Marrow Transplant, Fortis Hospital, Noida and his team took a timely decision to go ahead with BMT to save his life.

A bone marrow transplant is a procedure to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Bone marrow is the soft, fatty tissue inside your bones. The bone marrow produces blood cells. Stem cells are immature cells in the bone marrow that give rise to different blood cells. Bone marrow transplant has now revolutionised. It is like a peripheral blood stem cell transplant, meaning, it is just like a blood transfusion (like platelet apheresis) which does not require any anesthesia.

Upon further investigation it was revealed that chemotherapy was required before BMT. Following the chemotherapy, on the 10thday of admission the team of doctors engulfed stem cells in the patients body which provided the body with a new source of healthy cells. Safe hospital environment and the doctors expertise in the area ensured that the BMT was done smoothly, without any complications and within 15 days the patient had been discharged. Usually, a patient takes 25-30 days to recover but here, due to patients will to recover and the facilities provided to him in the hospital he recovered at a faster pace.

Dr Rahul Bhargava, Director and Head, Hematology and Bone Marrow Transplant, Fortis Hospital, Noida, said,The case was complicated as the patient was suffering from high-risk multiple myeloma, which is a rare form of cancer. We took the necessary precautions and performed chemotherapy first to which he responded well and post that a Bone Marrow Transplant (BMT) was performed successfully. The process was smooth, and no complications arose during the same. We request patients to not fear BMT and undergo the process when required.

Talking about the clinical excellence at Fortis Hospital, NOIDA,Mr Hardeep Singh, Zonal Director, Fortis Hospital, Noidasaid, The team at Fortis Hospital, Noida try their best to save lives and do not give up even if there is 1% chance of survival. The patient was suffering from high-risk multiple myeloma for which immediate bone marrow transplant was required. The case was managed extremely well and with a lot of patience by Dr Rahul Bhargava and his team. I applaud the team of doctors for their continued commitment towards clinical expertise and patient care.

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Kirron Kher is suffering with Multiple Myeloma: Know the causes, symptoms and more about this type of blood cancer – Jagran English

Sunday, April 4th, 2021

Actress Kirron Kher is suffering from Multiple Myeloma, a type of blood cancer. Read to know the causes, symptoms, treatment, risks and more about the fatal disease.

New Delhi | Jagran Health Desk:Kirron Kher is suffering from Multiple Myeloma which is a type of blood cancer. The actress's husband Anupam Kher made an official announcement about her illness through a post on his official social media handle.

He wrote,"Just so that rumours don't get the better of a situation Sikandar and I would like to inform everyone that Kirron has been diagnosed with multiple myeloma, a type of blood cancer. She is currently undergoing treatment and we are sure she weill come out of this stronger than before. We are are very blessed that she is being looked after by a phenomenal set of doctors. She's always been a figher and takes things head on. She's all heart nd that's why she has so many people that love her. So keep sending your love to her in your prayers and in your heart. She is well on her way to recovery and we thank everyone for their support and love. Anupam and Sikander."

What is Multiple Myeloma?

Multiple Myeloma is a progressive hematologic disease. It is a cancer of plasma cells, which are type of blood cells in the bone marrow. When cells multiply unconditionally, they crowd out normal cells, therefore the body does not work the way it should. This disease causes damage to the immune system, bones, red blood cells etc. Cancerous plasma cells gather in the bone marrow, produce abnormal proteins, which causes complications.

What are the symptoms of Multiple Myeloma?

When the disease is in its early stage there may be no signs of symptoms as such. But some of the visible symptoms of this disease are as follows:

Diagnosis of Multiple Myeloma

Doctors advise diagnostic tests considering many factors such as:

Tests of Multiple Myeloma

Here are some of the tests which are done for diagnosis of multiple myeloma:

Causes of Multiple Myeloma

Although the causes of myeloma are not very clear. But, the disease occurs when abnormal cells multiply rapidly, they accumulate and crowd out healthy blood cells. Abnormal antibodies (monoclonal protein or M proteins) produced by myeloma cells cause problems, such as damage to kidneys, bones etc.

Complications during Multiple Myeloma

Risk Factors ofMultiple Myeloma

Treatment ofMultiple Myeloma

Treatment of Multiple Myeloma includes medication, chemotherapy, radiation, corticosteroids, stem-cell transplant etc.

Posted By: Sanyukta Baijal

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Decitabine Improved Outcomes for Patients With Refractory Prolonged Isolated Thrombocytopenia – Hematology Advisor

Sunday, April 4th, 2021

The use of decitabine was linked to improved platelet counts and survival rates in patients with refractory prolonged isolated thrombocytopenia (RPIT), which can be a complication of allogeneic hematopoietic cell transplantation (HCT), according to the results of a study recently published in Blood Advances.

Isolated thrombocytopenia is a frequent and severe complication of HCT that is associated with worse outcomes, the study investigators explained in their report. According to the investigators, RPIT has been thought to relate to such factors as disease recurrence, treatment history, factors related to the transplant donor, and other transplantation complications. However, they suggested that the absence of a consistent definition for prolonged isolated thrombocytopenia has hindered understanding of its patterns.

This prospective, phase 3 clinical trial (ClinicalTrials.gov Identifier: NCT02487563) included patients who had undergone allogeneic HCT and developed RPIT and who were treated at any of 6 participating hematology centers in China.

Patients were randomly assigned across 3 treatment arms: low-dose decitabine with recombinant human thrombopoietin (arm A), decitabine only (arm B), or conventional treatment (arm C). Platelet response at 28 days following treatment was the primary study endpoint, and this was defined as a sustained increase of 30 x 109/L or more, independent of transfusion for 3 days.

Across the participating centers, 2616 allogeneic HCT recipients were identified, of whom 256 had developed thrombocytopenia for more than 60 days following transplantation, and 97 met criteria for study inclusion. A total of 91 patients were evaluated for response.

The response rate for arm A was 66.7%, for arm B it was 73.3%, and for arm C it was 19.4%. Arms A and B were not statistically different for response (P =.779), while the response rate for arm C was statistically lower than the others (P <.001).

At a median follow-up of 11 months, the estimated 1-year survival rates were 64.4% for arm A and 73.4% for arm B, which were both greater than in arm C (41.0%). When the decitabine arms were combined, the survival rate was 68.2%, which was significantly higher than 1-year survival in arm C (P =.008).

The treatment arms receiving decitabine also showed significantly elevated total megakaryocyte counts, platelet-shedding megakaryocytes, and megakaryocyte polyploidy, while arm C did not. This suggested there are improvements in megakaryocyte proliferation and maturation with decitabine, according to the investigators.

In conclusion, this multicenter randomized study demonstrates the efficacy and safety of decitabine for patients with RPIT after HCT, with improved response and prolonged survival, the study investigators wrote in their report.

Reference

Tang Y, Chen J, Liu Q, et al. Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial. Blood Adv. 2021;5(5):1250-1258. doi:10.1182/bloodadvances.2020002790

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Lake in the Hills police officer and father of 4 kids battling rare cancer forced to retire – Lake and McHenry County Scanner

Sunday, April 4th, 2021

Lake in the Hills Police Officer Mike Domagala with one of his children. | Provided Photo.

A Lake in the Hills police officer with 20 years of service, who is a father of four kids, says he will no longer be able to work as he battles a rare blood cancer, and a fundraiser has been started for him.

Mike Domagala began his law enforcement career in Fox River Grove in 2002. He was hired by the Lake in the Hills Police Department in 2012.

Domagala, 43, was diagnosed in July with multiple myeloma, which is a rare blood cancer that is not curable but is treatable.

According to the Mayo Clinic, cancerous plasma cells accumulate in the bone marrow and crowd out healthy blood cells. The cancer cells produce abnormal proteins that can cause complications.

Domagala is married and has four children, ages 5, 12, 16, and 23. At the onset of the cancer discovery, he had to have a pelvic biopsy, two bone marrow biopsies, blood work, PET scans, CT scans, and MRIs, according to a GoFundMe account.

Domagala has undergone multiple cycles of chemotherapy that started on August 4 and then a stem cell transplant in December.

He has not been able to patrol as an officer since his diagnosis. On March 25, Domagala said in an update that he had his 100-day post bone marrow appointment, which went well.

I also had my hematologist appointment for my maintenance therapy which will be a chemo pill every day and a bone healing transfusion once a month. The overwhelming support my family and I have received through this has been amazing and helps me continue to fight through this difficult time, he said.

Domagala said in February that he was optimistic about getting back to work. However, he said in his March 25 update that his doctor told him he would not be able to return to working the streets as a police officer.

I have been a police officer for almost 20 years and have always wanted to be since I was a child. I have no idea what I am going to do but I will figure it out as this is still sinking in, he said.

Fellow police officer Erik Watters started a GoFundMe account for Domagala in November and it continues to bring in donations from the public.

The fundraiser money is going towards uncovered medical expenses, travel expenses for treatments and to help his family with everyday expenses.

Mike maintains a strong fighting spirit and finds his strength in his love for his family. Mikes greatest concern is in continuing to provide for his family while he covers all of his uncovered medical expenses, Watter said.

The GoFundMe has a $50,000 goal and has raised $28,800 so far as of Thursday.

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Insulin 100: How the road to a diabetes cure is yielding better treatments – News@UofT

Sunday, April 4th, 2021

The pancreas, saysGary Lewis, an endocrinologist at Toronto General Hospital and director of the Banting & Best Diabetes Centre at the University of Torontos Temerty Faculty of Medicine, is like an exquisitely sensitive and perfectly networked computer.

Second by second,he notes,the pancreassecretesjust the right amount ofinsulinor glucagontolower or raiseblood sugarintotheportal veinthat leadsdirectlyto the liver, the site of key metabolic processes. Insulingis then distributedto every tissue in the body via general circulation.

Thats one reason a cure for diabetes has proven elusive 100 years after the discovery of insulin.

Another big reason is the complexity of how the disease arises. In type 1 diabetes, the immune system destroys the insulin-producing beta cells of the pancreas, creating a life-threatening spike in blood sugar. Type 2 diabetes usually comes on more slowly, as the body becomes resistant to insulin or the pancreas cant produce enough of it.

Genetics play a role in both types. Exposure to viruses and other environmental effects may be a factor in type 1. Lifestyle factors, including weight gain and physical inactivity, are strongly linked to type 2.

The bottom line, says Lewis, is that diabetes is a multifactoral disease, and were not close to a cure.

Ask about treatments, though, and Lewis gets excited.

The last two decades have brought a plethora of clinical and research advances, from new drugs to boost and sensitize the body to insulin and promote weight loss, to lifestyle interventions that improve diet, continuous monitoring of blood sugar, long- and short-lasting insulin, better insulin pumps, pancreatic transplantsand pre-clinical stem cell and immunosuppressive therapies.

Progress on treatments has been fantastic, especially for type 2, Lewis says. Im very, very hopeful.

The distinction between treatment and cure in medicine is often unclear. And for the 3.6 million Canadians living with diabetes, the distinction matters less and lessif the goal is a full and healthy life.

Type 2 diabetes accounts for about 90 per cent of diabetes cases in Canada. Prevalence is rising, but Canadians with type 2 diabetes are living longer and have fewer diabetes-related complications.

The clinic doesnt look like it did 30 years ago, says Lewis, who mainly treats patients with type 2. We see fewer amputees, less blindness. Patients are generally healthier, and their prognosis is often excellent if they maintain their blood sugar target and other key parameters.

Weight loss is a cornerstone of treatments to lower blood sugar, and recent research has strengthened the link between weight reduction and type 2 diabetes management. Some people with type 2 can lose weight and control blood sugar through dietary changes and exercise alone.

Bariatric surgery is very effective for weight loss and often results in diabetes remission, although it comes with surgical risks and is expensive.

If we could prevent obesity, we could greatly reduce the incidence of type 2, Lewis says. And experiments have shown wecan get a remission withlifestyle changes, so we know what works.

The problem is broad implementation.

Ive tried to lose weight and I know how difficult it can be, especially in an environment of convenient and inexpensive calories, Lewis says. Moreover, factors such as income, education, ethnicity, access to healthy food and living conditions can make lifestyle changes that curb obesity nearly impossible.

Social determinants of health are overwhelmingly the most important influence on who gets type 2 diabetes, and how well or poorly they do with it, Lewis says.

Fortunately, dozens of new drugs for diabetes have hit the market in the last two decades.

Medications for weight loss round out the armamentarium, and some also protect against kidney damage and lower cardiac risk. Current therapies can reduce body weight up to 10 per cent, although a loss of 20 per cent or more would have a greater effect on outcomes for patients with type 2 diabetes, saysJacqueline Beaudry, an assistant professor of nutritional sciences at U of T who studies links between obesity, hormones and diet.

Beaudry is probing the biology that underpins these medications, including the gut hormones GLP-1 and GIP. They control blood glucose and reduce appetite, but scientists are unsure how.

If we could understand their mechanisms of action, we could design better drugs, Beaudry says.

For people with type 1 diabetes, continuous glucose monitors, insulin pumps and even automated closed-loopsystems that run on mobile apps to deliver insulin as-needed have radically changed the patient experience.

Sara Vasconcelos left),an assistant professor at U of Ts Institute of Biomedical Engineering, has worked withCristina Nostro (right), an associate professor in the department of physiology,and her team in the McEwen Stem Cell Institute at UHNto extend the survival and functionality of pancreatic precursor cells generatedfrom human stem cells.

Cell therapy could prove more liberating still.

University labs and biotechs are working on implantable devices that house insulin-producing cells derived from stem cells.

To that end,Cristina Nostro, an associate professor in the department of physiology in the Temerty Faculty of Medicine,and her team in the McEwen Stem Cell Institute at University Health Network recently discovered a more efficient way to generate and purify pancreatic precursor cells from human stem cells in the lab.

They have also found a way to vascularize those cells by working withSara Vasconcelos, an assistant professor at U of Ts Institute of Biomedical Engineering. Together, they have extended the survival and functionality of the cells in animal models of diabetes.

The biggest problem with these therapies is that the immune system rejects them. The same challenge currently hinders pancreas and islet transplants.

The immune system is an amazing machine, were luckyits so good, says Nostro. But its very difficult to control when it goes awry, as in autoimmune conditions.

Nostro is working with immunologists at the university on a method to protect insulin-producing beta cells from immune rejection, and she says many researchers in the field are now focused on immune-protective approaches.

Another strategy for type 1 diabetes is to tamp down the autoimmune response before the disease progresses. The idea is to prevent immune cells that damage the pancreas while the body still produces beta cells.

Groups around the world are bringing different ideas and creative approaches to treat type 1 diabetes, thats the beauty of science, says Nostro. I am very hopeful about what the future holds. Who knows? Maybe we will see hybrid technologies combining a pump and cells. We have to keep an open mind.

This story was originally published in U of T Med Magazines Insulin Issue.

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Boxcar Scars Market |Exclusive Report on Latest Trends and Market Growth Opportunities – BioSpace

Sunday, April 4th, 2021

Boxcar scars are a type of acne scars which look like round, oval depression. As the scars are of different types, for example, based on redness, depth or location, its treatment also varies. Microdermabrasion, Dermabrasion, Fillers, Chemical Peels, Laser Therapy, Microneedling, Punch Excision, and Subcision, are some of the treatments required for treating boxcar scars. As these scars cant go away of their own, People considering the treatment is growing.

Growing Preference for Micro needling and Ablative Lasers

The ablative lasers are considered as the gold standard for treating acne scars, patients have witnessed a 75% improvement in atrophic acne scars at 18 months after this high energy carbon dioxide laser treatment. The technique when used on dark skinned people showed good to excellent results in 74%, however hyper pigmentation was witnessed among 29% of patients. Due to its long time recovery process and various side effects, the ablative lasers have become less popular.

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Of late, microneedling is gaining momentum as they provide the best cosmetic outcomes. Due to its collagen inducing effect, the encouraging results prove that microneedling is an effective and inexpensive method for dealing with boxcar scars. It takes very less time for recovering, a study found that only after 3 treatments, patients can visibly see the positive effect in their scarring and is relatively risk free. Microneedling can be performed with a dermaroller or a microneedling pen. Microneedling involves puncturing the skin multiple times using needles, a tattoo gun or roller. When microneedling is combined with platelet rich plasma or glycolic acid peels, improvement in acne scar improved up to 62%.

Emerging Technologies Can Boost Adoption of Boxcar Scar Treatment

Researchers and scientists are always on the motion of developing and introducing new and more effective treatments for replacing the traditional therapies. For instance, a latest technology for the treatment of scarring is laser speckle contrast imaging (LSCI). LSCI helps in detecting the backscatter which eventually detects the blood flow by illuminating the tissue with its coherent laser light. The technique is relevant for scarring because the healing process of scarring requires adequate tissue perfusion. LSCI can also be used to treat patients with burn wounds as it detects the severity of partial thickness in wounds.

Complications in Laser Therapy

Side effects due to laser therapy such as burns, dyspigmentation and infection may happen after the laser treatment. Laser treatment has a risk of overheating the tissue through excessive heat generation or by a failure of the cooling techniques. The risk of burns is higher for lasers that use a continuous beam. Risk of dyspigmentation is higher in dark skinned or tan individuals. Due to such side effects, the treatment by laser may go back scale. However, latest innovations in the sector may help in mitigating the issue.

Competitive Landscape

The key players in the market include Merz, Inc., Cerave, Lumenis, Enaltus LLC, Scarsheal, Inc., CCA Industries, Proactiv Company, Cynosure, Inc., PCA Skin, Solta Medical, Smith and Nephew plc, Scarheal, Inc., NewMedical Technology, Inc., Bausch Health, Suneva Medical, Inc., Sonoma Pharmaceuticals, Inc., etc.

Latest development by doi.org shows 755nm picosecond Alexandrite laser has been effective in patients with acne scars. In the split face study, people were treated with laser in half of their face and that half showed effective improvement results in Post inflammatory erythema and acne scars. Patients stated that treatment was tolerable, with only mild erythema discovered as a side effect.

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A study reported in the Journal of cosmetic dermatology in February 2020 by Darrow Stem Cell Institute demonstrated that platelet rich plasma shows better response, fewer side effects, and shorter downtime as compared to combined subcision and PRP. The experiment was performed on 45 patients with atrophic acne scars by dividing the group into 3 and giving intradermal injection to first group, chemical reconstruction of skin scars was performed on second group and combined skin needling and PRP was performed on the third group. The third group witness significant improvement without any major side effect thus, reaching to the conclusion that PRP is beneficial for acne scars.

PICOCARE 450 is a US FDA approved machine developed by WonTech for laser skin care treatment. The innovative machine is responsible for treating all skin types, show faster visible results in less sitting. The technology targets only the pigment to be removed and is suitable for treating chickenpox scars, ice pink scars, boxcar scars, acne scars, etc.

Regional Outlook

According to WHO, scars affect almost 80-90% of teenagers in the western world. As per the study by National Library of Medicine, boxcar scars are prevalent and is almost in 54% population as post acne scar. Thus, rising incidences of burn cases and also increasing prevalence of boxcar scar cases is expected to drive the market during the forecast period. According to the survey by Harris Poll around 10 million patients who have had dermal filler have experienced filler treatment a good option, thus, its demand is also one of the factor for the boost of boxcar scar market.

Asia Pacific is also leading market for the boxcar scar treatment. Due to increased incidences of burns in India having a record of 70lakh burn injury cases every year, the adoption of treatment is also expected to surge. Rising awareness about the treatment of scars is yet another factor boosting the growth. Japan in Asian region is an ideal market due to availability of various treatment options owing to the increase in health care expenditure.

UK is a major contributor to the European market. According to British Association of Plastic, rising number of plastic surgery units in UK is fuelling the adoption of large number of incidences. Microneedling as a treatment is escalating in Europe owing to its benefits for skin tightening, better skin texture, scar reduction, improved skin tone, etc. Moreover, growing adoption of anti-ageing procedures, and awareness regarding the treatments is escalating the market further.

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Segmentation

By Treatment Type

By Laser Product

By End-user

By Region

Key Questions Answered

Carbon dioxide and Pulse dyed laser are some of the laser products used for boxcar scar treatment

Proactiv Company, Cynosure, Inc., PCA Skin, Solta Medical, Smith and Nephew plc, Scarheal, Inc., NewMedical Technology, Inc., Bausch Health, etc.

Latest innovations, growing awareness regarding the treatment, and comfortable treatment is giving people the confidence to treat.

US, UK, France, Germany and Italy are some of the largest markets for boxcar scars

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Boxcar Scars Market |Exclusive Report on Latest Trends and Market Growth Opportunities - BioSpace

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Merck Receives Positive EU CHMP Opinion for Updated Label of KEYTRUDA (pembrolizumab) To Include Results of Phase 3 KEYNOTE-361 Trial in Certain Adult…

Sunday, April 4th, 2021

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 (EMA) has adopted a positive opinion recommending that the European label for KEYTRUDA, Mercks anti-PD-1 therapy, be updated to include data from KEYNOTE-361, a Phase 3, open-label trial that evaluated KEYTRUDA as a monotherapy and in combination with chemotherapy for the first-line treatment of certain patients with advanced or metastatic urothelial carcinoma. In Europe, KEYTRUDA is approved for the treatment of adult patients with advanced or metastatic urothelial carcinoma (bladder cancer) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 with a Combined Positive Score (CPS) 10. This approval was based on a single-arm study, KEYNOTE-052; KEYNOTE-361 was conducted as part of a post-marketing commitment following the initial approval of KEYTRUDA for these patients.

As previously announced, KEYNOTE-361 did not meet its primary endpoints of progression-free survival (PFS) and overall survival (OS) for the combination of KEYTRUDA plus chemotherapy. However, the CHMP concluded that the benefit-risk profile remains positive and that including data from KEYNOTE-361 in the label allows physicians to evaluate the potential benefit-risk of KEYTRUDA on an individual basis.

KEYTRUDA has become an important treatment option for certain patients with locally advanced or metastatic bladder cancer in the European Union and other countries around the world, said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. We are pleased with todays positive opinion by the CHMP, which fulfills our post-marketing requirement for KEYTRUDA in these patients in the European Union and will enable continued access for patients in need of another treatment option.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA 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 1,400 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 patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications in the U.S.

Melanoma

KEYTRUDA 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 Cancer

KEYTRUDA, 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.

Head and Neck Squamous Cell Cancer

KEYTRUDA, 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 Lymphoma

KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA 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. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA 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 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.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High or Mismatch Repair Deficient Cancer

KEYTRUDA 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.

Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer

KEYTRUDA is indicated for the first-line treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).

Gastric Cancer

KEYTRUDA 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 Cancer

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or gastroesophageal junction (GEJ) (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:

Cervical Cancer

KEYTRUDA 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 Carcinoma

KEYTRUDA 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 Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). 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 Carcinoma

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

Tumor Mutational Burden-High

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. 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 TMB-H central nervous system cancers have not been established.

Cutaneous Squamous Cell Carcinoma

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) that is not curable by surgery or radiation.

Triple-Negative Breast Cancer

KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test. This indication is approved under accelerated approval based on progression-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Selected Important Safety Information for KEYTRUDA

Severe and Fatal Immune-Mediated Adverse Reactions

KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the programmed death receptor-1 (PD-1) or the programmed death ligand 1 (PD-L1), blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.

Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of antiPD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.

Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% of these patients interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.

Hepatotoxicity and Immune-Mediated Hepatitis

KEYTRUDA as a Single Agent

KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.

KEYTRUDA with Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently 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. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (ALT) (20%) and increased aspartate aminotransferase (AST) (13%) were seen, which was at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT 3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT 3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT 3 ULN subsequently recovered from the event.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Hypophysitis

KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Thyroid Disorders

KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.

Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients receiving KEYTRUDA as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.2%) and Grade 2 (10.8%) hypothyroidism.

Type 1 Diabetes Mellitus (DM), Which Can Present With Diabetic Ketoacidosis

Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold KEYTRUDA depending on severity. Type 1 DM occurred in 0.2% (6/2799) of patients receiving KEYTRUDA. It led to permanent discontinuation in <0.1% (1) and withholding of KEYTRUDA in <0.1% (1). All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Immune-Mediated Nephritis With Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Immune-mediated nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.1%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 89% (8/9) of patients. Nephritis led to permanent discontinuation of KEYTRUDA in 0.1% (3) and withholding in 0.1% (3) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Nephritis resolved in 56% of the 9 patients.

Immune-Mediated Dermatologic Adverse Reactions

KEYTRUDA can cause immune-mediated rash or dermatitis. Exfoliative dermatitis, including Stevens-Johnson syndrome, drug rash with eosinophilia and systemic symptoms, and toxic epidermal necrolysis, has occurred with antiPD-1/PD-L1 treatments. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate nonexfoliative rashes. Withhold or permanently discontinue KEYTRUDA depending on severity. Immune-mediated dermatologic adverse reactions occurred in 1.4% (38/2799) of patients receiving KEYTRUDA, including Grade 3 (1%) and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 40% (15/38) of patients. These reactions led to permanent discontinuation in 0.1% (2) and withholding of KEYTRUDA in 0.6% (16) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 6% had recurrence. The reactions resolved in 79% of the 38 patients.

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of <1% (unless otherwise noted) in patients who received KEYTRUDA or were reported with the use of other antiPD-1/PD-L1 treatments. Severe or fatal cases have been reported for some of these adverse reactions. Cardiac/Vascular: Myocarditis, pericarditis, vasculitis; Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barr syndrome, nerve paresis, autoimmune neuropathy; Ocular: Uveitis, iritis and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss; Gastrointestinal: Pancreatitis, to include increases in serum amylase and lipase levels, gastritis, duodenitis; Musculoskeletal and Connective Tissue: Myositis/polymyositis rhabdomyolysis (and associated sequelae, including renal failure), arthritis (1.5%), polymyalgia rheumatica; Endocrine: Hypoparathyroidism; Hematologic/Immune: Hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% of 2799 patients receiving KEYTRUDA. Monitor for signs and symptoms of infusion-related reactions. Interrupt or slow the rate of infusion for Grade 1 or Grade 2 reactions. For Grade 3 or Grade 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Fatal and other serious complications can occur in patients who receive allogeneic HSCT before or after antiPD-1/PD-L1 treatment. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute and chronic GVHD, hepatic veno-occlusive disease after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between antiPD-1/PD-L1 treatment and allogeneic HSCT. Follow patients closely for evidence of these complications and intervene promptly. Consider the benefit vs risks of using antiPD-1/PD-L1 treatments prior to or after an allogeneic HSCT.

Increased Mortality in Patients With Multiple Myeloma

In 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 an antiPD-1/PD-L1 treatment in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based 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 Reactions

In 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 with advanced NSCLC; 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%).

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.

Link:
Merck Receives Positive EU CHMP Opinion for Updated Label of KEYTRUDA (pembrolizumab) To Include Results of Phase 3 KEYNOTE-361 Trial in Certain Adult...

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BeyondSpring Announces Submission of New Drug Application to US FDA and China NMPA for Plinabulin and G-CSF Combination for the Prevention of…

Sunday, April 4th, 2021

NEW YORK, March 31, 2021 (GLOBE NEWSWIRE) -- BeyondSpring Inc. (the Company or BeyondSpring) (NASDAQ: BYSI), a global biopharmaceutical company focused on the development of innovative cancer therapies, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and the China National Medical Products Administration (NMPA) for use of plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia (CIN). Plinabulin in combination with a G-CSF therapy, which received breakthrough therapy designation from the U.S. FDA and the China NMPA for concurrent administration with myelosuppressive chemotherapeutic regimens in patients with non-myeloid malignancies for the prevention of CIN, has the potential to raise the standard of care in CIN for the first time in 30 years.

CIN remains a severely unmet medical need. Treatment or prevention of CIN with G-CSF has been the standard of care since Neupogen was approved in 1991. The main benefit of G-CSF treatment, however, is in Week 2 after chemotherapy. Week 1 after chemotherapy is considered the neutropenia vulnerability gap where over 75% of CIN-related clinical complications occur, including febrile neutropenia, infection, hospitalization and death. Plinabulin is the first agent seeking FDA approval that has the potential to fill this gap by working in Week 1 to prevent the onset and progression of CIN. Therefore, combining plinabulin and G-CSF may maximize the protection of patients for the full cycle of chemotherapy, as demonstrated in the PROTECTIVE-2 Phase 3 registration study.

CIN is a major concern for physicians and their patients undergoing cancer treatment. Plinabulin provides benefits above and beyond what is currently available on the market and has the potential to be a game-changer for patients undergoing chemotherapy treatment, said Dr. Douglas Blayney, Professor of Medicine at Stanford University Medical School and global PI for CIN studies. CIN, which can lead to life-threatening infections, is the number one reason for the 4Ds in chemotherapy (Decrease, Delay and Discontinue dose and Downgrade regimen). We hope plinabulin will allow patients to better tolerate chemotherapy, thus enabling patients to stick to their optimal treatment plan and avoid serious CIN complications.

The NDA submission is based on positive data from BeyondSprings PROTECTIVE-2 Phase 3 registration study which showed that plinabulin in combination with pegfilgrastim demonstrated superior CIN prevention benefit, compared to pegfilgrastim alone. The study met the primary endpoint, with a statistically significant improvement in the rate of prevention of grade 4 neutropenia (improved from 13.6% to 31.5%, p=0.0015) and met all key secondary endpoints, including duration of severe neutropenia (DSN) and absolute neutrophil count (ANC) nadir. In addition, the combination reduced clinical complications such as incidence and severity of febrile neutropenia (FN) and incidence and duration of hospitalization for FN patients. The combination is well tolerated, with an over 20% reduction of grade 4 Treatment-Emergent Adverse Events (TEAE) in the combination compared to that of pegfilgrastim alone. The NDA submissions will include five supportive trials that show consistent CIN prevention in various chemotherapy regimens and cancers in over 1,200 patients.

This NDA submission is the culmination of years of research to prove that plinabulin can improve the long-established standard of care and address an unmet medical need to further alleviate the risk burden of CIN for patients receiving chemotherapy, said Dr. Lan Huang, co-founder, CEO, and chairman of BeyondSpring. With CIN responsible for potentially delaying treatment and causing life-threatening infections, we hope that receiving the improved care represented by the plinabulin and G-CSF combination will allow patients to better tolerate chemotherapy and potentially see increased treatment success rates. We are grateful for the patients participation in plinabulins clinical trials and the participation and contributions of our investigators and our many other clinical partners.

Each year in the U.S., 110,000 patients receiving chemotherapy are hospitalized after developing CIN, a severe side effect that increases the risk of infection with fever (also called febrile neutropenia, or FN), which necessitates ER/hospital visits. Due to the COVID-19 pandemic, the updated National Comprehensive Cancer Network (NCCN) guidelines expanded the use of prophylactic G-CSFs, including pegfilgrastim, from high-risk patients only (chemo FN rate >20%), to include intermediate-risk patients (FN rate between 10-20%), to reduce the number of hospital/ER visits related to CIN. The revision of the NCCN guidelines effectively increases the addressable market of patients who may benefit from treatment with plinabulin, if approved, to approximately 440,000 cancer patients in the U.S. annually.

There is a large unmet medical need and a growing market for CIN prevention and treatment in China as well. According to Lancet Oncology, 60% of East Asia cancer patients are treated with chemotherapy1. In 2020, there were 4.6 million new cancer patients in China which could correspond to 2.8 million patients using chemotherapy and needing CIN prevention agents. According to IQVIA data, the G-CSF drug market (for CIN treatment) in China is growing at over 30% a year.

About PROTECTIVE-2 (Study 106) Phase 3 Registration Study The Phase 3 portion of PROTECTIVE-2 was a double-blind and active-controlled global registration study. It was designed as a superiority study to compare the safety and efficacy of plinabulin (40 mg, Day 1 dose) + pegfilgrastim (6 mg, Day 2 dose) versus a single dose of pegfilgrastim (6 mg, Day 2 dose) in patients with breast cancer, treated with docetaxel, doxorubicin and cyclophosphamide (TAC, Day 1 dose) in a 21-day cycle. TAC is an example of high FN risk chemotherapy and is the regimen used in all G-CSF biosimilar registration studies.

The primary endpoint was the rate of prevention of Grade 4 neutropenia and secondary endpoints included DSN and mean ANC nadir in Cycle 1. Literature shows that despite the use of pegfilgrastim, 83 to 93 percent of patients treated with TAC still suffer Grade 4 neutropenia (or rate of Grade 4 neutropenia prevention at 7-17%), which demonstrates the severe unmet medical need for improved treatment2,3.

The ANC data, which are used to calculate these endpoints, were obtained through central laboratory assessments by Covance Bioanalytical Methods using standardized and validated analytical tests. Covance was the clinical contract research organization (CRO) for patient recruitment and monitoring of global sites for this study.

About CINChemotherapy-induced neutropenia (CIN) is the primary dose-limiting toxicity in cancer patients who receive chemotherapy and is the primary cause for the 4Ds (Decrease, Delay, Discontinue dose and Downgrade regimen). The 4Ds lead to a decrease of the anti-cancer benefit of chemotherapy, e.g., >15% of dose reduction correlated to >50% survival reduction4. The National Comprehensive Cancer Network (NCCN) recently updated its treatment guidelines for CIN prophylaxis using G-CSFs to include both high- and intermediate-FN risk patients treated with chemotherapies, to preserve hospital and ER resources for COVID-19 patients, and to maximize protection from CIN. The NCCNs action effectively doubled the number of patients recommended to receive CIN prophylaxis.

About PlinabulinPlinabulin, BeyondSprings lead asset, is a selective immune-modulating microtubule-binding agent (SIMBA). A global Phase 3 clinical trial in CIN (PROTECTIVE-2) with plinabulin in combination with pegfilgrastim versus pegfilgrastim alone has been completed and is the basis for an NDA filing in the U.S. and China for the prevention of CIN. In this trial, plinabulin reduced the neutropenia vulnerability gap associated with G-CSF therapy alone. Additionally, a global Phase 3 study for the treatment of later-stage NSCLC in EGFR wild-type patients (DUBLIN-3) is now fully enrolled and will evaluate the combination of plinabulin and docetaxel versus docetaxel alone for overall survival in NSCLC patients. Plinabulin triggers the release of the immune defense protein, GEF-H1, which leads to two distinct effects: first is a durable anticancer benefit due to the maturation of dendritic cells resulting in the activation of tumor antigen-specific T-cells to target cancer cells5,6 and the second is early-onset action in CIN prevention after chemotherapy by boosting the number of hematopoietic stem/progenitor cells (HSPCs)7. Effects on HSPCs could explain the potential for plinabulin not only to prevent CIN but also to increase circulating CD34+ cells in patients. As a pipeline in a drug, plinabulin is being broadly studied in combination with various immuno-oncology agents that could boost the effects of the PD-1 / PD-L1 antibodies.

About BeyondSpringHeadquartered in New York City, BeyondSpring is a global biopharmaceutical company focused on developing innovative immuno-oncology cancer therapies to improve clinical outcomes for patients who have high unmet medical needs. BeyondSprings first-in-class lead asset plinabulin is a pipeline in a drug. It is filed for approval in the US and China for the prevention of chemotherapy-induced neutropenia (CIN) and has a fully enrolled pivotal study to test an anti-cancer benefit with an overall survival primary endpoint in non-small cell lung cancer (NSCLC). Additionally, it is being broadly studied in combination with various immuno-oncology agents that could boost the effects of PD-1 / PD-L1 antibodies. In addition to plinabulin, BeyondSprings extensive pipeline includes three pre-clinical immuno-oncology assets and a subsidiary, SEED Therapeutics, which is leveraging a proprietary targeted protein degradation drug discovery platform.

References:

Investor Contact:Ashley R. RobinsonLifeSci Advisors, LLC+1 617-430-7577arr@lifesciadvisors.com

Media Contact:Darren Opland, Ph.D.LifeSci Communications+1 646-627-8387darren@lifescicomms.com

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Types of leukemia: Prevalence, treatment options, and prognosis – Medical News Today

Sunday, February 14th, 2021

Leukemia is a type of cancer that affects the blood and bone marrow, where blood cells are formed. All types of leukemia cause rapid, uncontrolled growth of abnormal bone marrow and blood cells.

The main differences between the types include how fast the disease progresses and the types of cells it affects.

There are four main types of leukemia, which we describe in detail below:

Lymphocytic leukemia affects the lymphocytes, a type of white blood cell. Myeloid leukemia can affect the white blood cells, red blood cells, and platelets.

According to the National Cancer Institute, roughly 1.5% of people in the United States will receive a leukemia diagnosis at some point.

In this article, explore the four main types, their symptoms, the treatment options available, and the outlook.

The full name of this type of cancer is acute lymphocytic leukemia, and acute means that it grows quickly. Lymphocytic means that it forms in underdeveloped white blood cells called lymphocytes.

The disease starts in the bone marrow, which produces stem cells that develop into red and white blood cells and platelets.

In a healthy person, the bone marrow does not release these cells until they are fully developed. In someone with ALL, the bone marrow releases large quantities of underdeveloped white blood cells.

There are several subtypes of ALL, and the subtype may influence the best course of treatment and the prognosis.

One subtype is B-cell ALL. This begins in the B lymphocytes, and it is the most common form of ALL in children.

Another subtype is T-cell ALL. It can cause the thymus, a small organ at the front of the windpipe, to become enlarged, which can lead to breathing difficulties.

Overall, because ALL progresses quickly, swift medical intervention is key.

As research from 2020 acknowledges, healthcare providers still do not know what causes ALL. It may occur due to genetic factors or exposure to:

Although genetic factors may play a role, ALL is not a familial disease.

Learn more about ALL here.

ALL is the most common form of leukemia in children.

The risk of developing it is highest in children under 5 years old. The prevalence slowly rises again in adults over 50.

ALL symptoms can be nonspecific difficult to distinguish from those of other illnesses.

They may include:

In a person with AML, the bone marrow makes abnormal versions of platelets, red blood cells, and white blood cells called myeloblasts.

The full name of this disease is acute myeloid leukemia, and acute refers to the fact that it is fast-growing.

It forms in one of the following types of bone marrow cell:

Doctors classify AML by subtype, depending on:

AML can be difficult to treat and requires prompt medical attention.

Learn more about AML here.

The most common risk factor is myelodysplastic syndrome, a form of blood cancer that keeps the body from producing enough healthy blood cells.

Other factors that increase the risk of developing AML include:

Most people who develop AML are over 45. It is one of the most common types of leukemia in adults, though it is still rare, compared with other cancers.

It is also the second most common form of leukemia in children.

Symptoms of AML can vary and may include:

CLL is the most common form of leukemia among adults in the U.S. and other Western countries.

There are two types. One progresses slowly, and it causes the body to have high levels of characteristic lymphocytes, but only slightly low levels of healthy red blood cells, platelets, and neutrophils.

The other type progresses more quickly and causes a significant reduction in levels of all healthy blood cells.

In someone with CLL, the lymphocytes often look fully formed but are less able to fight infection than healthy white blood cells. The lymphocytes tend to build up very slowly, so a person might have CLL for a long time before experiencing symptoms.

Learn more about CLL here.

Genetic factors are the most likely cause. Others might include:

CLL is rare in children. It typically develops in adults aged 70 or over. However, it can affect people as young as 30.

CLL typically causes no early symptoms. When symptoms are present, they may include:

Also, 5090% of people with CLL have swollen lymph nodes.

CML is a slow-growing type of leukemia that develops in the bone marrow.

The full name of CML is chronic myeloid leukemia. As the American Cancer Society explain, a genetic change takes place in the early forms of the myeloid cells, and this eventually results in CML cells.

These leukemia cells then grow, divide, and enter the blood.

CML occurs due to a rearrangement of genetic material between the chromosomes 9 and 22.

This rearrangement fuses a part of the ABL1 gene from chromosome 9 with the BCR gene from chromosome 22, called the Philadelphia chromosome. The result of this fusion is called BCR-ABL1.

BCR-ABL1 produces a protein that promotes cell division and stops apoptosis, the process of cell death, which typically removes unneeded or damaged cells.

The cells keep dividing and do not self-destruct, resulting in an overproduction of abnormal cells and a lack of healthy blood cells.

This occurs during the persons lifetime and is not inherited.

CML typically affects adults. People aged 65 and older make up almost half of those who receive a CML diagnosis.

The symptoms of CML are unclear, but they may include:

The symptoms may vary, depending on the type of leukemia. Overall, a person should get in touch with a doctor if they experience:

Learn more about the symptoms of leukemia here.

Treatment for ALL typically involves three basic phases: induction, consolidation, and maintenance. We describe these in detail below.

Treatment for AML involves the first two phases. The induction phase may include treatment with the chemotherapy drugs cytarabine (Cytosar-U) and daunorubicin (Cerubidine) or idarubicin (Idamycin). The doctor may also recommend targeted drugs.

The goal of this phase is to kill the leukemia cells, causing the cancer to go into remission, using chemotherapy.

The doctor may recommend:

People having chemotherapy may need to see their doctors frequently and spend time in the hospital, due to the risk of serious infections and complications.

This phase of the treatment lasts for about 1 month.

Even if the treatment so far has led to remission, cancer cells may be hiding in the body, so more treatment is necessary.

The consolidation phase may involve taking high doses of chemotherapy. A doctor may also recommend targeted drugs or stem cell transplants.

This phase, consisting of ongoing chemotherapy treatments, usually lasts for 2 years.

Since CLL tends to progress slowly, and its treatment can have unpleasant side effects, some people with this condition go through a phase of watchful waiting before starting the treatment.

For a person with CML, the focus is often on providing the right treatment for the phase of the illness. To do this, a doctor considers how quickly the leukemia cells are building up and the extent of the symptoms. Stem cell transplants can be effective, but further treatment is necessary.

Overall, the initial treatment tends to include monoclonal antibodies, targeted drugs, and chemotherapy.

If the only concern is an enlarged spleen or swollen lymph nodes, the person may receive radiation or surgery.

If there are high numbers of CLL cells, the doctor may suggest leukapheresis, a treatment that lowers the persons blood count. This is only effective for a short time, but it allows the chemotherapy to start working.

For people with high-risk disease, doctors may recommend stem cell transplants.

A persons prognosis depends on the type of leukemia.

Learn more about survival rates for people with leukemia here.

About 8090% of adults with ALL experience complete remission for a while during treatment. And with treatment, most children recover from the disease.

Relapses are common in adults, so the overall cure rate is 40%. However, factors specific to each person play a role.

The older a person is when they receive an AML diagnosis, the more difficult it is to treat.

More than 25% of adults who achieve remission live for 3 years or more after treatment for AML.

A person may live for a long time with CLL.

Treatments can help keep the symptoms under control and prevent the disease from spreading. However, there is no cure.

Stem cell transplants can cure CML. However, this treatment is very invasive and is not suitable for most people with CML.

The United Kingdoms National Health Service estimate that 70% of males and 75% of females live for at least 5 years after receiving a CML diagnosis.

The earlier a person receives the diagnosis, the better their outlook.

Leukemia is a type of cancer that affects the blood and bone marrow. It can affect people of all ages.

There are four main types of leukemia. They differ based on how quickly they progress and the types of cells they affect.

Treatments for all types of leukemia continue to improve, helping people live longer and more fully with this condition.

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Roche receives first FDA clearance for urine sample type for BK virus quantitative test to aid in the improvement of care for transplant patients -…

Sunday, February 14th, 2021

BKV can cause severe complications in immunocompromised transplant patients. Higher BKV DNA levels can often be present in urine prior to plasma, serving as an early predictor of an impending infection. A urine sample stabilised in cobas PCR Media allows the integrity of urine results to be maintained, making storage and transportation simpler without the need for sample refrigeration.

"Transplant patients face a number of significant challenges, including complications that can arise from viruses like BKV," said Ann Costello, Head Roche Diagnostic Solutions. "With the FDA clearance of this non-invasive and easily collectable sample type, we now offer choices for clinicians using a standardised, automated solution to routinely monitor and manage infection risks. Together with our viral load tests for Cytomegalovirus and Epstein-Barr virus, we are committed to bringing better care to transplant patients."

The cobas BKV Test runs on the widely available, high-throughput cobas 6800/8800 Systems. It is also approved for use in CE markets with EDTA plasma and urine stabilised in cobas PCR Media as sample types.

About the cobas BKV TestThe cobas BKV Test is a real-time polymerase chain reaction (PCR) test with dual-target technology that provides quantitative accuracy and guards against the risk of sequence variations that may be present in the BK virus. The cobas BKV Test has robust coverage with a limit of detection of 21.5 IU/mL and an expanded linear range from 21.5 IU/mL to 1E+08 IU/mL in EDTA plasma. Urine stabilised in cobas PCR Media has a limit of detection of 12.2 IU/mL and a linear range from 200 IU/mL to 1E+08 IU/mL.

The test offers an alternative to lab-developed tests (LDTs) or Analyte Specific Reagent (ASR) combinations, potentially minimising variability and complexity in testing, reducing workload and alleviating risk for laboratories. The test supports the goal of result standardisation across institutions by providing reproducible, high-quality results for clinical decision-making.

The fully automated cobas BKV, cobas CMV and cobas EBV Tests can run on the cobas 6800/8800 Systems simultaneously, providing absolute automation with proven performance and flexibility, leading to time savings and increased efficiency.

About BK polyomavirusBK polyomavirus (BKV) is a member of the polyomavirus family that can cause transplant-associated complications including nephropathy in kidney transplantation and hemorrhagic cystitis in hematopoietic stem cell transplantation. Infection can occur early in life, often with no symptoms. After primary infection, the virus can remain inactive throughout life, only to possibly reactivate in immunocompromised individuals, such as patients who receive solid-organ transplants. For kidney transplant patients, BKV infection is considered the most common viral complication, causing polyomavirus nephropathy (PVN) in up to 10 percent of kidney transplant recipients, and about 50 percent of PVN-affected patients will experience transplant graft failure.2 BKV is also associated with hemorrhagic cystitis after allogeneic hematopoietic stem cell transplantation.3

About the cobas 6800/8800 SystemsWhen every moment matters, the fully automated cobas 6800/8800 Systems offer the fastest time to results with the highest throughput and the longest walk-away time available among automated molecular platforms. With proven performance, absolute automation and unmatched flexibility delivering unparalleled throughput 24/7 cobas 6800/8800 Systems are designed to ensure a lab's long-term sustainability and success now, more than ever. Learn more now: http://www.cobas68008800.com

About RocheRoche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people's lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world's largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the twelfth consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit http://www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References[1] Jha V. Post-transplant infections: An ounce of prevention. Indian J Nephrol. 2010;20(4):171-178.[2] Jamboti, J. S. (2016) BK virus nephropathy in renal transplant recipients. Nephrology, 21: 647 654. doi: 10.1111/nep.12728. [3] Hirsch HH, Randhawa PS; AST Infectious Diseases Community of Practice. BK polyomavirus in solid organ transplantation-Guidelines from the American Society of Transplantation Infectious Diseases Community of Practice. Clin Transplant. 2019;33(9):e13528. doi:10.1111/ctr.13528

Media Contact:

Elizabeth BaxterRoche Molecular Solutions Media Relations[emailprotected] 925.523.8812

Mike WeistUS Media RelationsRoche Diagnostics Corporation[emailprotected]317.371.0035

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Energy drinks may damage the heart, researchers warnshould the FDA get involved? – Cardiovascular Business

Sunday, February 14th, 2021

Drinking certain energy drinks may cause significant damage to the heart, according to new findings published in Food and Chemical Toxicology.

Because the consumption of these beverages is not regulated and they are widely accessible over the counter to all age groups, the potential for adverse health effects of these products is a subject of concern and needed research, lead researcher Ivan Rusyn, MD, PhD, a professor at Texas A&M University in College Station, said in a prepared statement.

Rusyn et al. assessed a total of 17 popular energy drinks, studying their chemical profiles and looking for any associations with potential cardiac complications. Energy drinks sold by Adrenaline, Shoc, Bang Star, C4, CELSIUS, HEAT, EBOOST, Game Fuel, GURU, Kill Cliff, Kickstart, Monster Energy, Red Bull, Reign, Rockstar, RUNA, UPTIME, Venom Energy and Xyience Energy were all part of the teams analysis.

Overall, the authors found that stem cell-derived cardiomyocyteshuman heart cells grown in a laboratoryshowed signs of an increased beat rate after being exposed to some energy drinks. Also, theophylline, adenine and azelate were all ingredients the team associated with potentially contributing to QT prolongation in cardiomyocytes.

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FDA Approves G1 Therapeutics’ COSELA (trilaciclib): The First and Only Myeloprotection Therapy to Decrease the Incidence of Chemotherapy-Induced…

Sunday, February 14th, 2021

- COSELA is the only FDA-approved therapy that helps proactively deliver multilineage myeloprotection to patients with extensive-stage small cell lung cancer being treated with chemotherapy -

- Myeloprotective efficacy of COSELA resulted in reductions in the incidence and duration of severe neutropenia, and impacted anemia and the need for rescue interventions such as growth factors and red blood cell transfusions -

- G1 will host conference call Tuesday, February 16, 2021 at 8:00 a.m. ET -

RESEARCH TRIANGLE PARK, N.C., Feb. 12, 2021 (GLOBE NEWSWIRE) -- G1 Therapeutics Inc. (Nasdaq: GTHX), a commercial-stage oncology company, today announced that the U.S. Food and Drug Administration (FDA) has approved COSELA(trilaciclib) for injection to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer (ES-SCLC). It is the first and only therapy designed to help protect bone marrow (myeloprotection) when administered prior to treatment with chemotherapy. COSELA is expected to be commercially available through G1s specialty distributor partner network in early March.

The approval of trilaciclib (COSELA) is an important advance in the treatment of patients with extensive-stage small cell lung cancer receiving chemotherapy, said Dr. Jeffrey Crawford, Geller Professor for Research in Cancer in the Department of Medicine and Duke Cancer Institute. The most serious and life-threatening side effect of chemotherapy is myelosuppression, or damage to the bone marrow, resulting in reduced white blood cells, red blood cells and platelets. Chemotherapy-induced myelosuppression may lead to increased risks of infection, severe anemia, and/or bleeding. These complications impact patients quality of life and may also result in chemotherapy dose reductions and delays.To date, approaches have included the use of growth factor agents to accelerate blood cell recovery after the bone marrow injury has occurred, along with antibiotics and transfusions as needed. By contrast, trilaciclib provides the first proactive approach to myelosuppression through a unique mechanism of action that helps protect the bone marrow from damage by chemotherapy.In clinical trials, the addition of trilaciclib to extensive-stage small cell lung cancer chemotherapy treatment regimens reduced myelosuppression and improved clinical outcomes.The good news is that these benefits of trilaciclib will now be available for our patients in clinical practice.

Chemotherapy is an effective and important weapon against cancer. However, chemotherapy does not differentiate between healthy cells and cancer cells. It kills both, including important hematopoietic stem and progenitor cells (HSPCs) in the bone marrow that produce white blood cells (immune cells that help fight infection), red blood cells (cells that carry oxygen from the lungs to the tissues), and platelets (cells that prevent bleeding from cancer, surgeries, chronic diseases, and injuries). This chemotherapy-induced bone marrow damage, known as myelosuppression, can lead to increased risk of infection, anemia, thrombocytopenia, and other complications. Myeloprotection is a novel approach of protecting HSPCs in the bone marrow from chemotherapy-induced damage. This approach can help reduce some chemotherapy-related toxicity, making chemotherapy safer and more tolerable, while also reducing the need for reactive rescue interventions.

Chemotherapy is the most effective and widely used approach to treating people diagnosed with extensive-stage small cell lung cancer; however, standard of care chemotherapy regimens are highly myelosuppressive and can lead to costly hospitalizations and rescue interventions, said Jack Bailey, Chief Executive Officer at G1 Therapeutics. COSELA will help change the chemotherapy experience for people who are battling ES-SCLC. G1 is proud to deliver COSELA to patients and their families as the first and only therapy to help protect against chemotherapy-induced myelosuppression.

COSELA is administered intravenously as a 30-minute infusion within four hours prior to the start of chemotherapy and is the first FDA-approved therapy that helps provide proactive, multilineage protection from chemotherapy-induced myelosuppression. The approval of COSELA is based on data from three randomized, placebo-controlled trials that showed patients receiving COSELA prior to the start of chemotherapy had clinically meaningful and statistically significant reduction in the duration and severity of neutropenia. Data also showed a positive impact on red blood cell transfusions and other myeloprotective measures. The trials evaluated COSELA in combination with carboplatin/etoposide (+/- the immunotherapy atezolizumab) and topotecan chemotherapy regimens. Approximately 90% of all patients with ES-SCLC will receive at least one of these regimens during the course of their treatment.

The majority of adverse reactions reported with COSELA were mild to moderate in severity.The most common adverse reactions (10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia. Serious adverse reactions occurred in 30% of patients receiving COSELA. Serious adverse reactions reported in >3% of patients who received COSELA included respiratory failure, hemorrhage, and thrombosis. Grade 3/4 hematological adverse reactions occurring in patients treated with COSELA and placebo included neutropenia (32% and 69%), febrile neutropenia (3% and 9%), anemia (16% and 34%), thrombocytopenia (18% and 33%), and leukopenia (4% and 17%), respectively.

Quite often, people diagnosed with extensive-stage small cell lung cancerrely on chemotherapy to not only extend their lives, but also to acutely alleviate their symptoms, said Bonnie J. Addario, lung cancer survivor, co-founder and board chair of the Go2 Foundation for Lung Cancer. Unfortunately, the vast majority will experience chemotherapy-induced side effects, resulting in dose delays and reductions, and increased utilization of healthcare services. G1 shares our organizations goal to improve the quality of life of those diagnosed with lung cancer and to transform survivorship among people living with this insidious disease. We are thrilled to see new advancements that can help improve the lives of those living with small cell lung cancer.

Approximately 30,000 small cell lung cancer patients are treated in the United States annually. G1 is committed to helping patients with extensive-stage small cell lung cancer in the U.S. gain access to treatment with COSELA. For more information on access and affordability programs, patients and providers should call the G1toOne support center at 833-G1toONE (833-418-6663) from 8:00 a.m. to 8:00 p.m. Eastern time.

G1 received Breakthrough Therapy Designation from the FDA in 2019 based on positive data in small cell lung cancer patients from three randomized Phase 2 clinical trials. As is common with breakthrough-designated products that receive priority review, G1 will conduct certain post-marketing activities, including in vitro drug-drug interaction and metabolism studies, and a clinical trial to assess impact of trilaciclib on disease progression or survival in patients with ES-SCLC with chemotherapy-induced myelosuppression treated with a platinum/etoposide-containing or topotecan-containing regimen with at least a two year follow up. G1 intends to initiate the post-approval clinical trial in 2022.

Webcast and Conference Call The management team will host a webcast and conference call at 8:00 a.m. ET on Tuesday, February 16, 2021 to discuss the FDA approval of COSELA (trilaciclib). The live call may be accessed by dialing 866-763-6020 (domestic) or (210) 874-7713 (international) and entering the conference code: 6195528. A live and archived webcast will be available on theEvents & Presentationspage of the companys website: http://www.g1therapeutics.com. The webcast will be archived on the same page for 90 days following the event.

COSELA (trilaciclib) Co-Promotion Agreement with Boehringer Ingelheim

InJune 2020, G1 announced a three-year co-promotion agreement withBoehringer Ingelheimfor COSELA in small cell lung cancer in theU.S.andPuerto Rico. G1 will lead marketing, market access and medical engagement initiatives for COSELA. The Boehringer Ingelheim oncology commercial team, well-established in lung cancer, will lead sales force engagement initiatives.G1 will book revenue and retain development and commercialization rights to COSELA and payBoehringer Ingelheima promotional fee based on net sales. The three-year agreement does not extend to additional indications that G1 is evaluating for trilaciclib. Press release details of the G1/Boehringer Ingelheimagreement can be foundhere.

About Small CellLung Cancer

In the United States, approximately 30,000 small cell lung cancer patients are treated annually. SCLC, one of the two main types of lung cancer, accounts for about 10% to 15% of all lung cancers. SCLC is an aggressive disease and tends to grow and spread faster than NSCLC. It is usually asymptomatic; once symptoms do appear, it often indicates that the cancer has spread to other parts of the body. About 70% of people with SCLC will have cancer that has metastasized at the time they are diagnosed. The severity of symptoms usually increases with increased cancer growth and spread. From the time of diagnosis, the general 5-year survival rate for people with SCLC is 6%. The five-year survival rates for limited-stage (the cancer is confined to one side of the chest) SCLC is 12% to 15%, and for extensive stage (cancer has spread to the other lung and beyond), survival rates are less than 2%. Chemotherapy is the most common treatment for ES-SCLC.

COSELA(trilaciclib) for InjectionINDICATIONCOSELA is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer (ES-SCLC).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION

WARNINGS AND PRECAUTIONS

Injection-Site Reactions, Including Phlebitis and Thrombophlebitis

Acute Drug Hypersensitivity Reactions

Interstitial Lung Disease/Pneumonitis

Embryo-Fetal Toxicity

ADVERSE REACTIONS

DRUG INTERACTIONS

To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Please see full Prescribing Information here

For more information about COSELA, please call 1-800-790-G1TX (1-800-790-4189)

About G1 TherapeuticsG1 Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on the discovery, development and delivery of next generation therapies that improve the lives of those affected by cancer, including the Companys first commercially available product COSELA (trilaciclib), a first-in-class therapy approved by the U.S. Food and Drug Administration to help protect against chemotherapy-induced myelosuppression in patients with extensive-stage small cell lung cancer being treated with chemotherapy. Trilaciclib is also being evaluated in other solid tumors, including colorectal, breast and bladder cancers. G1 Therapeutics is based in Research Triangle Park, N.C. For additional information, please visit http://www.g1therapeutics.com and follow us on Twitter @G1Therapeutics.

Tecentriq (atezolizumab) is a registered trademark of Genentech.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements in this press release include, but are not limited to, those relating to the therapeutic potential of COSELA (trilaciclib), and COSELAs (trilaciclib) possibility to improve patient outcomes, are based on the companys expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Factors that may cause the companys actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in the companys filings with theU.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein and include, but are not limited to, the companys ability to complete clinical trials for, obtain approvals for and commercialize any of its product candidates; the companys initial success in ongoing clinical trials may not be indicative of results obtained when these trials are completed or in later stage trials; the inherent uncertainties associated with developing new products or technologies and operating as a development-stage company; and market conditions. Except as required by law, the company assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

Contacts:Will RobertsG1 Therapeutics, Inc.Vice President, Investor Relations and Corporate Communications(919) 907-1944wroberts@g1therapeutics.com

Christine RogersG1 Therapeutics, Inc.Associate Director, Corporate Communications(984) 365-2819crogers@g1therapeutics.com

A PDF accompanying this announcement is available athttp://ml.globenewswire.com/Resource/Download/fb9c3593-c36f-4769-9c66-c1ca2e1f78f7

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/25e03769-0cd1-482e-9a70-8b3c81bc46c3

COSELA (trilaciclib) image

COSELA (trilaciclib) image

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Easter Ross mum of blood cancer tot urges would-be stem cell donors to show the love this Valentine’s Day; Alness lass Adeline Davidson’s plight…

Sunday, February 14th, 2021

Adeline Davidson and dad Jordan.Picture: Callum Mackay

THE Easter Ross mum of a little girl with an extremely rare form of blood cancer has urged life-saving donors to step up to help others.

Steph Davidson made the appeal as blood cancer charity DKMS asked people to show the love this Valentine's Day by signing up for a worldwide register of potential blood stem cell donors.

Her daughter Adeline (3) is awaiting a blood stem cell donation from a stranger and has endured many "false starts" and complications as a result of the coronavirus crisis.

Ms Davidson, who lives in Alness, is backing the DKMS campaign at a time when UK-wide registrations have slumped by 28 per cent.

Big-hearted Highlanders have bucked that trend with an increase in registrations during the pandemic.

With no match within her family, a blood stem cell donation from a complete stranger is Adelines best chance of survival.

The brave little girl, who has missed out on her first year at nursery and valiantly gone through painful and invasive treatment over the last year, has finally got a date for her lifesaving transplant.

With one donor pulling out at the last minute, and her transplant date pushed back several times due to the pandemic, her family have their heart set on finally having their healthy and happy little girl back home and able to play with her friends and family.

Her mum said: Shes such a sweet and friendly little girl, so confident and the best big sister to her little brother and sister. Its just felt like the world has been against us this past year with so many treatments, needles and false starts.

"We are so incredibly grateful to this stranger, who could be anywhere in the world. I want to give them the biggest hug in the world. I cant begin to imagine how awful it is for other families who have a loved one in need of a lifesaving transplant where no match has been found.

"As a parent it makes you feel so powerless being unable to protect your child. Anyone who is healthy and able to register, please, please do. Its such a small commitment for you and could give someone a second chance at life Adelines not even had a chance to start hers and we were so close to it being taken away from her.

The Valentines Day campaign by DKMS is asking people to celebrate with their loved one by taking five minutes to sign up to the stem cell donor register to potentially save the love of someone elses life.

Every 20 minutes, someone in the UK is diagnosed with blood cancer. Around 2000 people each year are dealt the shocking news that they need a blood stem cell transplant.

For these people the perfect match doesnt necessarily have a compatible Zodiac sign or share the same taste in films they need to have a genetically similar make up to give them the best shot at a second chance of life.

With two in three of those people not finding a perfect match within their family, they must turn to the worldwide donor registry and rely on a stranger to save their lives. By signing up to the register you could one day be a match for someone who needs you to help save their life.

The pandemic has had a destructive impact on the lives of people with blood cancer. Not only has it led to a huge drop in the number of people registering as donors, it has meant fewer people are visiting the GP with cancer symptoms, and resulted in hospital appointments and treatments being postponed or cancelled. Due to this, DKMS expects to see a surge in blood cancer diagnoses and increased demand for blood stem cell donors when we are back to normal, making it all the more important that people register now.

Jonathan Pearce, chief executive of DKMS UK, said: At DKMS, we are dedicated to the fight against blood cancer and are proud to have registered over 780,000 blood stem cell donors. Hearing the stories of people like Adeline shows why people registering as blood stem cell donors is so important.

"With the shocking drop in registrations over the last 10 months we are calling on Scots to save the love of someone elses life this Valentines Day. We want every worried family to get the reassuring call that a match for their loved on has been found. If youre inspired by Adelines story, please register as a stem cell donor to give the ultimate gift this Valentines Day by saving a life.

How to sign up

Signing up to save the life of someone like Adeline is easy to do. Register as a potential lifesaver online at dkms.org.uk to receive your home swab kit. It takes a few moments to swab. When you return your swab kit you go on standby to help save someones life.

Taking the first steps to register as a potential blood stem cell donor can be done within a few minutes from the comfort of your own home. If you are aged between 17-55 and in general good health you can sign up for a home swab kit online. Your swabs can then be returned with the enclosed pre-paid envelope to DKMS in order to ensure that your details are added to the UKs aligned stem cell registry.

Related: Alness parents make heartfelt plea after coronavirus-related donor blow

Waiting game as stem cell donor found in United States

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Global Induced Pluripotent Market Positive Outlook, Revenue Generation & Leading Manufacturers, Forecast 2026||CELGENE CORPORATION; Astellas…

Sunday, February 14th, 2021

A consistent statistical surveying report like this Induced Pluripotent report stretches out your reach to the achievement in your business. All the information and measurement remembered for the report is supported up by notable investigation devices which incorporate SWOT examination and Porters Five Forces investigation. Statistical surveying contemplates did in this report are chivalrous which help organizations to take better choices and create predominant methodologies about creation, advertising, deals and advancement. Market definition, market division, key improvements in the market, serious investigation and examination approach are the significant section of this Induced Pluripotent market report which are again explained accurately and explicitly.

Induced Pluripotent statistical surveying report has been formed with most up-to-date insight and examination to give greatest advantages to the healthcare business. The Induced Pluripotent market report features the worldwide key makers to characterize, depict and break down the market rivalry scene through SWOT investigation. A variety of goals of the showcasing research has been considered to produce this best statistical surveying report. The market information introduced in the report assists with perceiving diverse market openings present globally. Serious investigation acted in this Induced Pluripotent report makes you mindful about the moves of the vital participants in the market, for example, new item dispatches, extensions, arrangements, joint ventures, associations, and acquisitions.

Few of the major competitors currently working in the induced pluripotent market areBristol-Myers Squibb Company; CELGENE CORPORATION; Astellas Pharma Inc.; Thermo Fisher Scientific; Cell Applications, Inc.; Axol Bioscience Ltd.; Organogenesis Holdings; Merck KGaA; FUJIFILM Holdings Corporation; Fate Therapeutics; KCI Licensing, Inc.; Japan Tissue Engineering Co., Ltd.; Vericel; ViaCyte, Inc.; STEMCELL Technologies Inc.; Horizon Discovery Group plc; Lonza; Takara Bio Inc.; Promega Corporation and QIAGEN.

Objective of the Report

Market Drivers

Market Restraints

Get Full Table Of content @ https://www.databridgemarketresearch.com/toc/?dbmr=global-induced-pluripotent-market

Key Developments in the Market:

Segmentation: Global Induced Pluripotent Market

By Product Category

By Cell Type

By Application

By End-User

By Geography

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Data bridge is an aftermath of sheer wisdom and experience which was formulated and framed in the year 2015 in Pune. We ponder into the heterogeneous markets in accord with our clients needs and scoop out the best possible solutions and detailed information about the market trends. Data Bridge delve into the markets across Asia, North America, South America, Africa to name few.

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After Bone Marrow Donation Saves 9-Year-Old Boy With Cancer, Boston Mom Fights To Raise Awareness – Here And Now

Sunday, February 7th, 2021

Every year, about 10,000 people in the U.S. need a stem cell transplant but cant find a donor.

The intense medical procedure, which can help those with leukemia, lymphoma, sickle cell anemia and other blood diseases, can save lives but securing a donor can be like finding a needle in a haystack.

Be The Match is a nonprofit, national registry where people can sign up to donate their stem cells. More than 35 million people around the world have volunteered yet only a small percentage of those donors are Americans, and even the registry admits most Americans dont know it exists.

The mother of a 12-year-old boy with leukemia has set out to change that.

Mandy Goldman is a hairdresser who lives with her husband and four children outside Boston. She remembers the devastating day five years ago when doctors told her the chemotherapy they gave her son Mateo Goldman, 9 years old at the time, didnt work.

They told us that our only option of curing Mateo was a bone marrow transplant, she says, a risky procedure that often involves a host of complications. But they had no other choice, she says.

The family got to work on the monumental task finding Mateo Goldman a close enough match.

Linda Matchan first reported the Goldman familys experience for The Boston Globe. In her research, she found very, very few people had any awareness of the need for bone marrow and stem cells donors. The awareness campaign around the subject is severely lacking compared to other campaigns like the importance of donating blood, she says.

For example, there's a little boy right now in North Carolina named Thor Forte, who's 10 and has sickle cell disease. And he has been waiting for literally half his life, five years, for a donor to be available, Matchan says. He's a tough match, but they finally did find somebody. And then when the time came for the procedure, the person backed out. So two years later, the boy is still waiting.

Fortunately, quickly after finding out Mateo Goldman didnt match with anyone in his family, he was paired with a donor on the registry from Germany. Mandy Goldman says Laura Stterlin of Frankfurt was ready to go and donate, ultimately saving her son.

Mateo Goldman wrote Stterlin, whose name he did not know at the time, a thank you note reading: Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family, he says.

And unlike usual Make-A-Wish requests, Mateo Goldman asked to meet Stterlin in person halfway across the world. The trip to Germany was planned for summer of 2020 but has since been canceled due to the pandemic.

In 2019 when she was reporting this story, Matchan had a trip planned to Germany. She ended up meeting Stterlin and hearing the story of how she became a donor. Stterlin said she was at a sporting event with her husband when she got hungry and went on the hunt for some grub.

Dear Donor, thank you for giving me the bone marrow. You feel like youre already part of my family.

Germany has a robust public service campaign to get citizens to donate bone marrow, Matchan says. So it came to no surprise to Stterlin when she came across a kiosk to sign up.

Just three months later, she got a call and an email from the registry saying that there is somebody in the United States for whom she could be a match and was asked if she would donate, Matchan says. A couple of days later, she went into the hospital and did the donation.

Stterlins stem cells then crossed the Atlantic Ocean, making their way to America during a snowstorm.

The cells started working in Mateo Goldman right away but not without some difficulties, Mandy Goldman says. He battled total body stiffness from graft-versus-host disease, a complication of the transplant.

But, you know, Matteo's an amazing kid, she says, so through it all, he was smiling and making the best of it, even though he was suffering for a lot of the time.

Two years later, in July of 2020, the cancer came back. But since Mateo Goldmans first transplant, the science had evolved greatly.

So much so that his older brother, Leo Goldman, became a candidate to donate his cells for the second stem cell transplant.

I didn't realize how I could get my brother's cells, Mateo Goldman, now 12 years old, says. Once that sank in, I felt that it would connect me and my brother more.

Right before Christmas last year, the family got extraordinary news: Mateo Goldman had zero cancer in his bone marrow, Mandy Goldman says.

Now the mom of four is on a mission to raise awareness on stem cell donations and share the story of how it saved her sons life.

The amazing feeling Leo got from being able to be the person who saved his brother's life is something he's going to carry with him forever, she says. And even Laura [Stterlin], she gave him three and a half years of his life that we get to spend with him. I just really want to educate people about how empowering it is to do something so incredible for somebody else.

When she started talking to others to raise awareness, she was shocked to discover how fearful people were in committing to be a donor.

If people could see the trauma these patients go through her son had a drain placed in his stomach, total body radiation, chemotherapy that left him head-to-toe in a skin-burning rash she says then maybe they wouldnt be scared to dedicate a small action for someone whose only cure is through a stem cell transplant.

Once people are educated about how much of a difference it makes, she says, then I feel like they would do it.

Click here to learn more about the Be The Match Registry.

Tinku Rayproduced and edited this interview for broadcast.Serena McMahonadapted it for the web.

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After Bone Marrow Donation Saves 9-Year-Old Boy With Cancer, Boston Mom Fights To Raise Awareness - Here And Now

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Leukemia in children: Symptoms, causes, treatment, outlook, and more – Medical News Today

Sunday, February 7th, 2021

Leukemia is a type of cancer that affects the blood. The two most common types in children are acute lymphoblastic leukemia and acute myelogenous leukemia.

In a person with leukemia, blood cells are released into the bloodstream before they are fully formed, so there are fewer healthy blood cells in the body.

Below, we describe the types of childhood leukemia, the symptoms, and the treatments. We then look at when to contact a doctor, what questions to ask, and where to find support.

Childhood leukemia is the most common form of cancer in children. It affects up to 3,800 children under the age of 15 in the United States each year.

Leukemia occurs when bone marrow releases new blood cells into the bloodstream before they are fully mature.

These immature blood cells do not function as they should, and eventually, the number of immature cells overtakes the number of healthy ones.

Leukemia can affect red and white blood cells and platelets.

The bone marrow produces stem cells. A blood stem cell can become a myeloid stem cell or a lymphoid stem cell.

Lymphoid stem cells become white blood cells. Myeloid stem cells can become:

Leukemia is typically acute or chronic, and chronic types are rare in children. They can include chronic myeloid leukemia or chronic lymphocytic leukemia.

Most childhood leukemias are acute, meaning that they progress quickly and need treatment as soon as possible.

Acute lymphoblastic leukemia (ALL) is the most common type in children, accounting for 75% of childhood leukemia cases.

It affects cells called lymphocytes, a type of white blood cell.

In a person with ALL, the bone marrow releases a large number of underdeveloped white blood cells called blast cells. As the number of these increases, the number of red blood cells and platelets decreases.

There are two subtypes of ALL: B-cell and T-cell.

In most childhood cases of ALL, the cancer develops in the early forms of B-cells. The other type, T-cell ALL, typically affects older children.

Research from 2020 reports that the majority of people diagnosed with ALL are under 18 and typically between 2 and 10 years old.

The American Cancer Society report that children under 5 years old have the highest risk of developing ALL and that this risk slowly declines until a person reaches their mid-20s.

The outlook for ALL depends on the subtype, the persons age, and factors specific to each person.

Myeloid leukemias account for approximately 20% of childhood leukemia cases, and most myeloid leukemias are acute.

Acute myelogenous leukemia (AML) affects white blood cells other than the lymphocytes. It may also affect red blood cells and platelets.

AML can begin in:

Juvenile myelomonocytic leukemia (JMML) accounts for approximately 12% of leukemia cases in children.

This rare type is neither acute nor chronic. JMML begins in the myeloid cells, and it typically affects children younger than 2 years.

Symptoms can include:

The symptoms of leukemia may be nonspecific similar to those of other common childhood illnesses.

A doctor will ask how long the child has been experiencing the symptoms, which can include:

Children may experience specific symptoms depending on the type of blood cell that the leukemia is affecting.

A low number of red blood cells can cause:

A low number of healthy white blood cells can cause infections or a fever with no other sign of an infection.

A low platelet count can cause:

Various factors can increase a childs risk of leukemia, and most are not preventable.

The following genetic conditions can increase the risk of leukemia:

Also, having a sibling with leukemia may increase the risk of developing it.

These can include exposure to:

If a child has symptoms that might indicate leukemia, a doctor may perform or request:

A bone marrow aspiration involves using a syringe to take a liquid sample of bone marrow cells. The doctor may give the child a drug that allows them to sleep through this test.

During the diagnostic process, a person might ask:

The doctor may recommend a variety of treatments for childhood leukemia, and the best option depends on a range of factors specific to each person.

The treatment usually consists of two phases. The first aims to kill the leukemia cells in the childs bone marrow, and the second aims to prevent the cancer from coming back.

The child may need:

Before or during treatment, a person might ask the doctor:

Questions to ask after the treatment might include:

Children who have undergone leukemia treatments require follow-up care, as the treatments often cause late effects.

These can develop in anyone who has received treatment for cancer, and they may not arise for months or years after the treatment has ended.

Treatments that can cause late effects include:

These complications may affect:

The late effects that may come can also depend on the type of treatment and the form of leukemia.

Because many leukemia symptoms can also indicate other issues, it can be hard to know when to contact a doctor.

Overall, it is best to seek medical advice if a child shows symptoms or behaviors that are not normal for them.

If a child has received a leukemia diagnosis, the effects can extend to parents, other family members, caregivers, and friends.

A person can find support and additional resources from:

The following organizations based in the United Kingdom also provide support and guidance:

Childhood leukemia can affect mental health, as well as physical health.

Learn more about mental health resources here.

According to the American Cancer Society, most children with leukemia have no known risk factors. There is no way to prevent leukemia from developing.

Because there are very few lifestyle-related or environmental causes of childhood leukemia, it is very unlikely that a caregiver can do anything to help prevent the disease.

A childs outlook depends on the type of leukemia. It is important to keep in mind that current estimates do not take into account recent advances in technology and medicine.

For example, the most recent 5-year survival rate estimates reflect the experiences of children who received their diagnoses and treatments more than 5 years ago.

The American Cancer Society report that the 5-year survival rate for children with ALL is 90%. The same rate for children with AML is 6570%.

Childhood leukemia is typically acute, which means that it develops quickly. As a result, a person should contact a doctor if they notice any of the symptoms.

The most common type of childhood leukemia is ALL, representing 3 out of 4 leukemia cases in children.

Treatment may include a combination of chemotherapy, targeted drugs, immunotherapy, stem cell transplants, surgery, and radiation.

The prognosis depends on the type of leukemia and the childs age.

This diagnosis can affect mental as well as physical health, and the effects can extend to caregivers, family members, and friends. Many different resources are available for support.

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Leukemia in children: Symptoms, causes, treatment, outlook, and more - Medical News Today

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Understanding bone marrow transplant: The guidelines and the protocols – The New Indian Express

Thursday, February 4th, 2021

The outbreak of the Covidpandemic has made many patients reluctantto undergotreatments. While their apprehension seems to overpower them, doctors need to ensure thatstrict guidelines and protocols which assure the best quality service are followed.

Among elective surgeries andtransplants, bone marrow transplant cases have increased substantially in the past few months. Adhering to guidelines for pre-transplant evaluation and the management of a common complication, graft versus host disease (GVHD)is essential.

With the diversity of practice and expertise, the following guidelines will provide a pivotal tool for learning about the rapidly updated therapy landscape in Hematopoietic stem cell transplantation (HSCT).

The guidelines intended to provide a systematic approach for transplantation and help streamline clinical practices and educate new generations of physicians-in-training. Additionally, guidelines can help to evaluate a potential transplant recipient anddetermine if the patient is an eligible candidate for the procedure.

Types and selection of transplantation:

Selection of the type of transplantation for a patient depends on factors such as the type of malignancy, availability of a suitable donor, age of the recipient, the ability to collect a tumor-free autograft, the stage, the malignancy's susceptibility to the GVM effect, and status of disease -- bone marrow involvement, the bulk of disease, chemosensitivity to conventional chemotherapy. This method is particularly applicable for Autologous or Allogeneic Transplantation where one can have a sibling donor or a matched unrelated donor. In the case of a matched unrelated donor, ensure that the collection is adequate and stem cells are available well in time especially if they are imported from countries in Europe.

A haploidentical transplant is another type of transplant that uses healthy, blood-forming cells from a half-matched donor to replace the unhealthy ones. The ideal donor in this case is a family member.

That said, for bone marrow transplant blood products are the backbone and it is important to ensure to have adequate supply before you begin with the transplant.

What are the guidelines and protocols that can be adopted in current times?

Some measures for consideration are: Minimize face-to-face visits including monitoring and consider shifting to telehealth where feasible. Some adaptive community measures like the hospital in the home services, community practices for blood collection, imaging, and support services. For radiation oncology treatment, consider reducing fractions when supported by evidence Consider alternative and less resource-intensive treatment regimes. Minimize unnecessary visitors to cancer centers, for instance, limiting to only patients and their essential caregivers based on frailty and language needs Screen for possible symptoms of COVID-19 and triage patients for admission. If necessary, the admission has to be directed to oncology/hematology departments rather than emergency departments. Immunocompromised patients are likely to have atypical presentations of COVID-19 For suspected checkpoint inhibitor-related pneumonitis prioritizes COVID-19 testing for an early decision regarding corticosteroid therapy.

These are some guidelines that you should heed during a bone marrow transplant. While it is imperative to be updated about the guidelines, timely intervention can reduce the other possible complications during the process.

(The author is the Director, Medical Oncology and Hemato Oncology, atFortis Cancer Institute, Bangalore)

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Why Cynata is hopeful its COVID treatment trial will succeed where others have failed – Business News Australia

Thursday, February 4th, 2021

Cynata Therapeutics (ASX: CYP), founded by two clever stem cell researchers and one wise Australian techpreneur, is in the process of developing a treatment for COVID-19.

Using its in-house stem cell technology Cymerus, the ASX-listed biotech hopes to treat one of the deadliest complications of COVID-19 -acute respiratory distress syndrome (ARDS).

In doing so Cynata would achieve what competitor Mesoblast (ASX: MSB) couldn't with FDA approval.

By deploying an industrialised approach to stem cell therapeutics, Cynata CEO Ross Macdonald (pictured) is confident the clinical trial process won't leave the company hamstrung.

In 1981 scientists discovered a way to derive embryonic stem cells from early mouse embryos.

The discovery thrilled scientists, and eventually led to the development of a method to do the same in lab-grown human embryos by 1998.

While there have been plenty of discussions surrounding the ethics of using of embryonic stem cells, these major scientific movements have pushed researchers to discover new and inventive ways of treating a whole raft of diseases and infections.

One such researcher, Dr Ian Dixon, saw potential for the use of mesenschymal stem cells (MSCs) - a type of stem cell that can differentiate into a variety of cell types enabling the treatment of many diseases and infections.

However there was still an obstacle to overcome: how do you mass produce enough cells needed to commercialise a treatment?

Luckily, two researchers at the University of Wisconson, Professor Igor Slukvin and Dr Maksym Vodyanik, had invented a biotechnological breakthrough called Cymerus.

The technology was able to do exactly what Dixon needed: the consistent manufacture of MSCs on an ultra-large scale; basically what Henry Ford did to the industrialisation of the auto industry, but for stem cells.

So in 2003 Dixon partnered with the two researchers to start Cynata - now an ASX-listed biotechnology company trialing a number of different treatments for a wide variety of ailments.

Most recently, Cynata's focus has been on developing a treatment for a complication of COVID-19 called acute respiratory distress syndrome (ARDS).

The complication ravages COVID-19 infected patients, destroying their organs through what is known as a cytokine storm. The complication is estimated to kill up to half of COVID-19 patients that suffer from it.

Melbourne-based Cynata is currently in the very early stages of its investigation into whether its MSCs will be able to treat the coronavirus complication overwhelming hospitals globally.

If this all sounds familiar, you might be thinking of another ASX-listed biotech called Mesoblast (ASX: MSB).

In March last year Mesoblast, also based in Melbourne, saw its shares surge after announcing plans to evaluate its stem cell treatment solutions on COVID-19 patients.

The group commenced the arduous clinical trial process to see if its remestemcel-L therapy could treat ARDS by using bone marrow aspirate from healthy donors - a similar approach the company had already taken to treat a condition many suffer from after receiving bone marrow transplants.

Mesoblast was riding high on the ASX following positive announcements surrounding the clinical traila process, especially back in April 2020 when a trial at New York City's Mt Sinai hospital found its remestemcel-L therpay achieved "remarkable" results.

Serious attention gathered around Mesoblast, with the company even securing $138 in funds from investors to continue its important research.

The company went so far as to sign a commercialisation deal for the COVID-19 treatment with Novartis, and the US Food and Drugs Administration (FDA) fast tracked the approvals process for the potential game-changing treatment.

However, in December 2020, Mesoblast hit a stumbling block.

Mesoblast's COVID-19 treatment flunked the test - its remestemcel-L therapy failed to show a lower mortality rate for patients in the prescribed 30-day timeframe of treatment.

At that point Cynata had commenced research into its own ARDS treatment. But did Mesoblast's failure unnerve Cynata CEO Ross Macdonald? Not a chance.

"I'm more confident that our trial will be successful where theirs was a failure," Macdonald said.

"If you use a process like we have developed - we don't rely on multiple different [stem cell] donations. You start with exactly the same material every time."

To explain, Macdonald used the analogy of a local caf; you normally expect a coffee from one caf to taste more or less exactly the same every time you go there - the same beans are used every time.

Whereas Macdonald said Mesoblast's process is like going to the same caf every day, but each visit they use different beans from a different supplier which leads to inconsistency in taste and flavour.

Cynata's approach with its MSCs is in line with the first example - what you get the first time from them will be replicated in each and every dose of the drug - while MSB's is like the latter.

"Yes, you still got the coffee, but the experience of the taste is totally different than it was yesterday," he said.

"The FDA said to Mesoblast, well you've got a manufacturing problem that is reliant upon multiple donors prepared to donate bone marrow and that is flawed.

"So with that in mind it's perhaps not surprising that they had a pretty disappointing result in the clinical trials."

Additionally, Macdonald said the initial investor reactions to MSB's early COVID-19 trail results were overblown.

"The initial data from their trial that got everybody excited was, in my view, quite flawed, because they said "look at how many patients are dying in intensive care units with COVID compared the patients that we treated," he said.

"But the reality of the situation was quite different. The control group at that time - the death rate was way, way higher than you would typically see for ARDS, whether its COVID or anything else. And it was simply because of the chaos that existed in intensive care units in New York in the first wave.

"So we think that the initial enthusiasm was perhaps a little misguided."

When asked why Mesoblast is receiving so much attention compared to Cynata, especially considering the above, Macdonald said it was simply because MSB is bigger and has been around for longer. For context, MSB has a market capitalisation of $1.46 billion, whereas Cynata's is just $94.56 million.

"I'd love to know why there is less attention, and how we can get our market cap above a billion dollars," joked Macdonald.

"I think the answer though is that they've been around for a lot longer than we have, they have spent a hell of a lot more money than we've spent - their monthly spend is more than we've spent for pretty much our entire existence.

"But I think the fundamental reason why is that data drives value in biotech, so the more clinical data you generate that shows your product works, the more attention you attract from investors."

That's not to say Cynata is being totally ignored in favour of the larger Mesoblast.

The company secured a $15 million placement led by $10 million from healthcare investor BioScience Managers in December.

The funds will be used to expand Cynata's clinical development pipeline and scale their operations in Australia.

As such, the company is preparing to expand its clinical development pipeline to include idiopathic pulmonary fibrosis, renal transplantation, and diabetic foot ulcers.

"So we're starting to garner that attention now that says two things - one, cell therapies are definitely a medical revolution and two, Cynata is part of that new generation of companies," Macdonald said.

As for the company's pipeline, in addition to the COVID treatment trials, Cynata is planning on launching three new clinical candidates that will get under way this year.

There's also Cynata's osteoarthritis trial, which Macdonald describes as significant for the biotech company; with 2 million patients in Australia and 30 million in the United States the company is hoping to tap into an $11 billion plus addressable market.

"It will ultimately show whether MSCs are useful in that particularly devastating condition," he said.

"It doesn't just affect people who want to go and play golf or tennis, it affects, particularly manual labourers who can no longer work.

"So the cost to the economy of osteoarthritis is quite significant, which is of course one of the reasons why the Australian Government is funding this trial."

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Business News Australia

Continue reading here:
Why Cynata is hopeful its COVID treatment trial will succeed where others have failed - Business News Australia

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