StemCell Therapy

In this day and age, corporations are really able to do virtually anything and everything they could want. If they wish to open a new office or launch a new division, they are often able to do so. Why then, are so many companies turning to philanthropic measures? Let's take a look.

Public Pressure

Nowadays, brand loyalty can often mean nothing to customers. While you once would have picked one company and stayed with them for your entire life, this is not the case any longer. If a brand does something that is not approved of by many of their customers, there is a chance that they will leave and go to a competitor.

As a result, many corporations have turned to philanthropic pursuits. This is a great way for them to respond to the concerns of their customers while also acknowledging some of the major events and issues currently facing us worldwide. For example, many corporations are making a commitment to going carbon-neutral within their business dealings. While this will require an awful lot of work on their part, and might also involve some serious redirection within the business, it is a fantastic move that will satisfy old clients and potentially attract new ones too.

Caring for Causes

Of course, it is not just the public who have control over these philanthropic pursuits. Company directors and CEOs are also directing their corporations towards issues and areas that they personally care about.

Two great examples here are Tej Kohli and Bill Gates. Tej Kohli is the founder of the philanthropic Tej Kohli Foundation which is engaged in several missions around the world. In particular, they are committed to trying to eliminate corneal blindness, one of the most common types of blindness in the world. Bill Gates and his wife Melinda set up the Bill and Melinda Gates Foundation. They have been engaged in healthcare philanthropy to help improve the living conditions for many thousands of people all across the world.

Uplifting Others

As there continues to be a growing wealth gap between the top percentiles of our society, corporations are beginning to look into ways they can offer help to some of those lower down. This could be in the form of a scholarship to attend higher education, or it could be through the form of work experience within the company.

It could even be as simple as constructing a local community centre for everyone to use. There are many small ways that a corporation can provide better opportunities to those in poorer areas. We never know what someone can achieve when given the right tools for success.

This is a trend that is most definitely not going away. With more and more corporations turning to philanthropic efforts, we are likely to see a lot of good come out of this world in the future. Take a look at some of your favourite companies to see what they are doing, or even look at some of the paths that you could adopt as a business owner.

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Why Corporations Are Turning To Philanthropy - International Business Times UK

The National Research & Training Center on Blindness & Low Vision (NRTC) is proud to announce our receipt of another competitive national grant. This grant, Independent Living Services for Older Individuals Who Are Blind Training and Technical Assistance, is sponsored by the Rehabilitation Services Administration under the U.S. Department of Education. This 5-year grant, which provides approximately $3 million in funding, begins October 1, 2020.

Sylvia Stinson-Perez is the project director for this grant known as the OIB-TAC. She is excited to take the strong foundation from the last five years of the NRTCs work on this grant and add new and innovative projects. There are three major products related to this grant:

Michele McDonnall, NRTC Director, is happy that the funding will allow the NRTC to continue to improve the provision of services to older individuals who are blind.

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The NRTC Awarded Second Major National Grant | The National Research and Training Center on Blindness and Low Vision - Mississippi State Newsroom

Avrobio has licensed a Hunter syndrome lentiviral gene therapy from the University of Manchester in the U.K. The deal positions Avrobio to join Regenxbio and Sangamo Therapeutics in the race to develop gene therapies for use in patients with the rare disorder.

Hunter syndrome, also known as mucopolysaccharidosis Type II, is driven by a mutation that limits a patients ability to break down sugar molecules generated as a byproduct of cell activity. The buildup of the molecules causes progressive damage to organs such as the brain, affecting the development of children with the lysosomal disorder.

Takedas approved drug Elaprase gives Hunter syndrome patients the enzyme they need to break the molecules down, but its inability to cross the blood-brain barrier renders it ineffective against the CNS manifestations of the disease.

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Avrobio has become the latest company to identify gene therapy as a potentially better way to treat Hunter syndrome. The biotech, which is active in other lysosomal disorders, has agreed to pay the University of Manchester $8 million upfront for the global rights to a stem cell gene therapy that is set to enter the clinic in the second half of next year.

Brian Bigger, Ph.D., a professor of cell and gene therapy at the U.K. university, developed the candidate and co-authored a paper about it in 2018. The paper describes work on a braintargeted hematopoietic stem cell gene therapy designed to give patients the lysosomal enzyme iduronate-2-sulfatase (IDS).

Avrobio will make the therapy, now called AVR-RD-05, by modifying a patients own hematopoietic stem cells with a transgene for IDS expression and a protein tag intended to improve stability of the enzyme. If AVR-RD-05 works as hoped, the cells will engraft in the bone marrow and make copies of themselves that also carry the transgene.

The differentiation of the cells into components of the immune system could enable AVR-RD-05 to cause a lasting, body-wide increase in the presence of the enzyme at the root of Hunter syndrome. Avrobio will fund a 9.1 million ($11.8 millon) investigator-sponsored phase 1/2 trial to assess whether the gene therapy performs as hoped. The biotech is also on the hook for up to $80 million in milestones.

Avrobio will face competition if it gets AVR-RD-05 all the way to market. Takeda is the incumbent, Regenxbio and Sangamo have gene therapies in the clinicalthough the latter has underwhelmed so farand Denali Therapeutics is developing an IDS enzyme designed to cross the blood-brain barrier.

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Avrobio bags gene therapy to join Regenxbio and Sangamo in race - FierceBiotech

Professor Robert MacLaren on the exciting potential of CRISPR gene editing and patients seeing stars for the first-time following treatment

Professor Robert MacLaren, of Oxford University, is leading work to develop gene therapies for inherited retinal conditions through Nightstar Therapeutics. The company was acquired in 2019 by US biotechnology company, Biogen, for $US 877 million.

One night I was at my computer and a patient emailed me to say he could see a night star. This was a positive thing. I said, If you are still there can you go and check which eye it was. He came back and said, It is definitely the eye that I had the gene therapy in.

Subsequently, when we developed the gene therapy company, the name NightStar was an obvious one because since then several of our patients have described seeing night stars after gene therapy. It symbolizes looking up at the heavens and hope for the future, but there is also that practical element of achieving better vision at night.

Although I am an ophthalmologist treating patients, when we are looking at the mechanisms of the disease, we get right down to the molecular level, looking at how the DNA is assembled and how it is processed.

It is very exciting as a physician in any specialty to be able to treat disease at the genetic level. Ultimately if the disease occurs at the genetic level and you treat it there, you are treating the origin of the disease itself. If you are just giving drugs to try and correct the genetic disease or compensate for it, it may help but that is not a real treatment. It provides insight into the mechanisms of life itself seeing how DNA is processed and how the genes evolve.

You can treat potentially any disease with that, whereas gene replacement therapy is limited to small genes. This is still good but ideally if you correct the mutation it is a better treatment than replacing the whole gene.

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Gene therapy: It provides insight into the mechanisms of life itself - AOP

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review.

If approved, Lenti-D (elivaldogene autotemcel or eli-cel) could transform the prospects of people with CALD, the most severe form of the neurodegenerative disease ALD that usually emerges in boys during early childhood and causes physical and mental disabilities as well as behavioural problems.

Around 40% of patients develop the cerebral form of ALD, which in turn affects around one in 17,000 live births.

A few weeks ago, Bluebird reported new data from the phase 2/3 STARBEAM trial of Lenti-D which showed that 87% of CALD patients were still alive and free of major functional disabilities after at least two years follow-up.

The EU filing comes ahead of a filing for eli-cel in the US, which Bluebird says should take place sometime towards the middle of next year, having been delayed by the coronavirus pandemic.

If approved, eli-cel would provide a one-shot treatment for CALD, holding back the progressive breakdown in the protective myelin that sheathes neurons.

It would be the first alternative to a stem cell transplant to treat the disease, a therapy that can provide significant improvements and even halt progression in some patients if given early enough.

However it requires high-dose chemotherapy to destroy the bone marrow, and that poses significant risks to patients in its own right, and can also lead to graft-versus-host disease, a potentially life-threatening complication in which the bone marrow donors immune cells attack the recipients cells and tissues.

CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

ALD protein is needed to clear toxic molecules called very long-chain fatty acids (VLCFAs) in the brain, and if mutated causes the VLCFAs to accumulate and damage the myelin sheath.

Using eli-cel, the patients own stem cells are modified in the lab to produce a working version of the ABCD1 gene, producing functional ALD protein that can help to flush VLCFAs from the body.

CALD is a devastating disease, often marked by rapid neurodegeneration, the development of major functional disabilities, and eventual death, said Gary Fortin, head of severe genetic disease programmes at Bluebird.

If approved, eli-cel would represent the first therapy for CALD that uses a patients own haematopoietic stem cells, potentially mitigating the risk of life-threatening immune complications associated with transplant using cells from a donor, he added.

Aside from STARBEAM, which will follow treated patients for up to 15 years, Bluebird is also conducting the phase 3 ALD-104 trial of eli-cel in CALD, which is due to generate results in 2024.

The EU filing for eli-cel comes shortly after Bluebirds development partner received a 27 March 2021 FDA review date for anti-BCMA CAR-T cell therapy ide-cel, a potential therapy for multiple myeloma.

The biotech already has approval in Europe for Zynteglo, a gene therapy for haematological disease beta thalassaemia, and is due to file its related therapy LentiGlobin for sickle cell disease next year. The two therapies have been tipped to generate $1.5 billion-plus in peak sales by some analysts.

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EMA starts rapid review of Bluebird's gene therapy for rare disease CALD - - pharmaphorum

The FDA has lifted the clinical hold on a phase 1/2 clinical trial of Solid Biosciences gene therapy treatment for Duchenne muscular dystrophy (DMD). Solid Bio secured clearance to resume dosing in the trial after making manufacturing changes to cut the number of viral particles given to patients.

SGT-001, the adeno-associated viral (AAV) vector-mediated gene transfer therapy being tested in the phase 1/2 trial, has suffered a series of setbacks since entering the clinic, most recently when the FDA put the study on hold in response to a case of acute kidney injury. The FDA imposed the hold 11 months ago. In July, Solid Bio said the FDA wanted to see more data before lifting the hold.

The request led Solid Bio to share further information on its gene therapy manufacturing process and its latest safety and efficacy data. The additional information proved sufficient to persuade the FDA to lift the clinical hold.

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Solid Bio will resume dosing using a gene therapy made under a revised manufacturing process. The new process is intended to remove most empty viral capsids, thereby enabling Solid Bio to cut total viral load without reducing the dose. The focus on viral load reflects concerns systemic delivery of AAV vectors can damage organs and cause inflammation.

To get the FDA to lift the hold, Solid Bio shared data from a quantitative, in vitro microdystrophin expression assay designed to show the comparability of SGT-001 manufactured under the old and new processes. Solid Bio shared those results in response to the FDAs request for information in July.

The biotech is taking other precautions to manage the potential risk posed by SGT-001. Solid Bio has capped the maximum weight of the first two patients to receive SGT-001 after the hold lifts at 18 kg. As the dose of SGT-001 is determined by weight, heavier patients receive more vector genomes. The adverse events seen in some gene therapy trials, such as the deaths in Audentes Therapeutics trial, have happened in patients who were heavier and therefore received a higher viral load.

Solid Bio is further mitigating the potential for SGT-001 to cause harm by amending the protocol to include the prophylactic use of eculizumab, the anti-complement inhibitor sold by Alexion as Soliris, and C1 esterase inhibitor, while also increasing the prednisone dose in the month after treatment.

The protocol changes position Solid Bio to resume its pursuit of DMD gene therapy leader Sarepta Therapeutics, which suffered a setback of its own last month when the FDA asked it to use an extra potency assay in a planned clinical trial. Pfizer is also in the race but, like Solid Bio, has run into safety issues that could give Sarepta an edge.

Shares in Solid Bio, which had slumped to $2 apiece, rose 70% in response to the end of the hold.

Link:
FDA lifts clinical hold on Solid Bio gene therapy trial - FierceBiotech

ALDERSON, W.Va. (WVVA) A Greenbrier County teen is about to become one of the first five patients in history to receive an experimental gene therapy for Hurler's Syndrome.

The Alderson native is in many ways your typical 13-year-old. "I like to help my dad feed cows....and donkeys," said Kendra Goins.

But life hasn't always been easy for Kendra. The extremely rare condition makes it impossible for her body to breakdown certain sugars. In addition to causing damage to her organs, the condition makes it difficult for her body to grow.

But whenever anyone has anything to say about it, her sister, Kiristen is always the first time stand up.

"Me with my big head is always the one to jump in," said Goins, who said she has gotten into quite a few quarrels over the issue at school in defense of her sister.

She worries though that she won't be able to jump in when Kendra heads to California soon for a clinical trial. Kendra is heading into the treatment with her first bone marrow transplant nearing the end of its course.

"I've spoken with experts across the country from the chemists who made the drug to the doctor who has used it. This looks like her golden ticket," said Kendra's mother Sheryl Goins.

Administered through the brain, the goal of the gene therapy is to help her body produce the enzymes she needs to survive.

While the cost of the clinical trial and airfare is covered, the family said they need help with expenses they will incur during their three-month stay.

To learn more about how you can help, visit https://www.gofundme.com/f/kendra039s-gene-therapy?utm_source=facebook&utm_medium=social&utm_campaign=p_cp%20share-sheet&fbclid=IwAR3yzHCexch5a_awjaYy06ijB28zMXJ-72WkfQ-SPEwMQgy5s8x_carlz34

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Greenbrier County teen to be among first in nation to receive gene therapy for Hurler's Syndrome - WVVA TV

Oct. 5, 2020 08:09 UTC

HARLOW, England--(BUSINESS WIRE)-- Diamond Pharma Services (Diamond), a leading technical services and regulatory affairs consulting group, has announced that it provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics (GenSight), including the preparation, authoring support, agency communication and submission of GenSights first Marketing Authorisation Application to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ. The EMA decision is expected in H2 2021.

LUMEVOQ (Lenadogene nolparvovec) is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. LHON mainly affects young males, and the ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There is a high unmet medical need for LHON patients, of which there are 800-1200 in the EU and the US annually.

Headquartered in Paris, France, GenSight is a biopharma company focused on developing and commercialising innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Diamond has provided GenSight with regulatory, pharmacovigilance, quality and compliance support for LUMEVOQ leading up to the MAA assessment.

Maureen Graham, Managing Director, Regulatory, Diamond Pharma Services, said: We are pleased GenSight Biologics chose to work with our team of experts at Diamond to provide European Regulatory, Pharmacovigilance, Quality and Compliance support for LUMEVOQ, its first MAA submission, and the first for a gene therapy treating a mitochondrial disease. It has been a personal ambition of mine to have the opportunity to work on a gene therapy within the ophthalmic arena, and GenSight has allowed Diamond that opportunity and that privilege.

This submission adds to Diamonds broad experience in providing support to companies developing advanced therapy medicinal products (ATMPs), which includes over 50 programmes at various stages of development, and two MAA approvals - Glybera and Yescarta.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201005005339/en/

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Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ -...

The Food and Drug Administration (FDA) has granted Fast Track designation to the investigational gene therapy candidate, PF-06939926 (Pfizer), for the treatment of Duchenne muscular dystrophy (DMD).

PF-06939926 is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of human dystrophin gene under the control of a human muscle-specific promoter. The Company has chosen the rAAV9 capsid due to its potential to target muscle tissue.

The designation was based on data from an ongoing phase 1b study evaluating the safety and tolerability of a single intravenous infusion of PF-06939926 in 9 ambulatory boys with DMD aged 6 to 12 years. Preliminary results showed that PF-06939926 was well tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The Company plans to launch a double-blind, placebo-controlled phase 3 study to evaluate the efficacy and safety of PF-06939926 in boys with DMD. The study will include patients who are at least 4 years old and less than 8 years old; all participants will need to be on a daily dose of glucocorticoids for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. The primary outcome of the study (change from baseline in North Star Ambulatory Assessment) will be assessed at 52 weeks; patients will be followed for 5 years after treatment.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. We are working to advance our planned phase 3 program as quickly as possible.

The FDAs Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.

For more information visit pfizer.com.

Pfizer receives FDA Fast Track designation for Duchenne muscular dystrophy investigational gene therapy. https://www.businesswire.com/news/home/20201001005382/en/Pfizer-Receives-FDA-Fast-Track-Designation-for-Duchenne-Muscular-Dystrophy-Investigational-Gene-Therapy. Accessed October 2, 2020.

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Investigational Gene Therapy Fast Tracked for Duchenne Muscular Dystrophy - Monthly Prescribing Reference

NEW YORK, Oct. 05, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today announced that the Company will present at upcoming conferences and will announce six-month safety and efficacy data from the second cohort of its AXO-Lenti-PD program the morning of Tuesday, October 6, 2020. Additionally, as previously announced the Company will hold a Parkinsons Disease R&D Day on Friday, October 30.

Information on the upcoming presentations can be found below:

Chardan 4th Annual Genetic Medicines Conference

2020 ARM Virtual Cell and Gene Meeting on the Mesa

AXO-Lenti-PD Parkinsons Disease R&D Day

A live webcast of the presentations will be available in the Events section of Axovant's website at http://www.axovant.com. Replays will be available for approximately 30 days following the conferences.

About Axovant Gene Therapies

Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit http://www.axovant.com.

Contacts:

Media & Investors

Parag MeswaniAxovant Gene Therapies Ltd.(212) 547-2523media@axovant.cominvestors@axovant.com

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Axovant to Participate in Upcoming October Conferences and Provide AXO-Lenti-PD Program Update the Morning of October 6th - BioSpace

Global Cancer Gene Therapy Market: Overview

Cancer could be defined as uncontrolled cell growth in the body leading to organ malfunction. If untreated, it can lead to death. Uncontrolled growth of cell is managed by the body in several ways, one of them is by deploying white blood cells to detect and eradicate these cancerous cells. It has been discovered that the immune system could be manipulated to influence cancerous cells to destroy itself.

Read Report Overview https://www.transparencymarketresearch.com/cancer-gene-therapy-market.html

Radiation and chemotherapy therapy have consistent and reliable effects to decrease cancerous cells in the body. Recently, immunotherapy for hematological cancers has experienced a recognition and is of interest for many researchers Scientists have developed methods to isolate, replicate, and develop cancer-destroying cells from the patients blood cancer and injecting those cells back for the destruction of their cancers, with durable remissions.

New options for the treatment is needed to be developed if order to achieve elimination of cancer suffering and death by 2020. According to NCI, 5-year survival rate for cancers such as lung (15%), glioblastoma (5%), pancreatic (4%), and liver (7%) remains very low. Current available treatments have several side effects, the systemic toxicity due to chemotherapy results in nausea, mild cognitive impairments, and mouth ulcerations, in addition to long-term side effects such as increasing risk of developing other types of cancers. Therefore, new and innovative treatment methods are required to reduce the suffering of cancer patients.

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Global Cancer Gene Therapy Market: Drivers and Restraints

The emerging field of cancer Gene Therapy offers varied potential treatments. Gene therapy involves a range of treatment types, which use genetic material to alter cells (either in vivo or in vitro) to help cure the disease. Cancer Gene Therapy shown efficacy in various in vitro and preclinical testing. Preclinical testing for cancer gene therapy has been performed on glioma, pancreatic cancer, liver cancer, and many other cancers.

Increase in prevalence of cancer, rise in government funding and initiatives, growth in pipeline of cancer gene therapy products, and collaborations to develop and launch gene-therapy products are some factors driving the market. According to NCBI researchers, development of genetically-modified T-cell therapies for treatment of cancer has had maximum clinical impact among other gene therapies. However, high treatment cost is a major limitation in the cancer gene therapy market. The reason behind the huge cost for cancer gene therapy is the necessity of rigorous, exhaustive clinical trials; also treatment by cancer gene therapy differs from person to person depending upon the genetic acceptance of every patient, unlike other drugs thereby limiting the market growth.

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Global Cancer Gene Therapy Market: Key Segments

Based on type, the cancer gene therapy market is segmented into gene transfer immunotherapy and oncolytic virotherapy. Immunotherapy uses genetically modified cells and viral particles to stimulate the immune system to destroy cancer cells. Immunotherapy include treatment with either cytokine gene delivery or tumor antigen gene delivery. Oncolytic virotherapy uses viral particles, which replicate within the cancer cell causing the death of the cell. It is an emerging treatment modality that is expected to shows great promise, particularly in metastatic cancer treatment. It includes treatment with adenovirus, retrovirus, lentivirus, herpes simplex virus, adeno-associated virus, simian virus, alphavirus, and vaccinia virus. Gene transfer is the newest treatment modality that is expected to introduce new modified genes into cancerous cell or associated tissue for destruction of cell or to slow down cancer growth. This technique is flexible as a wide variety of vectors and genes are used for clinical trials with positive outcomes. As gene therapy advance, they could be used alone or in combination with other treatments to control the disease. Gene transfer or gene replacement is performed using naked/plasmid vectors, electroporation, sonoporation, magnetofection, and gene gun.

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Based on region, the global cancer gene therapy market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa. North America is anticipated to hold the largest market share. The U.S. dominates the cancer gene therapy market owing to its increase in funding for research & development and other government initiatives. Key players in the biotech industry are engaging in research & development of gene therapy products. Moreover, rising demand for DNA vaccines and growing interest of venture capitalists to investment in commercialization of gene-based cancer therapies are likely to propel the market. The cancer gene therapy market in Asia Pacific is anticipated to expand at a rapid pace as in China cancer gene therapy is anticipated to attribute for largest revenue, due to the recent launch of Gendicine and rising healthcare expenditure with strong R&D facilities.

Global Cancer Gene Therapy Market: Key Players

Key players operating in the global cancer gene therapy market are Adaptimmune, ZioPharm Oncology Altor Bioscience, MolMed, bluebird bio, Shanghai Sunway Biotech company limited , MultiVir, Shenzhen SiBiono GeneTech, Corporation.

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Cancer Gene Therapy Market is Expected to Expand at an Impressive Rate by 2025 - The Daily Chronicle

CAMBRIDGE, Mass., Oct. 05, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), an innovative genetic medicines company creating a new class of non-viral gene therapy, announced today that Phillip Samayoa, vice president of strategy and portfolio development, will present a company overview at the annual Cell & Gene Meeting on the Mesa. The presentation will be available for registered participants to view on demand throughout the conference, to be held virtually Oct. 12-16, at meetingonthemesa.com.

Geoff McDonough, M.D., Generation Bios president and chief executive officer, will participate in a panel on the future of gene delivery. That discussion will be available to view on demand on the conference website starting Oct. 13.

About Generation Bio

Generation Biois an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.For more information, please visitgenerationbio.com.

Contact:

InvestorsChelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com910-777-3049

MediaStephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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Generation Bio to Present at 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

Researchers at the Massachusetts Institute of Technologys (MIT) research enterprise Critical Analytics for Manufacturing Personalized Medicine (CAMP) have been awarded Intra-CREATE grants from the National Research Foundation (NRF).The grants will support research on retinal biometrics for glaucoma progression and neural cell implantation therapy for spinal cord injuries.

Krystyn Van Vliet, co-lead Principal Investigator at Singapore-MIT Alliance for Research and Technology (SMART) CAMP, and Professor of Materials Science and Engineering at MIT, said: Singapores well-established biopharmaceutical ecosystem brings with it a thriving research ecosystem that is supported by skilled talents and strong manufacturing capabilities. We are excited to collaborate with our partners in Singapore, bringing together an interdisciplinary group of experts from MIT and Singapore, for new research areas at SMART.

In addition to our existing research on our three flagship projects, we hope to develop breakthroughs in manufacturing other cell therapy platforms that will enable better medical treatments and outcomes for society.

Hosted by SMART CAMP, the first research project, Retinal Analytics via Machine learning aiding Physics (RAMP), brings together an interdisciplinary group of ophthalmologists, data scientists, and optical scientists from SMART, Singapore Eye Research Institute (SERI), Agency for Science, Technology and Research (A*STAR), Duke-NUS Medical School, Massachusetts Institute of Technology (MIT), and National University of Singapore (NUS). The team will seek to establish first principles-founded, and statistically confident models of, glaucoma progression in patients, which will enable rapid and reliable forecast of the rate and trajectory of glaucoma progression, leading to better targeted treatments.

MIT Mechanical Engineering Professor Barbastathis, co-leader of the research and Principal Investigator at SMART CAMP said: We look forward to leveraging the ideas fostered in SMART CAMP to build data analytics and optical imaging capabilities for this pressing medical challenge of glaucoma prediction.

The second research project, Engineering Scaffold-Mediated Neural Cell Therapy for Spinal Cord Injury Treatment (ScaNCellS), gathers an interdisciplinary group of engineers, cell biologists, and clinician scientists from SMART, Nanyang Technological University (NTU), NUS, IMCB A*STAR, A*STAR, French National Centre for Scientific Research (CNRS), University of Cambridge, and MIT.

The team will seek to design a combined scaffold and neural cell implantation therapy for spinal cord injury treatment that is safe, efficacious, and reproducible, paving the way forward for similar neural cell therapies for other neurological disorders. The project aims to achieve its goals through an enhanced biological understanding of the regeneration process of nerve tissue and optimised engineering methods to prepare cells and biomaterials for treatment.

Chew Sing Yian, Principal Investigator at SMART CAMP and Associate Professor of the School of Chemical and Biomedical Engineering, and Lee Kong Chian School of Medicine, at NTU said: Our earlier SMART and NTU scientific collaborations on progenitor cells in the central nervous system are now being extended to cell therapy translation. This helps us address SCI in a new way, and connect to the methods of quality analysis for cells developed in SMART CAMP.

Cell therapy, one of the fastest-growing areas of research, will provide patients with access to more options that will prevent and treat illnesses, some of which are currently incurable. Glaucoma and spinal cord injuries affect many, said Hanry Yu, co-lead Principal Investigator at SMART CAMP.

Our research will seek to plug current gaps and deliver valuable impact to cell therapy research and medical treatments for both conditions. With a good foundation to work on, we will be able to pave the way for future exciting research for further breakthroughs that will benefit the healthcare industry and society.

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New support for personalised medicine and cell therapy - Health Europa

Addressing gaps in testing and education within precision oncology can assist in improving access to patients of color and other underserved populations.

Addressing gaps in testing and education within precision oncology can assist in improving access to patients of color and other underserved populations, said Karen Winkfield, MD, PhD, incoming executive director, Meharry-Vanderbilt Alliance.

Transcript

AJMC: In the era of precision medicine, what can oncologists do to ensure that newer therapies are the right choice for minority patients?

Dr Winkfield: Precision oncology has really come a long way, and I do think that oftentimes people think about it as just genetic testing or genomic testing. That's important, and we do know that there's a gap, even in terms of testing, for some things as simple as an oncotype for breast cancer patients. We know that Black patients are not getting the oncotype test at the same rate as other racial and ethnic groups.

So, really, just making sure that there's access is one thing that can be important. Whether it be partnering with some of the testing facilities to make sure that there is opportunity for individuals who may be underinsured or uninsured to gain access to some of these precision oncology tests, that would be wonderful.

The other thing is making sure people understand, again, community engagement. I'm going to keep coming back to that, because patients need to understand the importance of it and know that they're not being a guinea pig, etc, but that we want to make sure that their treatment is personalized. The personalization is not just to them as an individual, but also to their tumor and what their tumor might be doing. So, that communication is important as well.

So, sometimes it may require a second biopsy. So, you can imagine, if a person has gone through therapy and then their tumor is not responding, and you say, "Oh, we need to get some more tissue," there can be some misunderstandings about that. So, really being open with your patients about the rationale and the reason for getting additional tissue, but, again, making sure that patients don't have very high out-of-pocket cost is one of the things that I think will go a long way to improving access to patients of color and other underserved populations to precision oncology.

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Dr Karen Winkfield on Improving Minority Access, Education for Precision Oncology - AJMC.com Managed Markets Network

OncoHost, announced on 5th October that Professor Yuval Shaked, co-founder and Chief Scientific Advisor at OncoHost, and Professor of Cell Biology and Cancer Science at the Technion Israel Institute of Technology, will deliver a presentation titled A Proteomics-Based Platform for Predicting Response to Immunotherapy and Personalizing Treatment Plans at the MAP 2020 Virtual Congress - ESMO this Friday, October 9th at 17:20 PM CEST.

The presentation will show how through the analysis of host response profiles (i.e. the patients reaction), oncologists may be able to harness this information to better predict clinical outcomes and suggest the ideal combination treatment with immunotherapy.

Despite major clinical success, immunotherapy treatments have demonstrated efficacy in only a small proportion of patients with non-small cell lung cancer (NSCLC), said Prof. Yuval Shaked. OncoHosts studies have indicated that individual host response to anti-cancer treatment can generate pro-tumorigenic activities and support tumor re-growth and spread. It is therefore vital to analyze and earlier predict host response to treatment in order to improve outcomes and reduce unnecessary side effects experienced by so many patients.

Using machine learning (ML)-based analysis and algorithms to identify highly predictive cohort-based proteomic signatures in patients, personalized response prediction can be used to improve precision medicine in oncology. Personalized treatment plans can then be created through information based on cohort-based statistical analysis, personalized adaption and finally, translation to clinically relevant targets.

Prof. Shakeds presentation will include a real-life case study on whom a Host Response profile analysis was performed, and will be followed by a live Q&A.

MAP is the leading congress in precision medicine in oncology in Europe, established by key opinion leaders in the field. The name of the Congress was recently changed into Molecular Analysis for Precision Oncology to better reflect its aims of focusing on science, translational research, education and collaboration. This years event will be held virtually on October 9-10.

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OncoHost Data on Host Immunotherapy Response to be Presented This Week - Technology Networks

BINYAMINA, Israel, Oct. 5, 2020 /PRNewswire/ -- OncoHost, a global leader in host response profiling for improved personalized cancer therapy, announced today that Professor Yuval Shaked, co-founder and Chief Scientific Advisor at OncoHost, and Professor of Cell Biology and Cancer Science at the Technion Israel Institute of Technology, will deliver a presentation titled A Proteomics-Based Platform for Predicting Response to Immunotherapy and Personalizing Treatment Plans at the MAP 2020 Virtual Congress - ESMOthis Friday, October 9th at 17:20 PM CEST.

The presentation will show how through the analysis of host response profiles (i.e. the patient's reaction), oncologists may be able to harness this information to better predict clinical outcomes and suggest the ideal combination treatment with immunotherapy.

"Despite major clinical success, immunotherapy treatments have demonstrated efficacy in only a small proportion of patients with non-small cell lung cancer (NSCLC)," said Prof. Yuval Shaked. "OncoHost's studies have indicated that individual host response to anti-cancer treatment can generate pro-tumorigenic activities and support tumor re-growth and spread. It is therefore vital to analyze and earlier predict host response to treatment in order to improve outcomes and reduce unnecessary side effects experienced by so many patients."

Using machine learning (ML)-based analysis and algorithms to identify highly predictive cohort-based proteomic signatures in patients, personalized response prediction can be used to improve precision medicine in oncology. Personalized treatment plans can then be created through information based on cohort-based statistical analysis, personalized adaption and finally, translation to clinically relevant targets.

Prof. Shaked's presentation willinclude a real-life case study on whom a Host Response profile analysis was performed, and will be followed by a live Q&A.

MAP is the leading congress in precision medicine in oncology in Europe, established by key opinion leaders in the field. The name of the Congress was recently changed into Molecular Analysis for Precision Oncology to better reflect its aims of focusing on science, translational research, education and collaboration. This year's event will be held virtually on October 9-10.

About OncoHost

OncoHost combines life-science research and advanced machine learning technology to develop personalized strategies to maximize the success of cancer therapy. Utilizing proprietary proteomic analysis, the company aims to understand patients' unique response to therapy and overcome one of the major obstacles in clinical oncology today resistance to therapy. OncoHost's Host Response Profiling platform (PROphet) analyzes proteomic changes in blood samples to monitor the dynamics of biological processes induced by the patient (i.e., the host) in response to a given cancer therapy. This proteomic profile is highly predictive of individual patient outcome, thus enabling personalized treatment planning. PROphet also identifies potential drug targets, advancing the development of novel therapeutic strategies as well as rationally based combination therapies.

For more information, visithttp://www.oncohost.com

Follow OncoHost onLinkedIn

OncoHost Media Contact: Ellie HansonFinn Partners [emailprotected]+1 929-222-8006

SOURCE OncoHost

https://oncohost.com/

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OncoHost to Present Data on Predicting Host Response to Immunotherapy at the MAP 2020 Virtual Congress - ESMO - PRNewswire

While ride-sharing services like Grab, Uber, and Gojek have become a pervasive part of life, many countries in the Asia Pacific region are still unconvinced when it comes to micro-mobilities such as bike and scooter sharing. While the convenience offered by these is great, especially in this Covid-19 era when people may remain wary of crowding in buses and metro trains, there is a need for in-depth knowledge of these new transportation options to help guide policy and regulation.

A group of scientists in the Senseable City Lab at MIT and the Future Urban Mobility (FM) Interdisciplinary Research Group at the Singapore-MIT Alliance for Research and Technology (SMART), MITs research enterprise in Singapore, set out to better understand the phenomenon and inform policy-making through a comparative analysis of bike-sharing and scooter-sharing activities in Singapore.

The researchers shared their findings in a paper titled Understanding spatio-temporal heterogeneity of bike-sharing and scooter-sharing mobility published in the journal Computers, Environment and Urban Systems. The study is based on real usage records containing location and time of departures and arrivals in two distinct areas in Singapore.

We constructed historical trajectories of the bike-sharing and scooter-sharing trips and compared usage patterns of the two systems at the Marina Bay area and the NUS campus, says Rui Zhu, a postdoc at SMART FM. Our results showed increased sharing frequency and decreased fleet size for scooter-sharing, suggesting that it performs better than bike-sharing.

More specifically, the sharing frequency was increased from less than one time per day for bike-sharing to more than three times per day for scooter-sharing, but the researchers believe that can be improved even further to create a more profitable service.

The study also found that shared scooters in Marina Bay were frequently left away from their designated parking spaces or charging stations, indicating costly and labor-intensive maintenance since employees need to collect and transport scooters between stations continuously. However, the statistics also showed that over 28 percent and 26 percent of trips departed from and arrived at non-stations respectively, suggesting that users actually utilized most of the inappropriately returned scooters.

In addition, the study revealed quantitative changes in trips over time, distances, and duration, and the influence of weather on the demand of micro-mobilities.

In Singapore and a few other cities, dockless bike-sharing systems rose and fell in just one year, followed by an explosion of docking scooter-sharing systems. But we didnt have the necessary insights for appropriate business and policy decisions, Zhu explains. Our study goes deeper into the problems and possibilities of micro-mobility sharing and suggests how these services can be improved.

To facilitate a sustainable scooter-sharing service, the researchers suggest optimizing the fleet size of stations and their locations, regulating returning behaviors more strictly, enabling scooters to have autonomous repositioning functionality, and increasing the useful battery life of scooters.

To increase battery life, they suggest installing a photovoltaic module on scooters for solar charging during trips and parking time or equipping conventional dock-based stations with grid charging or solar charging platforms, allowing for an environmentally friendly solution that will be able to reduce carbon footprints.

While the business model and user behavior greatly impact the success of mobility-sharing services, government policy also plays a significant role. Supportive policies or regulations on controlling fleet sizes and limiting usage to discrete areas are huge drivers for the sustainable development of the new transportation modes. With this study, SMARTs researchers hope to fill existing gaps in knowledge about micro-mobility sharing to help inform policy decisions.

SMART was established by MIT in partnership with the National Research Foundation of Singapore (NRF) in 2007. SMART is the first entity in the Campus for Research Excellence and Technological Enterprise (CREATE) developed by NRF. SMART serves as an intellectual and innovation hub for research interactions between MIT and Singapore, performing cutting-edge research in areas of interest to both. SMART currently comprises an Innovation Center and six Interdisciplinary Research Groups: Future Urban Mobility, Antimicrobial Resistance, BioSystems and Micromechanics, Critical Analytics for Manufacturing Personalized-Medicine, Disruptive and Sustainable Technologies for Agricultural Precision, and Low Energy Electronic Systems.

The Future Urban Mobility group harnesses new technological and institutional innovations to create the next generation of urban mobility systems to increase accessibility, equity, safety, and environmental performance for the citizens and businesses of Singapore and other metropolitan areas, worldwide.

SMART research is supported by the NRF and situated in CREATE.

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Comparing the benefits of scooter-sharing vs. bike-sharing - MIT News

Oct. 6, 2020 05:30 UTC

STRASBOURG, France & TOKYO & WALTHAM, Mass.--(BUSINESS WIRE)-- Regulatory News:

Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, NEC Corporation (NEC; TSE: 6701), a leader in IT and network technologies and BostonGene Corporation (BostonGene), a biomedical software company committed to defining optimal precision medicine-based therapies for cancer patients, today announced a strategic collaboration for two ongoing Phase 1 clinical trials of TG4050, an individualized therapeutic vaccine for ovarian and head & neck cancers based on Transgenes proprietary myvac platform and NECs AI-driven Neoantigen Prediction System in Europe and the United States.

Transgenes myvac platform brings together a series of highly innovative technologies, such as viral genome engineering, to achieve high-speed modular manufacturing of bespoke immunotherapies.

TG4050 is an individualized cancer vaccine based on the myvac platform; it is based on an optimized viral platform for cancer vaccination and integrates NECs artificial intelligence capabilities. This therapeutic vaccine aims at stimulating the immune system of patients to induce a T-cell response against tumor-specific antigenic alterations, called neoantigens. These neoantigens are derived from genomic mutations and selected using NECs Neoantigen Prediction System, an advanced AI technology that has already been applied in the field of oncology. TG4050 has been designed to target up to 30 patient-specific neoantigens. Transgene is sponsoring two Phase 1 trials that are expected to deliver a first proof of concept of this virus-based individualized approach.

As part of the collaboration, BostonGene will conduct genomic and transcriptomic analyses of primary patient tumors collected from patients enrolled in these two clinical trials to identify predictors of response to TG4050 and the cancer cell-intrinsic and -extrinsic factors that may mediate each patients response to the vaccine. BostonGenes platform integrates the genomic and transcriptomic analyses to simultaneously assess the activity of the tumor and the microenvironment through the identification of significant somatic alterations, evaluation of gene expression, estimation of tumor heterogeneity and classification of the microenvironment.

BostonGene generates a Tumor Portrait Report, involving the data-driven, visually appealing and self-explanatory tumor schematics elegantly depicting tumor activity, tumor cellular composition, and functionality of the immune-microenvironment and other tumor-associated processes. The comprehensive report will provide insights into the individual oncogenic state and immunogenicity of the patients tumor.

BostonGenes unique solution and deep expertise in Next Generation Sequencing (NGS) analysis provide us with the detailed profiles of a tumor and its micro-environment. These Tumor Portrait Reports will help us look at our patient data in light of the current published evidence and could help us accelerate the development of TG4050, said ric Qumneur, Pharm.D., Ph.D., Executive VP, Chief Scientific Officer of Transgene. This novel way of analyzing patient data is part of an ambitious translational program that supports the development of our myvac platform. By integrating these types of approaches into our studies, we seek to build an integrated framework for the use of viral-based immunotherapeutics.

NEC looks forward to strengthening its collaboration with BostonGene through these trials of TG4050. BostonGenes advanced analysis of NGS among cancer patients provides excellent profiling that we believe will add important insight into the understanding of each patients tumor environment and how it reflects on the clinical outcomes of our treatment, said Osamu Fujikawa, Senior Vice President at NEC Corporation.

BostonGene is proud to support Transgene and NEC during these critical Phase 1 clinical trials, said Nathan Fowler, MD, Chief Medical Officer at BostonGene. This collaboration represents our ongoing commitment to improve immunotherapy options and transform personalization of treatment for cancer patients.

About TG4050 TG4050 is an individualized immunotherapy being developed for solid tumors that is based on Transgenes myvac technology and powered by NECs longstanding artificial intelligence (AI) expertise. This virus-based therapeutic vaccine encodes neoantigens (patient-specific mutations) identified and selected by NECs Neoantigen Prediction System. The prediction system is based on more than two decades of expertise in AI and has been trained on proprietary data allowing it to accurately prioritize and select the most immunogenic sequences.

TG4050 is designed to stimulate the immune system of patients in order to induce a T-cell response that is able to recognize and destroy tumor cells based on their own neoantigens. This individualized immunotherapy is developed for each patient and can be produced in a very short time frame.

This best-in-class candidate is being evaluated in two Phase 1 clinical trials for patients with ovarian cancers (NCT03839524) and HPV-negative head and neck cancers (NCT04183166).

About myvac myvac is a viral vector (MVA) based, individualized immunotherapy platform that has been developed by Transgene to target solid tumors. myvac-derived products are designed to stimulate the patients immune system, recognize and destroy tumors using the patients own cancer specific genetic mutations. Transgene has set up an innovative network that combines bioengineering, digital transformation, established vectorization know-how and unique manufacturing capabilities. Transgene has been awarded Investment for the Future funding from Bpifrance for the development of its platform myvac. TG4050 is the first myvac-derived product being evaluated in clinical trials.

About NEC's Neoantigen Prediction System NEC's neoantigen prediction utilizes its proprietary artificial intelligence (AI), such as graph-based relational learning, which is combined with other sources of data to discover candidate neoantigen targets. NEC comprehensively evaluates the candidate neoantigens with a primary focus placed on its in-house major histocompatibility complex (MHC) binding affinity prediction trained on public and proprietary datasets. These allow NEC to effectively prioritize the numerous candidate neoantigens identified in a single patient.

About Transgene Transgene (Euronext: TNG) is a publicly traded French biotechnology company focused on designing and developing targeted immunotherapies for the treatment of cancer. Transgenes programs utilize viral vector technology with the goal of indirectly or directly killing cancer cells.

The Companys clinical-stage programs consist of two therapeutic vaccines (TG4001 for the treatment of HPV-positive cancers, and TG4050, the first individualized therapeutic vaccine based on the myvac platform) as well as two oncolytic viruses (TG6002 for the treatment of solid tumors, and BT-001, the first oncolytic virus based on the Invir.IO platform).

With Transgenes myvac platform, therapeutic vaccination enters the field of precision medicine with a novel immunotherapy that is fully tailored to each individual. The myvac approach allows the generation of a virus-based immunotherapy that encodes patient-specific mutations identified and selected by Artificial Intelligence capabilities provided by its partner NEC.

With its proprietary platform Invir.IO, Transgene is building on its viral vector engineering expertise to design a new generation of multifunctional oncolytic viruses. Transgene has an ongoing Invir.IO collaboration with AstraZeneca.

Additional information about Transgene is available at: http://www.transgene.fr.

Follow us on Twitter: @TransgeneSA

About NEC Corporation NEC Corporation has established itself as a leader in the integration of IT and network technologies while promoting the brand statement of Orchestrating a brighter world. NEC enables businesses and communities to adapt to rapid changes taking place in both society and the market as it provides for the social values of safety, security, fairness and efficiency to promote a more sustainable world where everyone has the chance to reach their full potential. For more information, visit NEC at https://www.nec.com.

About BostonGene Corporation BostonGene Corporation is pioneering the use of biomedical software for advanced patient analysis and personalized therapy decision making in the fight against cancer. BostonGenes unique solution performs sophisticated analytics to aid clinicians in their evaluation of viable treatment options for each patient's individual genetics, tumor and tumor microenvironment, clinical characteristics and disease profile. BostonGenes mission is to enable physicians to provide every patient with the highest probability of survival through optimal cancer treatments using advanced, personalized therapies. For more information, visit BostonGene at http://www.BostonGene.com.

Transgene disclaimer This press release contains forward-looking statements, which are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those anticipated. The occurrence of any of these risks could have a significant negative outcome for the Companys activities, perspectives, financial situation, results, regulatory authorities agreement with development phases, and development. The Companys ability to commercialize its products depends on but is not limited to the following factors: positive pre-clinical data may not be predictive of human clinical results, the success of clinical studies, the ability to obtain financing and/or partnerships for product manufacturing, development and commercialization, and marketing approval by government regulatory authorities. For a discussion of risks and uncertainties which could cause the Companys actual results, financial condition, performance or achievements to differ from those contained in the forward-looking statements, please refer to the Risk Factors (Facteurs de Risque) section of the Universal Registration Document, available on the AMF website (http://www.amf-france.org) or on Transgenes website (www.transgene.fr). Forward-looking statements speak only as of the date on which they are made and Transgene undertakes no obligation to update these forward-looking statements, even if new information becomes available in the future.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201005005838/en/

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Transgene, NEC and BostonGene Announce Strategic Collaboration for Two Ongoing Clinical Trials for Patients with Ovarian and Head & Neck Cancers -...

The treatment given to POTUS is the abstraction we all desire personalized medicine. Just the right treatment, at the right time, in the right way. Of course, physicians' day-to-day dilemma is translating guidelines and treatments tested upon populations into efficacious care for the individual sitting across from your desk. And that dilemma is compounded when you deal with VIPs

It is not March; when the virus didn't respond to the treatments that had always worked so well for influenza - we threw everything we had at the problem. While we have made significant progress in treating COVID-19, I do not believe we have a set-in-place protocol for what to do when. There is a broad outline of medications that may reduce symptoms, improve outcomes, and reduce the length of stay lots of ingredients, lots of chefs, but no classic recipe.

All of the medications that the President has been publically acknowledged as receiving reduce symptoms.

Ethical medicine, "shared decision making" between patient and physician requires that I provide you with options, state which I feel is the best approach and why, and together we choose a path forward. The President's care, as with most VIPs, involves lots of chefs. Many chefs with nuanced opinions that by consensus, majority vote, or eminence eventually become a treatment plan. [1] One of the difficulties in treating VIPs, in general, is that in many instances, they believe their eminence extends to being a chef too. They can be very "hands-on" when it comes to choosing treatments and what to pursue.

The patient applies any factors they feel are relevant in that calculus. I think it is apparent that the President has included a political component to his choices. This is not the time to appear weak physically or emotionally. His motorcade to his supporters is an example of powerful eminence in action. As a rule, hospitals don't allow you to leave for a few hours and return; in many cases, chronic smokers with significant addiction to nicotine are not allowed to go outside for a cigarette.

Of course, it takes two to make a medical decision; you still need a physician. Can we reasonably believe that the phrase "an abundance of caution" has not impacted their clinical judgment? Additionally, by my count, the President has had at least three physicians, including his private one, since taking office. The idea that he has a deep enough long-term relationship with any of them that would allow for considered choice is silly; in that way, the President echoes some of our behavior, honoring primary care in word but not deed.

Words matter and their meaningvariessignificantly from one context to another. Consider Chris Christie, who "checked himself in" to a hospital over the weekend. Hospitals are not hotels; you don't check-in; you are admitted to the hospital by an attending physician who has determined you have met the admission criteria. You only would describe admission to the hospital as checking-in to make it seem more an optional vacation choice, less a medical need.

Or the announcement that the President may go home to continue care, you shouldn't take that to mean he is like a typical COVID-19 patient being discharged from the hospital; unless, of course, that patient has a fully equipped Emergency Department and physicians standing by in their home.

It is a fool's errand to guess at the President's clinical status based on his receiving treatment. Whether he received supplemental oxygen doesn't necessarily mean he was more ill or that the doctors acted out of an abundance of caution. What is supplemental oxygen, 2 liters/min or 8, nasal prongs, or face mask? We cannot tell.

He is receiving personalized care, not care from some guidelines that haven't even been formulated. His treatment, like ours, is or should be, is personalized to his needs. It may very well be that his perception of his non-medical needs overrides his physicians' medical judgment, but that is a problem all doctors and patients face.

[1] One of the problems for VIPS is that, in some cases, "too many cooks do spoil the soup." If you don't believe me take a look at themedical decisionssurrounding President Garfield, "At least a dozen medical experts probed the president's wound, often with unsterilized metal instruments or bare hands, as was common at the time." Or look at thecare of the Shah of Iran, whose cancer was treated by the world's best cardiac surgeon, with a very poor outcome.

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Managing the President's COVID-19 - American Council on Science and Health

A new modeling tool may be able to help doctors assess which treatments are best for individual patients with colorectal cancer. The artificial intelligence program analyzes a patient's disease details -- such as the stage of cancer and other chronic conditions -- and compares those details to other colorectal cancer cases to predict the patient's chance of surviving past 10 years.

"Predicting survival of cancer patients as a means to help determine treatments is not new," said Jean Emmanuel Bibault, MD, PhD, a radiation oncologist who led the study. "But current standard techniques are not very accurate, and we're hoping that by using AI we can bring more precise information to doctors as they make crucial decisions about care."

Predicting a cancer patient's survival time lends valuable insight into the best course of treatment for both the long and short term, helping to determine what is likely most suitable.

The online tool works by assessing 32 details about an individual patient, such as age, the stage of the cancer, exercise habits, cholesterol levels, history of chronic disease and much more.

After these details are input into the tool, the algorithm predicts how long that person might live and reports a number in years. The tool also provides context, citing the top reasons for its calculation, such as the stage of the cancer, the patient's age at diagnosis, or how the patient was initially treated.

"From a physician's point of view, we want to know how well our patients are going to do from the get-go. We're looking at two main things: how to choose the right therapy, and if we can alter their destiny," said Daniel Chang, MD, professor of radiation oncology, who is an author of the study.

"Some folks have a bit of a nihilistic point of view," he continued, "that survival is determined by the genetics of your cancer and of your body. But the question is: Can anything we, as doctors, do change that outcome if we do it sooner or do it differently from the start? That's where I see a lot of value for this research."

An abstract on the research appeared online inGut.

Bibault, Chang and professor of radiation oncology, Lei Xing, PhD, devised the algorithm powering the prediction tool with data made available through the National Institutes of Health, from thousands of de-identified patients who have or had colorectal cancer at various stages and are of varying ages.

The team trained the algorithm to track survival of thousands of patients, in conjunction with the details of their disease and some details about their course of treatment. In this way, the algorithm uses the outcomes and survival rates of past cohorts to calculate the chance of survival for future patients.

So far, the tool has been about 90% accurate in predictions it made on 472 patient cases that were not used to train the tool. The tool has not been used in a clinical setting.

"The treatments that we have nowadays are becoming more and more specialized, targeted, in many cases intensified. And the reality is that not everybody is going to benefit from new treatments, therapies or technologies in the same way," said Chang.

"This algorithm could allow us a better shot at personalized medicine, and enhance our ability to tailor the treatments to be as appropriate as possible," he added.

Although patients could use the tool on their own, Bibault said the ideal application would be for doctors and patients to use the tool together. That way, doctors would be able to contextualize the result and answer any patient questions.

The team's goal is to enhance the algorithm's accuracy and to find other applications for it.

"We have laid the foundation for this model," said Bibault, "and we're hopeful it can apply to other cancer types as well."

Photo by National Cancer Institute

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AI tool created to guide colorectal cancer care with more precision - Scope