Drug Discovery Industry Roundup with Barry Bunin — June 9, 2021
“F.D.A. Approves Alzheimer’s Drug Despite Fierce Debate Over Whether It Works” That’s how The New York Times headlined its story about the FDA’s accelerated approval of Biogen’s drug aducanumab, which will go by the brand name Aduhelm. The New York Times, notes “Patient advocacy groups had lobbied vigorously for approval.” The article reads, in part: “Recognizing that clinical trials of the drug had provided incomplete evidence to demonstrate effectiveness, the F.D.A. granted approval for the drug to be used but required Biogen to conduct a new clinical trial. If the new trial, called a Phase 4 trial, fails to show the drug is effective, the F.D.A. can—but is not required to—rescind its approval.” The article quotes Dr. Lon Schneider, Director of the California Alzheimer’s Disease Center at the University of Southern California, who helped conduct one of the aducanumab trials, as saying “There’s so little evidence for effectiveness. I don’t know what caught the F.D.A.’s fancy here.” Meanwhile FIERCE Biotech carries a piece headlined “FDA's 'Intellectually Insulting' Aducanumab Decision Opens Up a Regulatory Foothold for Leading Competitors.” And Endpoints News carries an article titled “Just How Sadly Misguided is the FDA on Aducanumab?” The world needs effective treatments against this devastating disease, so it will be interesting to see how the Phase 4 trial goes. And the ramifications, if and when it is completed.
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Are Naked Mole Rats the Fountain of Youth? Drug Discovery News carries a fascinating story: “Focus Feature: Researchers Collect Critters to Unlock Cancer Secrets.” The article begins: “Classical model systems such as cell culture and mice are key to understanding cancer. But what can we learn by examining how cancer works in other models? Animals like naked mole rats and Tasmanian devils offer a new perspective on how cancer works and how we can treat it.” Here’s something from the story to capture the interest of those of us interested in discovering new pharmaceutical agents: “Naked mole rats live longer than any other rodents. They can live nearly thirty-five years—a mouse the same size only lives four. While mice are more susceptible to cancer than humans, naked mole rats have an incredibly low incidence of cancer, and they don’t experience age-related disease at all.” Find a way to bottle those benefits and the world will come racing to your lab door.
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“Machine Learning is Booming in Medicine. It’s Also Facing a Credibility Crisis.” That’s the intriguing headline for a recent STAT News article about the need to ensure only quality information is fed into machine learning systems. Sounds logical enough, but the article points to several studies, conducted under the mantle of machine learning, that had serious flaws because of the old problem of “garbage in, garbage out.” The article reads, in part: “Researchers sprinted to see whether artificial intelligence could unravel Covid-19’s many secrets—and for good reason. There was a shortage of tests and treatments for a skyrocketing number of patients. … Hundreds of studies flooded onto preprint servers and into medical journals claiming to demonstrate AI’s ability to perform those tasks with high accuracy. It wasn’t until many months later that a research team from the University of Cambridge in England began examining the models—more than 400 in total—and reached a much different conclusion: Every single one was fatally flawed (in their opinion). … The review found the algorithms were often trained on small, single-origin data samples with limited diversity; some even reused the same data for training and testing, a cardinal sin that can lead to misleadingly impressive performance.” It’s a good read, with plenty of best practices for machine learning. AI and ML will certainly have a growing impact on medical research. The article emphasizes that these tools must be utilized correctly.
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Wearing Your Heart on your Sleeve Has New Meaning with Digital Fabric. Pharmaphorum carries an interesting article “Digital Fabric Could be Used to Measure Health Data” about a team from MIT that has published a paper on placing tiny digital devices into fabric that can be worn as a shirt or other items of clothing (and able to withstand up to 10 trips through the wash). The Pharmaphorum article reads: “That means fabric made using the material could be deployed for applications in physiological monitoring, human-computer interfaces, and on-body machine-learning—all of which raise intriguing possibilities for digital health applications.” How small are the chips? They are actually not embedded into the cloth, but into thread, which is stitched into the cloth with a needle. MIT PhD student Gabriel Loke, one of the lead authors of the paper, is quoted as saying: “When you put it into a shirt, you can’t feel it at all. This type of fabric could give quantity and quality open-source data for extracting out new body patterns that we did not know about before.”
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Biden Seeks Big Boost for Health Research. Applied Clinical Trials carries a look at President Biden’s budget “wish list” which supports increases for public health and research. Of course, it all must work its way through Congress, but the figures are encouraging. The article notes: “While the National Institutes of Health (NIH) would see a hefty 22% rise in funding for a $50.5 billion 2022 budget, $6.5 billion of that increase would fund the new Advanced Research Projects Agency for Health (ARPA-H) within NIH, a hot-button proposal that has raised many questions. Designed as a parallel to the Defense Department’s Defense Advanced Research Projects Agency (DARPA), stakeholders already are debating if ARPA-H should reside within NIH or be independent, how it could advance “transformational” research issues for multiple diseases, and how its projects would involve private sector R&D and technology transfer. A review of the ARPA-H initiative from the Congressional Research Service summarizes these issues and options for going forward. The administration also seeks an $8.5 billion budget for the Centers for Disease Control and Prevention (CDC), a notable $1.5 billion increase (up 21%) in funding for this usually neglected agency. And the Biomedical Advanced Research and Development Authority would gain $227 million for a $823 million spending plan to build the national stockpile and help prepare for future pandemics.”
Barry A. Bunin, PhD, is the Founder & CEO of Collaborative Drug Discovery, which provides a modern approach to drug discovery research informatics trusted globally by thousands of leading researchers. CDD Vault® is a hosted biological and chemical database that securely manages your private and external data.