Drug Discovery Industry Roundup with Barry Bunin — January 19, 2023

Barry Bunin, PhD Founder & CEO Collaborative Drug Discovery

Barry Bunin, PhD
Founder & CEO
Collaborative Drug Discovery

Drug Discovery Industry Roundup with Barry Bunin

“A.I. Turns Its Artistry to Creating New Human Proteins.” That’s the headline in a recent New York Times article about scientists using OpenAI’s DALL-E software to model proteins that have never existed. The hope is that the rise of new AI technologies will shrink the time needed to generate blueprints for new proteins from years down to weeks. “What we need are new proteins that can solve modern-day problems, like cancer and viral pandemics,” David Baker, Director of the Institute for Protein Design at the University of Washington, said. “We can’t wait for evolution. … Now, we can design these proteins much faster, and with much higher success rates, and create much more sophisticated molecules that can help solve these problems.” The article notes: “After artificial intelligence technologies produce these protein blueprints, scientists must still take them into a wet lab—where experiments can be done with real chemical compounds—and make sure they do what they are supposed to do.”

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Cortical-Expansion Genes: Creating Humans with Bigger Brains? “I have to begin by saying that this story gives me the shivers at the end of it. Read on and see if it does the same for you.” That’s how Derek Lowe opened a recent blog in Science about discovery of human de novo genes that can be used to trigger cortical expansion. After researchers inserted these gene sequences into mice, the animals showed “significant cortical expansion” as they matured. To me, this article was interesting in showing the surprising effects of genes on brain size (and hinting on what’s possible in the future). One can’t help but think about Aldous Huxley’s Brave New World.  As Dr. Lowe writes: “The Science commentary linked to above features a statement from one of the paper’s authors that a paper is coming that shows that these animal do indeed perform better in tests of cognitive function and memory. They are smarter mice, with larger brains. And now the hair may be rising up on the back of your neck, as it did on mine.” He writes that very good lab hygiene will be required so animals don’t escape. “I think we can all agree that it is not in our best interest to allow the earth’s mice and rats to become any more intelligent and wily than they are already. I wish that I were joking about that, but I’m not.” The blog attracted a lot of comments—with plenty of references to science fiction novels that predicted such events. One reader wrote: “How about using ancient DNA to resurrect saber-toothed cats and then making them super-intelligent? What could go wrong?” But Lowe ends on a serious note: “As the world knows, we have already seen irresponsible attempts to do germline editing and produce altered human babies. When will someone try adding in more of the cortical-expansion genes, to see what happens? I’ll leave it there.”

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FDA approves Alzheimer Drug Leqembi for Early and Mild Stages of Disease. The U.S. Food and Drug Administration has approved the Alzheimer's drug lecanemab (marketed as Leqembi) developed by Eisai and Biogen for patients in the earliest stages of the mind-wasting disease. Leqembi was approved under the FDA's accelerated review process, an expedited pathway that speeds access to a drug based on its impact on underlying disease-related biomarkers believed to predict a clinical benefit. Reuters quotes an Alzheimer's Association statement: “Without Centers for Medicare & Medicaid Services (CMS) and insurance coverage ... access for those who could benefit from the newly-approved treatment will only be available to those who can pay out-of-pocket." Providing context, The New York Times reports: “Last year, the federal Centers for Medicare and Medicaid Services sharply limited Medicare coverage for Aduhelm…that meant very few patients could afford Aduhelm’s $28,800-a-year price tag, and the drug has effectively been sidelined from the marketplace. If the agency determines that Leqembi has clearer evidence of helping patients, Medicare could cover it for all eligible patients and only impose a requirement that the patients’ experience be tracked.”

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“Cancer Vaccine Hunt Makes Progress…” That’s the news from a recent Washington Post story on new data released by Moderna and Merck that suggests that after decades of failures, researchers are finally figuring out the right way to design a vaccine that can teach immune cells how to recognize and combat tumors. I heard the CEO of Moderna recently present on these exciting results at the JP Morgan conference (along with how they are using their $18B cash to accelerate their mRNA vaccines clinical pipeline).  This is the first time a vaccine has shown statistically meaningful impact in a combination cancer therapy, which is truly exciting.  The article reads, “Earlier this month, the companies said that when used in concert with Merck’s cancer immunotherapy Keytruda, Moderna’s mRNA cancer vaccine reduced the risk of certain skin cancers from returning or patient deaths by 44% compared with Keytruda alone. That number justly generated a lot of excitement. It’s the first time an mRNA-based cancer vaccine has proven itself in a randomized study, and with an unambiguously positive outcome.” The article ends with this optimistic note: “Let’s hope that’s the catalyst for the true cancer vaccine revolution, one that could convert cancer from a potentially lethal illness into a chronic, stable disease.”  And we can all use a ray of hope.

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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. The CDD Vault is a hosted biological and chemical database that securely manages your private and external data.