Commenters discussed the start of a clinical trial for a prion disease drug candidate, highlighting the personal story of Sonia Vallabh and Eric Minikel and noting that Anastasia Khvorova’s lab collaborated with them and openly shared development data and the IND filing (creata, shymaple). Several participants summarized prion biology and risks: chasil listed affected species and warned prions can persist in soil for years, literalAardvark and rf15 clarified that different diseases reflect different prion strains, and lbourdages and Gathering6678 emphasized that prions can arise spontaneously in humans and are essentially fatal. Hypfer and skrebbel used vivid metaphors - prions as inert but catastrophically self-propagating shapes or a “self-propagating backdoor” - to convey how alarming these agents feel.
Opinion split over the candidate’s approach and prospects. shymaple and others expressed optimism that an effective therapy and the unusually transparent development process could be transformational, while rf15 warned the drug appears to target RNA to lower PrP production without clearing existing misfolded protein and may produce side effects observed in mice (sleep disruption, memory issues). Some contributors, like hypfer, offered speculative philosophical takes about prions’ extreme stability, underscoring a divide between hopeful enthusiasm for an open, novel intervention and caution about incomplete mechanisms, safety, and prions’ stubborn environmental persistence.
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