Clinical trial of a prion disease drug candidate begins enrolling participants

luu 29 points 4 comments October 09, 2026
www.broadinstitute.org · View on Hacker News

Discussion Highlights (3 comments)

chasil

Prion diseases include: -in sheep, scrapie -in humans, Kuru and Creutzfeldt-Jacob -in cows, BSE ("mad cow") -in deer, Chronic Wasting Disease (CWD) -in cats, feline spongiform encephalopathy Scrapie is particularly known, deadly, and long-lived. The toxin proteins can survive for a decade in soil, and infect a new host years after it emerged. https://en.wikipedia.org/wiki/Transmissible_spongiform_encep...

lbourdages

Prion diseases are one of the scarier things I've ever heard about. It is transmissible via fluids, but it can also just happen spontaneously in your body, and once that happens, it's basically a death sentence. There is so far still no cure. If this works, this will be a game changer.

rf15

It sadly sounds like it targets RNA for the production of all prion proteins (a somewhat important protein for normal brain function, but not essential) and doesn't do anything to clean up existing misfolded proteins. Even if successful, it will probably have side effects. Looking at the list of observed changes in mice this can be anything from destroying the sleep cycle to general memory problems.

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