Clinical trial of a prion disease drug candidate begins enrolling participants

In case anyone hasn't seen it, the story behind this couple is interesting:

https://www.cureffi.org/about/

The most scary thing about prions is that they're inert. They're not even evil or agressive. They just are.

The mere shape of them causes cascading exponential system failures. And that shape happens to be very stable and requires a lot of energy to be broken up.

Like some kind of rock that falls from the sky or materializes out of thin air, and slowly the whole village turns into identical inanimate rocks.

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...

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.

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.

What Sonia Vallabh and Eric Minikel have been working toward since her diagnosis in 2011 is truly inspiring. The drug candidate was developed by Anastasia Khvorova and her lab at UMass Chan Medical School, in collaboration with Vallabh, Minikel, and their research teams.

What makes this effort even more remarkable is their commitment to sharing the drug development process openly, including an IND filing that would typically remain confidential.

Whatever the eventual outcome( I’m optimistic) about the possibilities, the journey, the data, and the knowledge shared along the way will be valuable.

This is not just about developing a drug; it’s about making science more transparent, collaborative, and accessible for everyone working toward the same goal.