Hawker News

Clinical trial of a prion disease drug candidate begins enrolling participants

broadinstitute.org

69 pointsby luu11 comments

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

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

chasil[3 comments hidden]
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...

rf15[2 comments hidden]
They're all the same in my understanding, just named differently because they have been observed separately before any understanding about PrP existed. And yes, that means they're also all long-lived. These proteins do not decay like more useful molecules or more complex organic matter.
literalAardvark[hidden]
Some of them are the same, but some aren't. CWD is a prion separate from BSE for instance, but BSE is caused by the same prion as Creutzfeldt-Jacob.
hypfer[4 comments hidden]
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.

skrebbel[2 comments hidden]
Yeah it sounds like God hired a malicious freelance programmer who put in a self-propagating backdoor huh.
hypfer[hidden]
I suppose one could go on a wild tangent here and argue that - given they're significantly more stable - prions are the correct-er form of proteins.

They just do not get any work done within how known biological life works, but that could perhaps also be read as said whole tree of life relying on a misconfiguration of that building block?

This is wildly unqualified rambling on my part, but I wonder if life could develop that is based on that prion form of proteins.

From my understanding, no. But maybe "life developing" is the wrong metric for correctness?

FWIW, The universe itself seems to be dead-set on entering the most stable configuration of everything.

Gathering6678[hidden]
The scariest part for me is: it can just form randomly in one's body, and there's no way to tell until it's too late.
rf15[hidden]
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.

lbourdages[hidden]
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.

shymaple[hidden]
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.