Clinical trial of a prion disease drug candidate begins enrolling participants
broadinstitute.org
broadinstitute.org
https://www.wired.com/story/sleep-no-more-crusade-genetic-ki...
It's been a while, so I'm glad to see this promising update about their research :)
There was a youtube recently I think on the same trials https://youtu.be/wuXip1SCJGs
I remember reading about Sonia Vallabh (and their race against time) - I'm so glad they're getting somewhere. Kudos.
-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...
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.
> If it makes the jump to humans, were screwed.
The known cases involve "cannibalism", like feeding sheep with ill dead sheep. It probably requires a lot of prions to cause a the disease. Don't try it at home, but a few would cause no symptoms, unless someone eats our brain and they produce more prions and someone east their brain and after a few rounds of cannibalism the concentration of prions is high and cause problems.
The solution is to avoid tight loops, i.e. use the dead sheep scrapes to feed other animals or as fertilizer. Bacteria and bad luck destroy the prions, so after a while they disappear unless you have tight loops that amplify their number.
Also, I think the bodies of the sheep/cow with symptoms are burned or destroyed, because when the concentration of prions is high enough to cause symptoms they can be bad enough for other species.
There have been researchers who have been exposed and it and developed it pretty quickly. Canabalism isn't the only way, such as with wild deer.
"unless someone eats our brain and they produce more prions and someone east their brain and after a few rounds of cannibalism the concentration of prions is high and cause problems."
That's not really how it works. The prions will continue to replicate without the need for multiple rounds of cannibalism. There is no known safe limit of prions.
The US gov claimed, you guessed it, commerce clause.
https://www.animallaw.info/case/creekstone-farms-premium-bee...
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.
If this works, this will be a game changer.
Looking at the list of observed changes in mice this can be anything from destroying the sleep cycle to general memory problems.
> Looking at the list of observed changes in mice this can be anything from destroying the sleep cycle to general memory problems.
Prion diseases guarantee certain death right now though, so the side effects would need to be pretty bad for them to be worse than the disease. Even just delaying death by a few years would be pretty good, since most people die in under a year [0].
(This is somewhat similar to chemotherapy drugs, which also tend to have pretty bad side effects but are still generally accepted as better than the alternative.)
[0]: https://en.wikipedia.org/wiki/Creutzfeldt%E2%80%93Jakob_dise...
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.