Astellas' Bocidelpar - drug to restore mitochondrial function

Seems like Bocidelpar trial may have been canceled—this needs confirmation but bad news if true.

https://twitter.com/user/status/1598082141589590028

 

Merged thread
Making this its own thread because this is big news. And a big disappointment.


This was the $8 million dollar trial sponsored by The Japanese company Astellas.

Here is a note I got in response to an email I sent to OMF—they in turn forwarded me a short note from Systrom.

“From David Systrom: "Stopped after an interim review of half the needed patients (21) and 2 primary outcomes, a questionnaire and VO2pk….unblinding and review of all data for 32 patients underway"

The speculation is that it ended because it didn’t appear to be successful. But this speculation—don’t have a firm answer on this.

This is disappointing because this was a legitimate potential drug with a trial supervised by a good researcher.

 

https://twitter.com/user/status/1598784349662416896

 

Also not enrolling any more patients for Muscular Dystrophy trial for the same drug.

https://www.parentprojectmd.org/wp-...Community-Newsletter-to-DMD-community-003.pdf

 

Maybe some side-effects popped up and they couldn't justify moving forward. It's use in me/cfs seemed hugely speculative, I'm not sure what the expectation are/were.

 

If the true reason it stopped is lack of effectiveness, not because the drug company just gave up, at least there's some closure here.

 

Whatever the specific reasons behind this cessation, I find these 'suck it and see' trials based on in vitro chemistry and small and mostly speculative in vivo evidence annoying distractions. This isn't 1798 and we don't need to be stuffing the equivalent of cow pox pustules into the, albeit informed, modern equivalent of unsuspecting cowhands in the hope that an idea will pan out.

 

Cort Johnson write up here


Systrom at the CDC: The ME/CFS Mitochondrial Drug Trial Fails Plus More

https://www.healthrising.org/blog/2022/12/07/systrom-chronic-fatigue-syndrome-mitochondria-drug/