Astellas' Bocidelpar - drug to restore mitochondrial function

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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.
 
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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.
 
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