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Astellas' Bocidelpar - drug to restore mitochondrial function

Discussion in 'Drug and supplement treatments' started by John Mac, Jun 27, 2022.

  1. Jaybee00

    Jaybee00 Senior Member (Voting Rights)

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    Hutan, Louie41, bobbler and 2 others like this.
  2. Jaybee00

    Jaybee00 Senior Member (Voting Rights)

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    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.
     
    Last edited by a moderator: Dec 5, 2022
    RedFox, Hutan, sebaaa and 9 others like this.
  3. Jaybee00

    Jaybee00 Senior Member (Voting Rights)

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  4. Jaybee00

    Jaybee00 Senior Member (Voting Rights)

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    Starlight, DokaGirl, merylg and 4 others like this.
  5. dreampop

    dreampop Senior Member (Voting Rights)

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    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.
     
    DokaGirl, RedFox and Peter Trewhitt like this.
  6. Sean

    Sean Moderator Staff Member

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    Sounds like a safety issue to me.
     
    Peter Trewhitt and DokaGirl like this.
  7. RedFox

    RedFox Senior Member (Voting Rights)

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    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.
     
  8. CRG

    CRG Senior Member (Voting Rights)

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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.
     
  9. Jaybee00

    Jaybee00 Senior Member (Voting Rights)

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    Sean likes this.

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