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Thread: Of Mutants and Mitochondria

  1. #1

    Of Mutants and Mitochondria

    Although inherited SOD1 mutations cause only a small minority of all ALS cases, researchers believe that changes to SOD1 play a role in many cases of ALS. “Misfolded SOD1 is relevant to sporadic cases of ALS,” Vande Velde said, especially in light of new research that found the same buildup of mutant SOD1 in the motor neurons of sporadic ALS patients.

    Researchers hope that developing pharmaceuticals to block the unnatural deposits of mutant SOD1 on mitochondria will prevent the progression of ALS. One such drug (Olexisome) is currently being used in clinical trials in Europe.

    “Having misfolded SOD1 around is really not a good thing,” Vande Velde said, especially for mitochondria. Efforts to get rid of the mutant protein, she adds, may benefit ALS patients.

    So now we SOD1 misbehaving with mitochondria (above) and astrocytes (below).

    Where does ubiquilin 2 fit in the grand scheme of things or is this a completely separate mutation...? How many mutations can there be? My brain hurts!

  2. #2
    Ok ... I'm struggling with this one ... does anyone have information on Olexisome? I can't seem to find much about it. Is it known as something else?

  3. #3
    had a quick ping around but can't find anything more than a couple of references in forums.

  4. #4
    Jacqui Priestley
    Olexosime is the trade name for TRO 19622, the Trophos trial. More information available at

  5. #5
    Jacqui Priestley
    I mean Olesoxime!

  6. #6
    Hi Jacqui,

    Thank you. I knew I had heard it before but couldn't work out where.

    Anyone heard much about this trial? There hasn't been much hype with this trial like NP001 and dex. Looks like results are expected at the end of 2011.
    Last edited by MattJ; 31st August 2011 at 15:28.

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