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    • #18753

      Hi All,

      Once again, another clinical trial failure.  This time it’s Arimoclomol.

      On Friday 5/7/2021 the Orphazyme company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review.

      Another failed trial for Orphazyme’s ‘pipeline-in-a-product’ leaves shadow on drug’s future


    • #18770

      This is very sad. And it comes after the Nurown debacle.
      And as sometimes said by ALS scientists (Rothstein for instance said it 15 years ago, it was also suggested by Braak and colleagues), failing such drug trial means that the scientists do not understand what is ALS, what causes it and how to remedy to the condition.

      In Alzheimer disease they tried more than 2500 failed clinical trials, and last year there was some reckoning moment that the main hypothesis (beta amyloids as cause) was *maybe* wrong. But most scientists still work on that hypothesis as it means being able to make a career, as nobody will fund them if they work on any other hypothesis such as for example an Herpes virus infection. There is an excellent STATNEWS article describing the situation.

      For now in ALS we are just at 700 failed clinical trials, so this reckoning for ALS is probably still far away in the future.

      The only proverbial silver lining that stays in ALS is with Biogen therapies that target subsets of ALS patients (main mutation in SOD1, and dipeptide repeats in C9orf72).

      In the mid time, every few days the medias will inundate us with articles about ” an ALS breakthrough”.

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