Clinical Trial Drugs/Treatments

KevinM

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I just finished my virtual appointment with Dr. Pulley at UF Health, who I like quite a bit because he is so down to earth and willing to spend as much time as I want answering questions. He is helping to oversee a new trial with metformin, an oral medication that was originally used for diabetes but is also being used for Alzheimer’s and FTD, to see how it works with ALS. It is a 2:1 trial, in that 2/3 of the participants will receive the oral medication and 1/3 the placebo.

I asked him if any of the other treatments currently in Phase 2/3 trials stood out, and he said, no, in his review of every bit of research, none looked particularly more promising than another. In particular, he does not feel that stem cell therapy, be it Nurown or others, holds the high promise many think it does.

I know this throws cold water on the hopes of so many, but it does echo some of Laurie’s and Nikki’s comments about tempering expectations. We’ve all seen or heard about the videos of several patients appearing to improve dramatically after being convinced they received Nurown (instead of the placebo), but I hope we understand that the “breakthrough” everyone so desperately wants may not be right around the corner. Maybe he’s dead wrong, but he does know his stuff.

In the meantime, let’s just try to live for the day and find happiness wherever we can.
 

Nikki J

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I believe the metformin trial is c9 specific. That is how it is listed on clinical trials dot gov and , as it was explained to me, has a mechanism that was explored preclinically especially for c9. That said, metformin is being looked at for neurodegeneration / aging in general.

I expect, as you are not FALS, Dr Pulley was not commenting on sod1 antisense which is considered very hopeful in the treatment/ mitigation of sod1 and, in a longer time frame, I think will be looked at for prevention or at least delay of onset

re Nurown, it is too soon to know how it will turn out but I was told recently by someone working on the trial that results may be in by the end of the year. they were hopeful of course but until all the data are analyzed we don’t know.

we are still waiting to hear what will happen with amylyx which seems to show some promise.

At least at mgh Covid didn’t pause either the gene trials or Nurown but did pause observational studies, travel to meetings and make most visits virtual. This freed up some time for data analysis and planning which might help the treatment trials

I don’t think it is realistic for current PALS to count on anything but if you are early stage, slow progressing I wouldn’t abandon hope of something coming along to mitigate things. I would try very hard to avoid injury and not lose weight at a minimum. Riluzole and maybe radicava if you can tolerate. Nuedexta if you are bulbar.

keep an eye on trials.
 

KevinM

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Well said, Nikki. I will try to stay positive.

It’s interesting that Dr. Pulley didn’t mention the C9 mutation when describing this to me. I, too, saw that on clinicaltrials.gov, but forgot to confirm that with him. It sounded to me like they were looking for any participant that was clinically probable or definite.

Maintaining weight continues to be an issue with me, though I’m sort of holding my own. The loss of muscle mass because I’m LMN dominant makes it difficult. I’m doing my best, though, as are we all.
 

Nikki J

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Perhaps they are expanding the metformin trial but I know the chief investigator has a special interest in c9 and,as I said, the preclinical work was there. However, retigabine was originally planned for FALS and expanded before the trial started and the other biogen trial ( not aso) was originally supposed to be c9 but became all ALS when it was posted.

there is nothing to stop anyone asking for an off label metformin prescription
 

KimT

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Did he say what dosage was being used in the trial?
 
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