Cytokinetics closing down Courage Trial

[Hal]

Reaching out to see if anyone here has been on the Cytokinetics Courage trial. I’ve been on it for @ 20 weeks (out of the planned 48 weeks) and have been pleased with the result, but am now hearing they are shutting it down. 2 questions… first has anyone been on this trial and if, what’s been your tak…. Second, has anyone been in a trial that’s been shutdown? Right now I’m pretty disappointed…. Thanks

 

[Nikki J]

I am sorry. Thank you for participating. Cold comfort but there is some research that just being in a trial even getting placebo is clinically beneficial - probably because of the extra monitoring and the relationships you build with the trial staff

I never qualified for a trial. I did have several friends who were in the failed c9 trial. That one went to open label but when the results came out not only were they told it had failed. They were told the end dose made people progress faster.

It is a bitter pill to swallow I know. Are you at mgh? They tried to get their people from the c9 trial into other trials or if they didn’t qualify expanded access

 

[lgelb]

This is the announcement. It was terminated for futility. I would certainly check clinicaltrials.gov and the NEALS trial database to see what else you may be eligible for.

Everyone who participates, placebo or treatment, does other PALS a great service.

 

[Hal]

Thanks Nikki … I was at MGH through late last summer when we moved South as my symptoms have always been significantly worse in cold weather. Have to say I am wondering how much the financials weighed in on this decision given their stock price very much appreciated Cytokinetics backing away from their ALS commitment. Given the limited treatment options we as PALS have can also be at least partially addressed via diet, I think I will be moving forward with an increased focus on holistic ways to address ALS.

 

[lgelb]

The financial motive in terminating the trial of a treatment failure is that by "killing quickly," you do save money that can be spent on more promising options, across your entire portfolio. The stock price goes up not because this was an ALS treatment trial but because no more time or money will be -- harsh though it may sound -- wasted.

This committee review was pre-specified as to timing, as with all legitimate clinical trials. This was always going to be a go/no-go milestone. The only possible decision, in FDA-regulated and all ethically-conducted trials anywhere, is to terminate at a point when you have enough data to show that treatment is not helping.

I hope that no one mistakes this trial failure (and there have been many in ALS and every other disease state) for a neon sign that diet or any particular holistic approach is THE answer, because there is a lot of quackery in altmed that can cost a lot more than money. Steve Gleason's failed stem cell therapy, resulting in his loss of bowel control, was a very public example.

Moreover, the most evidence-based approaches to ALS combine traditional and complementary elements.

 

[Hal]

i am quite comfortable with both my knowledge and approach and have had multiple discussions with some of the top experts to support my position

 

[krnNdug]

I was on a previous trial by Cytokinetics, Vitality ALS - Tirasemtiv. Out of the 744 trial participants, only 40 of us benefited the maximum dosage. My FVC went from 56(my SVC qualified me) to 92 and 93.

I didn't realize how groggy I had become. I knew when my last dose was going to be 2 years before it happened it was July 31, 2022.

I was frustrated but it also brought a bit of fear, which is very rare in my life. I didn't know how fast the progression 8 would be now that I'm off the drug. It has gone down and is in the upper 70s. More weakness and swallowing challenges.

I'm glad I got a chance to be in a trial, it didn't matter if it was the drug or placebo.

Nikki is correct, the pALS who are on the placebo increase their quality of life and it will extend life by months. The needs for equipment are caught sooner which helps before a pALS would usually be seen and prescribed something. Also, we need the placebo to see if the drug works. If not, the numbers won't be defined as well.

Please try to get in a clinical trial. My hero is Ed Clark who applied for 5 trials but only was able to participate in 4. He tried the VITALITY- ALS but couldn't tolerate the drug. But he kept enrolling for as long as he could.