Which of the new treatments for ALS do you think is most effective?

[Sequoia]

Hello folks. I am considering applying for compassionate early access to these new drugs:
Dex, NP001, and CK....I need your opinion on which one seems to have the most effective slowing down of progression. If you are on any of these three, let me know about how it is working. Thanks pals and cals~8)

 

[Alyoop]

There is no way anyone can answer this question until the phase 3 trials are completed and the placebo cohorts are compared with the treatment cohorts. Sorry but any advice will be complete hearsay. That's why the gold standard studies have the placebo arms. It's all to do with the comparison between cohorts and the statisticians looking at the data and determining whether the results are statistically significant.

If you are looking at the different studies and which you might like to try, I suggest you look at the phase 2 results and the adverse event information. You might find those on a website somewhere, but they are probably hard to find as the data would be in neurological publications that you may not be able to access. Maybe if you call your neurologist, you might find that you could get help finding the information?

 

[Sequoia]

Thanks Alyoop....I will ask my neuro. But I would like to hear from folks who are on these 3 drugs and how they feel it is helping them or not.
I did hear from person an almost immediately strengthening after they had had the infusion of CK.

 

[ccurths]

I didn't know that was an option. Tell me how do you do this?

 

[trfogey]

Sequoia,

If you are registered at P-tients L-ke Me, there are FRS and FVC statistics and written evaluations (in most cases) for a couple of dozen patients each in the NP001 trial and the dexi trial, plus a dozen or so people that are doing the oral sodium chlorite experiment. Send me a PM and I'll email you links to the searches that will bring up those patient records.

In the times I've looked into those PLM patient records for NP001 and dexi, for the ones that were fairly sure (because they had known side effects after their dosings) that they were getting the drug, the FRS scores bumped up a point or two while they were in the trial, leveled off, then went back to their historical rate of decline pretty soon after they got their last dose. Of course, some of those same folks were proven slow progressors, especially for the NP001 trial's late enrollees, so sorting out the drug effect, the placebo effect, and a PALS feeling less depressed when answering the ALSFRS questions was going to be a challenge anyway.

 

[edjohnson]

From the detailed analysis Persevering completed I would say NP001 first, Dex second. Cytokinetics still pending, but some positive anecdotal comments.

 

[Sequoia]

Thanks ed, that's specific.....!