NurOwn Phase 3 - Meaningful Benefit

[rmt]

My husband and I were so disappointed when NurOwn didn't pass the original Phase 3 trial. I had given up hoping it would ever be an option, but maybe there is a glimmer of hope again.

<swapped out link--mod> BrainStorm Cell Therapeutics Announces the Presentation of New Analyses from the Phase 3 Trial of NurOwn® in ALS at the 4th Annual ALS ONE Research Symposium

 

[lgelb]

The FDA has approved three additional doses for those who were enrolled in the EAP.

 

[Vincent]

I was talking to one of Canada's top ALS researchers and without going to a lot of details just said " there's some unconventional data analysis". It just happens to be done by another Israeli lab. I smell a conspiracy. I had really hoped they would go away and terrorize the MS community and leave us alone.

 

[KevinM]

Interesting comment. I hope the “unconventional data analysis” comment was not directed at the peer reviewed data analysis article published by Dr. Merit Cudcowitz along with many of the top ALS specialists in the world. Here is a link to their analysis: A randomized placebo‐controlled phase 3 study of mesenchymal stem cells induced to secrete high levels of neurotrophic factors in amyotrophic lateral sclerosis

Hate to think these specialists didn’t know how to properly evaluate the data. Even if we suspend the idea that it might help those with less advanced disease, the bio marker data alone is compelling enough for further research.

 

[Clearwater AL]

Kevin's link.

For what it's value, last paragraph before Acknowledgments....

In summary, we present a thorough analysis of MSC-NTF phase 3 data, demonstrating a potential treatment effect on ALS disease progression linked to target biomarker changes and confirming treatment safety. While the primary and secondary endpoints failed to reach statistical significance in the overall study population, a pre-specified subgroup suggests across endpoints that MSC-NTF participants with less severe disease may have retained more function compared to placebo.

Two key words for thought, last sentence.... "may have"

 

[lgelb]

In fairness, Al, "suggests" and "may have" are standard when the P value exceeds the sought-after .05. The 19 percentage point difference between the treatment subgroup with a baseline FRS >/+35 and placebo groups (35% vs. 16%) in terms of stabilized ALSFRS score as defined below did not reach the .05 P value threshold that signifies the standard threshold for reporting a positive result. So it could well have been chance, and you have to say "may have."

And that analysis, where you are still only showing your desired treatment effect for a third of this treatment subgroup, was only done after the main analysis incorporating all the PALS treated failed to show a treatment effect, so you are kind of torturing the data here in an attempt to win approval and/or funding for more trials. There was also a late-breaking inclusion criterion for fast progressors in an attempt to amp up the treatment effect.

In addition to the squishy endpoint of "no more than 1.25 points lost," of course there's a lag time for treatment effects, losses are already underway when you start treatment, etc. It's not like, we shrunk this tumor and we know how. Thus, the extra doses past the main trial.

None of this rules out benefit for some, but kind of a Catch-22 because MCOs will only want to reimburse those most likely to benefit, but it's not yet clear who those are, and to find out would take more trials...

 

[Bestfriends14]

@Vincent my husband's neurologist, who is also well known for his ALS research, essentially said the same thing as your researcher said. He said this has been one of the most profitable studies he's seen in his entire career and he does not see them coming to a firm conclusion on anything any time soon ; it has been far too profitable.