Dear BrainStorm Amylyx, I join the #ALS community calling for you to apply for U.S. FDA approval of NurOwn and AMX. I also urge you to immediately unblind trial participants & fully enroll confirmatory trials, before or after approval. It’s your duty to prove effect.

Thank you, Dr. Danielle Carnival for your service! Big shoes to fill. As a member of the #ALS community, what “critical role” can we play in selecting your next CEO, I AM ALS?

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Take a read, important context. We need another trial on #NurOwn to prove efficacy. Until then, BrainStorm needs to #unblindnurown & put money where it’s mouth is & pay for expanded access if they believe in NurOwn. It’s incumbent on pharma to prove efficacy before payday.

True. BrainStorm’s current EAP is only 11-12 patients who likely responded in the trial. That won’t produce any data that FDA will use. They should use that money on another trial and provide access to ALL if they believe in Nurown.

And unblind your trial participants!!!!!!

We don’t need lower standards, we need better science. To get a drug approval, pharma needs to put in the work and prove effectiveness. No proof, no payday.

We can’t let ourselves be co-opted into using pharma talking points about lowering standards & equate them to “flexibility”. PALS should have access to all promising treatments that are safe. Those treatments shouldn’t be approved without showing efficacy. NO PROOF, NO PAYDAY.

The cynic in me fears the congressional hearing with the FDA on ALS and "other neurological diseases" could be taken over by discussions of "other neurological diseases" namely the Aduhelm approval. We need to make sure ALS is well represented both inside and outside the hearing.

So it’s not an #ALS hearing? Regardless, let’s not spoil this amazing opportunity by letting it go off the rails on #Aduhelm. There is real danger here in backfire & flexibilities being stripped away from FDA, like accelerated approval. We need more flexibility, not less.

.BrainStorm unblind now, show us the full data/ journal, apply for BLA if you think it works (or start another trial now on responders with EA, which you’ll be told to do regardless), but for gods sake stop letting #ALS patients die angry!

Kewl. You have no legitimacy to dictate how your own agency is investigated. You signed off 🖊

What EndALS said

So many issues with this piece in JAMA Internal Medicine from U.S. FDA officials (Dunn, Stein, & Cavazzoni) attempting to justify their poor choice in approving #aducanumab - don't bother reading it unless you want your blood to boil. I'll summarize instead: jamanetwork.com/journals/jamai… 1/

The evidence of absence on #NurOwn is deafening. Most #ALS researchers cower in labs hoping it will blow over, but it won’t until they stick out their necks & tell us how it is: is more evidence needed? The onus is on researchers to counter pharma TPs & preserve U.S. FDA integrity

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Another trial is needed to confirm efficacy. No payday before proof.