The June 3 FDA Rare Disease Roundtable reinforced several principles that directly apply to ALS and #NurOwn
• Rare disease decisions should consider the totality of evidence, not just a single traditional efficacy analysis.
• Patient perspectives and tolerance for uncertainty matter, especially in fatal diseases with no cure.
• Congress intended FDA to use regulatory flexibility when conventional trials are impractical.
• Real-world evidence, external controls, biomarkers, and natural history data all have a role in evidence generation.
For ALS patients facing certain progression and death, these are the very tools FDA has available to make patient-centered decisions when unmet need is profound.
There is no longer any question the NurOwn Citizens petition should be approved.@FDA_KyleD@SecKennedy@aVoice4ALS@iamalsorg@BrainstormCell@PeterPitts@Neuroscope_mp@MGHNeurology@CraigReagan2@MinoShah
https://t.co/VThvxUq5Ox
I’m from Hico, TX and someone impacted by #ALS. @judgecarter, please increase ALS research funding in the FY2027 budget. This funding helps drive treatments and hope for a cure. See why it matters: https://t.co/LH6RAZjkK8
Good luck to @iamalsorg in today’s FDA meeting.
ALS patients don’t need perfection from the system. They need a chance.
Every day spent waiting for certainty is a day patients may never get back. There is no better example than the 330+ days now patients have been waiting for the NurOwn Citizens Petition Approval which could have improved outcomes of ~5000 US lives in those 330+ days.
The question isn’t just whether FDA can be flexible in ALS.
The question is whether we can afford not to be.
#NurOwn @PeterPitts@Neuroscope_mp@houmanhemmati@FDA_KyleD@sabrevaya@bsw5020@aVoice4ALS
Pleading with @FDACBER and @US_FDA to heed RWE in #NurOwn Citizens Petition, including 7 YRS of unprecedented survival data In EAP compared to 2.5 yrs in natural history. Grant Accelerated Approval for people with #ALS just like you did these therapies.
SEE Petition at https://t.co/fpxngrtveK
@FDA_KyleD@FDACommissioner@SecKennedy@POTUS
Sure hope they respond to the 300+ page #NurOwn Citizens' Petition containing unprecedented 7+ yr survival data for #ALS dwarfing 2.5 yr natural history. @CBER had 180-day deadline and its now been over 330 days. Only communication was one form letter from Szarama.
Urgency has always been woefully lacking for terminal #rarediseases.
Unfathomable that outcome today is no different than when #LouGehrig died on this day in 1941.
NurOwn helps people live longer & live better.
.@PeterPitts was once critical of @BrainstormCell. He then looked at the science, the patients, and the unmet need and changed his mind.
At the same time, FDA leadership @FDA_KyleD@SecKennedy are taking a fresh look at how therapies for rare and fatal diseases should be evaluated.
Maybe the question isn’t whether the system should be more flexible.
Maybe the question is why ALS patients have had to wait so long for that flexibility to be applied. Thank you @hmac8
https://t.co/E7GxcfqoYF
Thank you Sir @PeterPitts for a very thoughtful, comprehensive review.
Although ALS is the primary example you use in this review, the same principles are applicable in terminal disease like Huntington's Disease. $QURE
@PeterPitts The next FDA Commissioner must follow the laws and ensue each Center acts consistent with Congressional intent for #rarediseases -- something every @FDACommissioner has failed to do since the passage of CURES in 2016.
I guess a new month also comes with a new loss of some function.. A big F U to #ALS..!!! I still cant wrap my head around how I can cope w/this cruel nasty disease! Even after 4yrs.. 😪
Regarding the original #NurOwn phase 3 trial and patient access via approval of the Citizens Petition:
1. FDA reviewed the Phase 3 protocol before enrollment.
2. FDA allowed the study to proceed.
3. FDA did not identify the ALSFRS-R floor effect as a fatal flaw before the trial.
However, Years later, analyses suggested the floor effect may have masked treatment benefit in more advanced patients. FDA has since negotiated a SPA with @BrainstormCell specifically designed to account for what was learned from the original study.
If FDA and the sponsor both learned after the trial that ALSFRS-R had a floor-effect problem, patients should not be punished for the limitation of the measuring tool.
In #ALS, a broken ruler shouldn’t outweigh clinical signal, statistically significant biomarker evidence, safety, and the realities of a 100% fatal disease.
The ALSFRS-R floor effect was a limitation of the measurement tool, not proof that NurOwn failed.
FDA exists to apply scientific judgment when evidence is imperfect. That’s exactly what regulatory flexibility is for.
Approve the NurOwn Citizen Petition. Use the FDA-cleared Phase 3b study as the confirmatory trial. Patients have waited long enough. @SecKennedy@FDA_KyleD@Neuroscope_mp@houmanhemmati@aVoice4ALS@PeterPitts@MinoShah@DrOz@FDACBER@CraigReagan2
Important piece by @PeterPitts
NurOwn belongs in this conversation too.
As Pitts wrote, “every delayed approval has a human cost.” For ALS, that cost is brutally literal: roughly 5,000 Americans are diagnosed each year, and most patients die within 2–5 years. (CDC)
While the NurOwn Citizen Petition sits unresolved, nearly 5,000 people with ALS have died waiting.
This is a 100% fatal disease. Patients do not have the luxury of perfect timelines, perfect data, endless process, or regulatory hesitation disguised as rigor.
If a treatment shows signs of extending life and preserving function in ALS, the FDA should be leaning into the urgency to approve, not delay. @houmanhemmati@FDA_KyleD@SecKennedy@DrPatrick@aVoice4ALS 👇
Five days ago I connected Peter Pitts’ argument on delayed approvals to the ongoing #NurOwn debate.
Today, he joins the @BrainstormCell Board.
What makes this noteworthy is that the discussion is shifting from whether regulatory flexibility should exist to how it should be applied.
In diseases with near-certain mortality and profound unmet need, the ethical burden should not rest solely on approving too early. It must also account for approving too late.
Peter Pitts has long been willing to confront that uncomfortable reality: that delay is not a neutral decision, and that patients bear the consequences when systems become more focused on eliminating uncertainty than addressing urgent unmet need.
His experience at the FDA, combined with his willingness to challenge conventional thinking, brings an important perspective to this conversation.
Welcome, @PeterPitts ! I hope your voice helps advance a more patient-centered approach to evaluating therapies for those facing terminal diseases with few remaining options. @FDA_KyleD@houmanhemmati@LoriMills4CA42@Christina4HD@KerriHT@SecKennedy@aVoice4ALS@MinoShah@DrPatrick@bullishbruk@realDonaldTrump
@RainConsulting@CraigReagan2@DrToonces The Right to try Bill was passed in 2017 by President Trump. It was to help all fatal diagnosed patients a chance at another day. It’s a sin how you ignore fatal lives. Wait until you’re one of them.
@DrToonces Spot on Dr. and also why I also keep highlighting NurOwn for ALS. There was actually data that showcased a patient whose progression stopped at the adcomm and it was ignored. 5 year survival in the NurOwn EAP is 100% vs. 20% in ALS natural history.
The old FDA claimed substantial evidence and barried it. @realDonaldTrump gave it a chance through Right to Try and a patient started walking again and is still living today in 100% fatal ALS 8+ years. @FDA_KyleD talk to @houmanhemmati and pull in Dr. Nathan Staff and Dr. Anthony Windabank while your at it from @MayoClinic. They understand the stem cell science behind #NurOwn and have witnessed the patient experience first hand.
Being a public servant should mean serving the public, working for the patient, and not serving statistical ideology. It’s time the FDA takes action! @SecKennedy