SHERIDAN, WYOMING – July 8, 2025 – BrainStorm Cell Therapeutics is once again at the center of the amyotrophic lateral sclerosis (ALS) treatment debate as patients formally petition the FDA to reconsider the approval of its investigational stem cell therapy, NurOwn. Citing new survival data and expanded access outcomes, the Citizens’ Petition seeks regulatory reassessment of the once-rejected therapy, underscoring the shifting landscape of ALS drug development and patient advocacy.
Renewed Push for Regulatory Reevaluation
The Citizens’ Petition, submitted last week by several individuals living with ALS, requests that the agency approve the stem cell therapy based on “new evidence and totality of evidence.”
In response, BrainStorm issued a public statement supporting the petition, stating that seeking a new review of NurOwn’s data “will provide a critical opportunity to reaffirm its potential as therapy for amyotrophic lateral sclerosis (ALS).”
Addressing Historical Trial Limitations
The Tel Aviv and New York–based company has faced years of regulatory hurdles. In 2021, the FDA advised BrainStorm that its Phase III trial— which did not meet the primary endpoint—was insufficient to support a Biologics License Application (BLA). The following year, BrainStorm was again rebuffed when the FDA issued a Refusal to File Letter for a BLA the company had submitted based on a correction to analyses of the Phase III trial— a change it claimed resulted in a statistically significant treatment difference.
The persistent biotech then sought and secured an advisory committee meeting for NurOwn, but this resulted in a 17-1 vote against the therapy and the company’s withdrawal of the BLA prior to its Dec. 8, 2023 PDUFA date.
Despite these outcomes, BrainStorm has long contended that the missed endpoint in the first Phase III trial was due to a “floor effect,” which occurs when the scale of measurement is unable to capture patients’ progression as their condition becomes severe.
That trial’s primary endpoint— a 1.25 points per month improvement on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)—was met by 34.7% of NurOwn participants versus 27.7% of placebo comparators. However, in a pre-specified subgroup of patients with early-stage disease, the difference was much larger, at 34.6% vs. 15.6%.
Expanded Access Data Drives Renewed Optimism
BrainStorm has since moved forward with plans for a Phase IIIb trial. According to clinicaltrials.gov, the study had an estimated start date of June 30. In an email Tuesday afternoon, a BrainStorm representative told BioSpace the trial will commence upon securing funding. Fifteen clinical trial sites have been selected and Clinical Trial Agreements are currently being executed, they said.
Additionally, recently reported data from 10 Phase III participants who entered NurOwn’s expanded access program (EAP) after the trial showed that nine survived more than five years from ALS symptom onset, according to a press release issued by BrainStorm on June 16. According to other published estimates, approximately 10% of people with ALS survive past 5 years, BrainStorm said. Median survival in the EAP was 6.8 years from symptom onset.
“These results strongly suggest that the observed survival outcomes in the EAP patients are unlikely to be due to chance alone and further underscore the need for continued scientific exploration,” BrainStorm said in Tuesday’s release.
In an email to BioSpace, a representative for the patient petitioners pointed to Phase III and EAP data including “100% five-year survival versus 20% ALS natural history” and “extended periods of progression-free survival (PFS) ranging from a few months up to 17 months when receiving NurOwn.” These data, they said, document changes that are “unprecedented in the history of ALS clinical trials.”
Strategic Implications for ALS Innovation
The petition comes during a challenging period for ALS research. After winning approval as just the third-ever drug for ALS in September 2022, Amylyx withdrew Relyvrio from the U.S. and Canadian markets last spring after it failed its highly anticipated Phase III PHOENIX trial. Despite this, the industry is regaining momentum, with companies like Korro Bio, Coya Therapeutics, and QurAlis actively pursuing novel targets for the devastating neurodegenerative disease.
BrainStorm’s continued regulatory engagement, combined with strong support from the ALS community, may signal a turning point for NurOwn and for future FDA decision-making on cell-based therapies.
Learn more at www.brainstorm-cell.com