BrainStorm's NurOwn Sparks New Hope for ALS Patients Amid Regulatory Challenges

BrainStorm Cell Therapeutics' experimental ALS treatment NurOwn has taken center stage once again as patients and advocates push for its approval, citing promising survival data and the urgent need for effective therapies. The renewed interest comes in the wake of regulatory setbacks and highlights the complex landscape of ALS drug development.

Citizens' Petition Calls for FDA Reconsideration

A group of ALS patients and trial participants have filed a Citizens' Petition with the FDA, urging the agency to reconsider NurOwn based on new evidence and the totality of data available. The petition, submitted on July 3, 2025, points to real-world data and an impressive 90% survival rate observed in an expanded access program (EAP).

Lead petitioner Nick Warack, who lives with ALS, stated, "We believe the recently published EAP data for NurOwn demonstrate a dose-dependent response that enhances the already impressive results seen from the 3 doses in the Phase 3 trial."

The petition has garnered support from neurologist Danielle Geraldi-Samara, who appealed to the FDA on July 17, citing the 21st Century Cures Act. This legislation encourages the consideration of real-world evidence and clinical outcomes assessments to accelerate drug development.

Survival Data Defies Expectations

BrainStorm's June 16 press release revealed that nine out of ten Phase III participants who entered the EAP survived more than five years from ALS symptom onset. This 90% survival rate significantly exceeds the estimated 10% five-year survival typically observed in ALS patients.

Stacy Lindborg, BrainStorm board member and former co-CEO, remarked, "At the end of the day, this defies logic," adding that these results should prompt further discussion.

The petition organizers draw parallels between NurOwn's survival data and that used to support accelerated approval of many cancer therapies, arguing for a similar approach in ALS treatment evaluation.

Regulatory Hurdles and Future Plans

NurOwn's journey through the regulatory process has been fraught with challenges. The FDA rejected BrainStorm's biologics license application in 2022, leading to an advisory committee meeting in 2023 where the therapy was voted down 17-1. Subsequently, BrainStorm withdrew its BLA before the December 2023 PDUFA date.

The company is now working to secure funding for a Phase IIIb trial, initially slated to begin in June 2025. However, the trial's estimated completion date of May 2029 has raised concerns among patients and families who fear they may not survive long enough to benefit from the treatment.

Chaim Lebovits, CEO of BrainStorm, expressed the company's commitment to completing the Phase IIIb trial while acknowledging funding uncertainties. "Any positive regulatory action regarding NurOwn's path forward would undoubtedly change the landscape for funding our Phase 3b trial," Lebovits stated.

As the ALS community awaits the FDA's response to the Citizens' Petition, the case of NurOwn continues to highlight the delicate balance between regulatory rigor and the urgent need for new treatments in diseases with limited therapeutic options.