- 90% of ALS patients treated with NurOwn (BCLI, Financial) survived over 5 years, far exceeding the typical 10% survival rate.
- The median survival duration reached 6.8 years, with 6 out of 10 participants still alive after 7 years.
- BrainStorm Cell Therapeutics is preparing for a Phase 3b trial under an FDA Special Protocol Assessment following these promising results.
BrainStorm Cell Therapeutics Inc. (BCLI), a leader in adult stem cell therapies for neurodegenerative diseases, has released noteworthy survival data from its Expanded Access Program (EAP) for NurOwn® in treating amyotrophic lateral sclerosis (ALS). According to the study, 90% of the cohort survived for more than five years after the onset of ALS symptoms, significantly outperforming the typical 10% survival rate associated with the disease.
The EAP involved 10 participants who had completed BrainStorm's Phase 3 clinical trial. The median survival was recorded as 6.8 years, with 6 out of 10 patients alive beyond the seven-year mark from symptom onset. These findings are viewed as a substantial endorsement for the forthcoming FDA-aligned Phase 3b trial of NurOwn, set to explore the therapy's potential further.
Initial evaluations showed participants had a mean ALS Functional Rating Scale-Revised (ALSFRS-R) score of 35.8, which declined to 31.4 by the beginning of the EAP. Despite the small sample size, the high survival rate supports the hypothesis of NurOwn's efficacy in prolonging life expectancy in ALS patients.
This new data boosts BrainStorm’s confidence in advancing to the next clinical trial phase. Under the FDA's Special Protocol Assessment, the company aims to provide compelling evidence supporting NurOwn’s potential as an effective therapy for ALS, addressing a critical need for effective treatment options in this field.
