The survival rates observed in the study raise serious concerns about the safety and efficacy of NurOwn.
The Food and Drug Administration (FDA)’s Cellular, Tissue and Gene Therapies Advisory Committee has recently cast a vote against the approval of NurOwn®, an investigational mesenchymal stem cell therapy for the treatment of amyotrophic lateral sclerosis (ALS). This decision has significant implications for the future of ALS treatment.
HCN Medical Memo
This decision by the FDA panel could have profound implications for healthcare professionals treating ALS. Although stem cell therapy has been viewed as a promising avenue for ALS treatment, this vote underscores the importance of rigorous clinical trials and safety data. Healthcare professionals must remain vigilant in evaluating new treatments and consider the potential risks associated with experimental therapies.
- The FDA panel voted 17 to 1 against the approval of NurOwn.
- NurOwn (debamestrocel) consists of autologous mesenchymal stem cells that have been induced to secrete neurotrophic factors.
- The therapy was believed to slow disease progression and improve symptoms in patients with ALS.
- The committee reviewed the Biologics License Application (BLA) for NurOwn, which included findings from a double-blind, placebo-controlled phase 3 trial.
- The study showed no statistically significant difference between the groups for the primary endpoint.
- Survival was observed to be worse in patients who received NurOwn.
- Survival Rates: At 28 weeks, there were 10 deaths in the NurOwn arm and three deaths in the placebo arm. Kaplan-Meier estimates of survival were reported to be 88.3% (95% CI, 79.3-93.6) for NurOwn and 94.4% (95% CI, 81.2-98.4) for placebo.
According to the ALS Association, more than 5,000 people are diagnosed with ALS each year in the US, with an estimated prevalence of 30,000 at any given time.
More on ALS