BrainStorm Cell Therapeutics has announced that the first patients have been enrolled in the phase 3 clinical trial of NurOwn for the treatment of amyotrophic lateral sclerosis (ALS) at the Massachusetts General Hospital and UC Irvine Medical Center in California.

The trial is expected to enroll approximately 200 patients and will be conducted at six leading ALS clinical sites in the US The primary outcome measure will be the ALSFR-S score responder analysis. The patient population will be optimized to include the pre-specified subgroups who demonstrated superior outcomes in the NurOwn Phase 2 ALS clinical trial. Top-line data are expected in 2019.

"NurOwn is a highly innovative and advanced stem cell therapy now being studied in a phase 3 trial in ALS. The support of this trial by the California Institute of Regenerative Medicine (CIRM) highlights the importance of addressing ALS unmet need beyond currently available therapies, which slow disease progression but do not maintain or restore function," said Chaim Lebovits, president and chief executive officer of Brainstorm. "BrainStorm plans to quickly advance the phase 3 trial to confirm promising phase 2 efficacy findings and bring much needed hope to ALS patients and their families."

Dr. Robert Miller, director of the Forbes Norris ALS Research Center at California Pacific Medical Center, added: "Stem cells are emerging as a potential viable candidate to treat ALS, and we are very excited to be participating in this important phase 3 trial. The clinical data from NurOwn have been encouraging, demonstrating clear evidence of clinical benefit and supported by analysis of inflammatory markers and neurotrophic factors in treated patients. NurOwn cells have the important advantage of being autologous, so patients do not need to be immunosuppressed. In addition, the production process is relatively straightforward, and the harvesting and delivery of cells is much less invasive than other stem cell systems that have been tested in the clinic. We have been conducting ALS clinical trials for more than two decades at California Pacific Medical Center and this is, by far, the most exciting trial in which we have been involved to date."

Maria Millan, M.D., president and chief executive officer of CIRM said: "CIRM's partnership with Brainstorm Therapeutics represents an international collaboration to find a treatment for ALS or 'Lou Gehrig's Disease,' a devastating neurologic and fatal condition for which there is currently no durable treatment or cure. CIRM is partnering with Brainstorm to follow up on the Company's promising phase 2 trial in patients with ALS."

Abla Creasey, Ph.D. senior director of strategic Infrastructure at CIRM added: "Brainstorm will conduct this trial at multiple sites in California, including our Alpha Clinics Network and will also manufacture its product in California using CIRM-funded infrastructure."  

This trial is supported by a $16 million non-dilutive grant from CIRM. A milestone payment of $5.5 million, representing approximately 30% of the grant, has been received.