NurOwn Now Being Produced at 2nd U.S. Manufacturing Site, Brainstorm Announces

BrainStorm Cell Therapeutics has partnered with the Connell and O’Reilly Families Cell Manipulation Core Facility at Dana-Farber Cancer Institute for the manufacturing of NurOwn, a therapy candidate for amyotrophic lateral sclerosis (ALS), for BrainStorm’s ongoing Phase 3 trial.

This is the second U.S. production site for NurOwn; the first was the City of Hope’s Center for Biomedicine and Genetics.

“Dana Farber is a world-renowned institution with decades of experience in stem cell transplantation. Its strong capabilities in this area and proven track record in the manufacture of NurOwn in the phase 2 trial, make it an ideal partner as we advance our Phase 3 trial to completion,” Chaim Lebovits, president and CEO of BrainStorm, said in a press release.

NurOwn is a cell therapy platform that takes mesenchymal stem cells (MSCs) from patients, and converts them into biological factories producing several molecules, called neurotrophic factors, known to support the survival of neurons in a variety of conditions and animal models of neurodegenerative disorders.

Besides allowing for more production of NurOwn for BrainStorm’s pivotal Phase 3 clinical trial (NCT03280056), the alliance with Dana-Farber will also support the manufacturing of the cell therapy for additional clinical indications.

The randomized, double-blind, placebo-controlled, multicenter Phase 3 trial is designed to evaluate the safety and effectiveness of  repeated administration of NurOwn in approximately 200 ALS patients. The trial is still recruiting participants at six sites in the U.S.

Participants are randomized to undergo three transplants with NurOwn or a matching placebo every two months. First, researchers collect a sample of the patients’ bone marrow, which contain MSCs. Those MSCs from patients in the treatment group are then induced to cells producing neurotrophic factors and transplanted back into the patients.

The study’s primary objective is to compare the safety and effectiveness of the treatment, measured by the ALS functional rating scale score, between the experimental and control groups.

“We are pleased to support this important clinical trial. ALS is a devastating disease with no effective therapies. Our participation will allow more rapid patient accrual and will lead to earlier completion of the study,” said Jerome Ritz, MD, executive director of the Connell and O’Reilly Families Cell Manipulation Core Facility.

According to Brainstorm, the first meeting of the Phase 3 Data and Safety Monitoring Board is expected by the end of August.