The Phase 3 clinical trial testing BrainStorm Cell Therapeutics‘ cell therapy candidate NurOwn in amyotrophic lateral sclerosis (ALS) patients is continuing as planned after a second safety assessment by the trial’s independent Data Safety Monitoring Board (DSMB) found no reasons to stop, the company announced.
The DSMB’s recommendation comes after a pre-specified interim analysis of the first 106 ALS patients treated repeatedly with NurOwn in this randomized, placebo-controlled clinical trial.
“After reviewing all of the safety data as of September 30th, the DSMB has recommended the study continue without any changes in the protocol. We did not identify any significant safety concerns,” Carlayne Jackson, MD, a professor of Neurology and Otolaryngology UT Health San Antonio and the DSMB chairperson, said in a press release.
DSMBs consist of research experts who monitor the progress of a clinical trial and review safety and efficacy data while the study is ongoing. This panel can recommend that a trial be stopped early because of safety concerns or evidence a therapy is not working as intended, or if the trial’s main goals have already been reached.
NurOwn consists of mesenchymal stem cells (MSCs; stems cells able to generate various cell types) collected from a patient’s bone marrow. These MSCs are expanded and matured into a specific cell type — called MSC-NTF — by growing them under conditions that induce them to secrete high levels of neurotrophic factors (NTFs) that support the growth, survival, and maturation of nerve cells.
MSC-NTF cells also deliver immune system regulating cytokines, small proteins important in cell signaling or messaging, to sites of damage, BrainStorm reports. It is thought this will help to slow or stabilize disease progression.
The double-blind Phase 3 trial (NCT03280056), fully enrolled at its six U.S. sites, is investigating use of NurOwn in 200 ALS patients whose symptoms became evident within two years of the study’s start. Patients are randomized 1:1 to either NurOwn or placebo, given via intrathecal (spinal canal) injection every two months.
The study’s primary measures of safety and efficacy are being determined using the ALS functional rating scale score (ALSFRS-R; a score of abilities like swallowing, speech, handwriting, walking, etc.) in patients after 28 weeks of treatment compared to placebo.
A secondary goal is assessing how biomarkers, such as cell-secreted neurothrophic factors, inflammatory agents, and cytokines, change in the blood and cerebrospinal fluid (the liquid surrounding the brain and spinal cord) after treatment with NurOwn.
BrainStorm is expecting to have topline trial data by the end of 2020, which will potentially support the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration requesting approval.
“We are very pleased with the DSMB recommendation that the Phase 3 clinical trial continue without any protocol modification. This represents an important clinical trial advancement for BrainStorm and for the development of NurOwn as an innovative cellular therapy approach for ALS patients,” added Ralph Kern MD, BrainStorm’s chief operating officer and chief medical officer.
This clinical trial is being funded by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and other types of investment.
NurOwn was given orphan drug status by both the FDA and the European Medicines Agency (EMA) as a potential ALS treatment.
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