Patient dosing has finished in the pivotal Phase 3 trial assessing the safety and efficacy of repeat administrations of NurOwn, a cell-based therapy for people with amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics, the therapy’s developer, announced.
The Phase 3 trial (NCT03280056), which enrolled about 200 participants, is underway at six U.S. sites: the University of California, Irvine; Cedars-Sinai Medical Center; California Pacific Medical Center; Massachusetts General Hospital; the University of Massachusetts Medical School; and the Mayo Clinic.
Patients were randomly assigned to either three doses of NurOwn or a placebo, both administered every other month by injections directly into the spinal canal (intrathecal) over a period of four months.
The study’s main goal is to assess the safety and efficacy of NurOwn, as measured by changes in the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) score seen at 28 weeks (seven months) following the first treatment. ALSFRS-R assess such daily life abilities as swallowing, speaking, dressing and washing oneself, climbing stairs, and turning over in bed.
Secondary goals include treatment effects on the levels of several disease biomarkers, as found in samples of patients’ blood and cerebrospinal fluid (the liquid circulating in the brain and spinal cord).
BrainStorm expects top-line trial data to be available before the end of the year.
“Completion of participant dosing in this clinical trial is an important milestone and brings us a step closer to potentially filing a biologics license application to make MSC-NTF cells [NurOwn] available to people with ALS,” Chaim Lebovits, CEO of BrainStorm, said in the company’s press release.
NurOwn is a cell-based therapy that uses mesenchymal stem cells (MSCs) — cells that are able to give rise to nearly all tissues found in the body, including bones, muscles, and connective tissue — isolated from a patient’s own bone marrow.
After isolation, MSCs are grown in a lab dish and differentiated into cells that produce high levels of neurotrophic factors — compounds that promote the growth and survival of nerve cells. The modified cells are then returned to patients through an injection into spinal canal.
Brainstorm is also investigating the potential of NurOwn treat other neurological disorders, including multiple sclerosis (MS), Huntington’s disease, Parkinson’s disease, and autism spectrum disorder.
The open-label Phase 2 trial (NCT03799718) assessing three doses of NurOwn in people with progressive MS may still be recruiting at two sites in the U.S. The cell-based therapy has not yet entered clinical testing for other disorders.
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