Phase 2 Trial of NurOwn Stem Cell Therapy in Progressive MS Planned for US, BrainStorm Announces

BrainStorm Cell Therapeutics is planning to launch a Phase 2 clinical trial in the United States to evaluate the safety and activity of its lead cell therapy candidate, NurOwn, in people with progressive multiple sclerosis (MS).

The company announced that has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) requesting to start the trial. A separate Phase 3 study of NurOwn in treating amyotrophic lateral sclerosis (ALS) patients is underway and enrolling.

“We plan to quickly initiate the Phase 2 clinical study of NurOwn in progressive MS to address the significant unmet need for MS patients,” Ralph Kern, MD, chief operating officer and chief medical officer of BrainStorm, said in a press release.

NurOwn is an autologous (self) stem cell therapy that takes the patient’s own mesenchymal stem cells (MSCs), modifies them and then returns them to the patient to promote nerve cell-supporting mechanisms.

These stem cells are engineered to produce signaling molecules that regulate the activity of surrounding cells, as well as to secrete neurotrophic factors (NTF) that support the growth, survival, and differentiation of nerve cells. These features support expectations that MSCs could be potential regulators of neurodegeneration, while protecting neurons from damage.

Submitting an IND is an “important step toward the development of NurOwn in progressive MS,” said Chaim Lebovits, president and CEO of BrainStorm, and “confirms our commitment to bring to the market innovative cellular therapies for neurodegenerative disease.”

The company, Kern added, has already “demonstrated [the] safety and efficacy experience of NurOwn in our amyotrophic lateral sclerosis (ALS) clinical program.”

Results from a Phase 2 trial (NCT02017912) showed that NurOwn could significantly slow functional decline compared to placebo in patients with early stage ALS. Treatment also showed immunomodulatory and neuroprotective effects via regulation of small RNA molecules in the cerebral spinal fluid.

BrainStorm is currently evaluating NurOwn’s safety and efficacy in about 200 adults with ALS in a Phase 3 trial (NCT03280056), now enrolling eligible patients at sites in the U.S. and Canada.