Mount Sinai Taking Part in Phase 2 Trial of NurOwn Cell Therapy in Progressive MS

Mount Sinai Medical Center has joined with BrainStorm Cell Therapeutics to explore the safety and efficacy of NurOwn as a potential treatment for progressive multiple sclerosis (MS) in an ongoing Phase 2 trial.

The New York center is the fourth clinical site participating in the trial, in addition to Keck School of Medicine of The University of Southern California (USC), Stanford University School of Medicine, and the Cleveland Clinic in Ohio.

Mount Sinai is ready to start enrolling patients under the supervision of neurologist Fred Lublin, MD, and his clinical team at The Corinne Goldsmith Dickinson Center for Multiple Sclerosis.

“We are happy to be a part of this exciting study to determine if neurally-directed stem cells can be a therapeutic approach to treating MS,” Lublin said in a press release.

NurOwn is a cell-based therapy that uses the patients’ own bone marrow-derived mesenchymal stem cells (MSC) to promote and support the repair of nerve cells.

Patient’s MSCs are modified in the lab to secrete growth factors that are believed to protect nerve cells from damage, to promote the repair of the protective myelin sheath in nerve cells (which is destroyed in MS), and potentially slow or halt disease progression.

The open-label Phase 2 clinical trial (NCT03799718) will enroll and treat up to 20 adults with either primary progressive MS (PPMS) or secondary progressive MS (SPMS).

All participants will undergo a bone marrow biopsy to collect MSCs, which will later on be injected back to the patient through three intrathecal administrations — injected directly into the cerebrospinal fluid — over 16 weeks.

During this time, and for the following 12 weeks, researchers will evaluate the safety of the  procedure, as well as the neuromodulatory effect of the modified MSCs.

To confirm that NurOwn cells are delivering neurotrophic factors and immunomodulatory signaling molecules as expected, the research team will look for an increase in the amount and type of these biomarkers in patients’ cerebrospinal fluid following the cell transplants.

“BrainStorm looks forward to partnering with and supporting Dr. Lublin and the dedicated clinical trial team at the Mount Sinai Hospital to quickly advance the Phase 2 progressive MS clinical trial,” said Ralph Kern, MD, MHSc, BrainStorm’s chief operating officer and chief medical officer.

For more information about the trial, including its sites and contacts, please visit this link.

NurOwn has been tested in animal models for various neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), where it showed a good safety profile and promising efficacy signs.

An ongoing Phase 3 trial (NCT03280056) testing NurOwn in people with ALS is expected to conclude in December 2020.