The trial (NCT03799718) now will enroll patients at the Keck School of Medicine of The University of Southern California (USC), and its academic medical center, Keck Medicine of USC. The other two clinical sites, currently recruiting participants, are the Stanford University School of Medicine, California, and the Cleveland Clinic, Ohio. (More information about enrollment is available here.)
Daniel Pelletier, MD, a professor of neurology and the Eric and Peggy Lieber Chair in Neurology at the Keck School of Medicine, will lead the trial.
“The BrainStorm team is pleased to have the opportunity to advance our Phase 2 clinical study for progressive MS at the USC Keck School of Medicine,” Chaim Lebovits, president and CEO of BrainStorm, said in a press release.
“We look forward to collaborating with Dr. Pelletier, an internationally renowned leader in the field of multiple sclerosis research and his team,” Lebovits added.
NurOwn is an investigational stem cell-based technology made of autologous bone marrow-derived mesenchymal stem cells. Autologous refers to cells that are harvested and reinserted into the same person. These stem cells secrete growth factors that may help neurons in close proximity to sites of injury or damage to survive longer.
NurOwn technology isolates autologous mesenchymal stem cells from patients, which are then expanded and manipulated in the lab to make them generate high levels of immunomodulatory cytokines (signals that affect the immune system) and neurotrophic factors (those related to the growth of nerve cells).
These therapeutic signals are communicated to the site of damage to elicit a desired biological effect, and ultimately slow or stabilize disease progression.
The technology has been tested in animal models for various neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), where it showed a good safety profile and promising signs of being effective. An ongoing Phase 3 trial (NCT03280056) is testing NurOwn in ALS patients.
“Our NurOwn technology is an innovative investigational therapy currently being studied in Phase 3 for ALS, and in Phase 2 for progressive MS patients,” Lebovits said.
In December 2018, the U.S. Food and Drug Administration approved the investigational new drug application for NurOwn, which allowed BrainStorm to launch this Phase 2 trial in progressive MS patients.