BrainStorm Cell Therapeutics announced that the production of its therapy NurOwn will be expanded to support upcoming clinical trials, namely a Phase 3 trial in amyotrophic lateral sclerosis (ALS) and a Phase 2 trial in progressive multiple sclerosis (MS).
BrainStorm’s proprietary, stem cell-based technology called NurOwn consists of autologous bone marrow-derived mesenchymal stem cells, meaning cells from an individual that are reinserted into the same person. These stem cells generate molecules called growth factors that can help neurons survive longer at or near the site of injury or damage, and are being evaluated as a treatment for various neurodegenerative diseases.
The NurOwn technology takes autologous mesenchymal stem cells from patients, which are then grown and manipulated under patented conditions 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.
NurOwn has been tested in several animal models for various diseases, and has shown a good safety profile and promising signs of efficacy in clinical trials in ALS.
BrainStorm will now increase its NurOwn manufacturing for a Phase 2 trial in progressive MS with the help of the Connell and O’Reilly Families Cell Manipulation Core Facility at Dana-Farber Cancer Institute in Boston, Massachusetts.
“Cellular therapies represent an exciting new therapeutic modality that can potentially benefit patients who do not have other effective treatment options. We are very glad to support BrainStorm’s plan to evaluate the potential role of NurOwn in the treatment of other debilitating neurologic diseases such as progressive multiple sclerosis,” Jerome Ritz, MD, executive director of the Cell Manipulation Core Facility at Dana-Farber, said in a press release.
The team at Dana-Farber, which is already supporting an ongoing Phase 3 ALS trial (NCT03280056), is collaborating with clinical researchers in advancing novel cell-based therapies in trials for neurodegenerative diseases, as well as cancer.
“We are excited to rapidly advance our investigational stem cell therapy for progressive multiple sclerosis patients and sincerely thank the Connell and O’Reilly Families Cell Manipulation Core Team for allocating resources and dedicated manufacturing space to allow us to initiate our Phase 2 progressive multiple sclerosis study in early 2019,” said Chaim Lebovits, president and CEO of BrainStorm.
In December 2018, the U.S. Food and Drug Administration approved BrainStorm’s Investigational New Drug application for NurOwn, which allowed the company to open this Phase 2 trial in progressive MS patients.