Phase II Trial for Stem Cell-Based Lou Gehrig’s Treatment Begins

BrainStorm Cell Therapeutics Inc., a developer of adult stem cell technologies for neurodegenerative diseases, announced today that the first patient was enrolled in its Phase II ALS trial at Massachusetts General Hospital (MGH) in Boston, Mass. ALS, or amyotrophic lateral sclerosis, is better known as Lou Gehrig’s disease. The condition degenerates nerve cells in the brain and spinal cord that control voluntary muscle movement.

BrainStorm’s Phase II trial is a randomized, double-blind, placebo controlled multi-center study designed to evaluate the safety and efficacy of transplantation of NurOwn, the company’s autologous mesenchymal stem cells secreting neurotrophic factors (MSC-NTF), in 48 ALS patients. The trial is also being conducted at the University of Massachusetts Memorial (UMass) Hospital in Worcester, Mass., and the Mayo Clinic in Rochester, Minn.

NurOwn is an autologous, adult stem cell therapy technology that differentiates bone marrow-derived mesenchymal stem cells (MSCs) into MSC-NTF cells. These neuron-supporting cells secrete elevated levels of neurotrophic, or nerve-growth, factors for protection of existing motor neurons, promotion of motor neuron growth, and re-establishment of nerve-muscle interaction. The ability to differentiate mesenchymal stem cells into MSC-NTF cells, and confirmation of their activity and potency before transplantation, makes NurOwn a first-of-its-kind approach for treating neurodegenerative diseases, the company claims.

Stem cell research is still a young field, and it has been an active area of research for ALS only for approximately eight years. Monica Carrasco, Ph.D., an associate research scientist at Columbia University, uses human stem cells to study the ways ALS functions. She recently told the Idaho State Journal that There is still a long road to travel before stem cell research into ALS reaches its full potential.

“You have to consider that only in 2006 was the technology developed to take skin samples and develop the skin samples into pluripotent stem cells,” Carrasco said. “This is unique and very new. It’s not just about using the cells; it’s about asking the right questions and understanding exactly what information you can get out of the cells. There is huge hope, but these are the really early days.”

BrainStorm Cell Therapeutics is an Israeli company with U.S. offices in New York, N.Y.