The ALS Association

ALS Ice Bucket Challenge Progress
Carrie Munk
The ALS Association
(571) 319-3047



Update on ALS BrainStorm Stem Cell Clinical Trials

Washington, D.C., (January 11, 2016) — Today, JAMA Neurology published a paper titled, “Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis – Results of Phase1/2 and 2a Clinical Trials.” This study comes out of a collaboration of researchers from BrainStorm Cell Therapeutics and the Hadassah-Hebrew University Medical Center both located in Israel.

The researchers set out to understand the safety and clinical effects of treatment with mesenchymal stem cells induced to secrete neurotrophic factors (MSC-NTF) in patients with ALS. Stem cells used in this study are adult mesenchymal stem cells derived from bone marrow samples given by participants in the trial. The bone marrow cells are turned into stem cells that secrete the NTFs. NTFs are a type of nutrient for cells and were previously shown to have protective effects in animal models of neurodegenerative disease.

The new study showed that implanting stem cells that produce neurotrophic factors (MSC-NTF) into either muscle or the intrathecal space surrounding the spinal cord, or both, after 6 months were shown to be safe and associated with only mild and transient adverse effects, principally headache and fever. The study is the first in which stem cells that have been modified to secrete neurotrophic factors have been implanted in people with ALS.

The study included 26 patients in all. Measurements of breathing ability and overall function suggested the treatment may have been associated with small reductions in the rate of disease progression. These results must be treated with caution, since the trial did not include a placebo comparator, making it difficult to know whether the effects were due to the treatment or to some other factor.

“We are encouraged to see that implantation of modified stem cells in this trial was safe for people with ALS,” said Association Chief Scientist Lucie Bruijn, Ph.D., M.B.A. “We look forward to learning more about the safety and efficacy of this therapeutic approach, as more trials are performed and results are reported.”

Information on additional clinical trials, including those exploring the potential of stem cell implantation, can be found on the NEALS website:

Details on the NEALS-sponsored BrainStorm trial of implanted NTF-MSCs can be found here:

About ALS
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front.  By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.  For more information about The ALS Association, visit our website at

Powered by Blackbaud
nonprofit software