ALS Study Shows Benefits of NurOwn Stem Cell Therapy

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astrocytesNew York and Israel-based, BrainStorm Cell Therapeutics, Inc., announced positive final results from a clinical trial of NurOwn™ in amyotrophic lateral sclerosis (ALS). The study reached its main goal, showing that NurOwn™ is safe and well-tolerated. In addition, the majority of the 14 participants in this study had an improvement (slowing) in disease progression at three months after the start of treatment.

In ALS (also known as Lou Gehrig’s Disease), nerve cells that control movement progressively die in the brain and spinal cord. This causes muscle weakness, paralysis, and eventually loss of breathing and death. NurOwn is a stem cell therapy that uses cells from the patient’s own bone marrow. The cells are genetically modified and injected back into the patient. They become a type of star-shaped brain cell called an astrocyte and secrete glial-derived neurotrophic factor (GDNF), a molecule that protects motor neurons from dying.

The study included 14 early-stage ALS patients. They received cells for three months, injected into the muscles and cerebrospinal fluid. They were then studied for an additional six months, with measurements taken once a month. Fifty percent had an improvement in the ALS Functional Rating Score-Revised (ALSFRS) and 67% had an improvement in forced vital capacity (FVC).  The ALSFRS measures ALS progression through a variety of tests of motor function and FVC measures lung function. The researchers estimated that NurOwn slowed the rate of progression of ALS by 45%, and also improved lung function.

“We are gratified to have the final data from this study and are very encouraged by the results,” remarked BrainStorm’s CEO Tony Fiorino, MD, PhD.  “This study not only extends our earlier phase 1/2 findings regarding the safety of NurOwn™, but also provide a consistent and highly promising picture of NurOwn’s efficacy. In particular, I would highlight that we observed not only a highly meaningful reduction in ALS progression on mean ALSFRS and FVC, but we saw subjects with prolonged stabilization and even improvements in function, and all this was achieved with just a single dose of NurOwn™.  We are excited to proceed to a multi-dose study to see if these positive results can be amplified and extended by administering repeated doses.”

Professor Dimitious Karussis of Hadassah Medical Center and the principal investigator of the trial, added “This is the second study of NurOwn™ I have completed in ALS patients, and my excitement for these cells as a possible treatment for ALS continues to grow.  I am impressed by the consistency of benefit of IT administration we have seen in both studies, and we saw in this study that almost every subject experienced clinical benefit, either on ALSFRS, FVC or both measures.  I believe that if future studies demonstrate a similar magnitude of benefit, NurOwn™ will become an important treatment option for patients suffering from ALS.”

The trial is a Phase 2a trial, and results from a larger Phase 3 trial are needed before the treatment can be approved by the Federal Drug Administration (FDA).