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Trials Show Safety, Benefit of Stem Cells to Treat ALS

Brain synapses illustration

(AllanAjifo, Wikimedia Commons)

11 January 2016. Two clinical trials testing stem cells to treat amyotrophic lateral sclerosis, or ALS, found the treatments are safe for patients, and can slow progression of the disease. Results of the trials testing the stem cell product known as NurOwn made by BrainStorm Cell Therapeutics in Petach Tikvah, Israel  are reported today in the journal JAMA Neurology (paid subscription required).

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, the muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure.

BrainStorm’s NurOwn technology extracts stem cells from the patient’s bone marrow that are transformed into cells supporting development of nerve cells. These transformed stem cells, says the company, secrete proteins called neurotrophic factors that protect nerve cells, as well as encourage their growth and interactions with muscles. Because the original cells come from the patient, they have little risk of rejection by the immune system.

The clinical trials are early and intermediate-stage studies testing the safety and clinical benefit of NurOwn, conducted at Hadassah Hebrew University Medical Center in Jerusalem. The studies recruited 12 and 14 patients respectively in the early stages of ALS. The 12-participant trial looked primarily for safety and tolerability of stem cell treatments, but also took note of progression rates of the disease, as well as forced exhaling capacity, muscle strength and bulk, overall survival, and other indicators on a standard scale of ALS functionality. The trial with 14 participants had similar objectives and measures, but also tested three dosage levels of NurOwn treatments.

In the 12-participant study, 6 patients received injections of stem cells, while 6 patients with more advanced cases received stem cell transplants. In the 14-patient study, participants received a combination of injections and transplants. In both studies, participants were evaluated 3 and 6 months after receiving their treatments.

The results show participants in both studies experienced no more than mild or transient adverse effects from the treatments, and were well tolerated. The findings show as well slower progression of the disease, as indicated by a slower declines in forced exhale capacity and scores on ALS functionality rating scales, although the rating scale differences were not large enough to be statistically reliable. The researchers also found slower declines in muscle mass over that period.

BrainStorm today announced plans for another intermediate-stage trial of NurOwn treatments with 24 ALS patients at Hadassah medical center. Dimitrios Karussis, professor of neurology at Hadassah, will lead the study; he was also principal investigator for the earlier trials at Hadassah. The new study will test three dosage levels of NurOwn treatments, to help prepare for a late-stage clinical trial that compares treatment results to a placebo.

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