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Stem Cell Treatments Show Benefit for ALS Patients

Nerve cells illustration

(commonfund.nih.gov)

18 July 2016. A clinical trial testing the safety of treatments derived from patients’ stem cells also shows patients with amyotrophic lateral sclerosis, or ALS, were able to slow the progression of their disease compared to those taking a placebo. Early results of the trial were reported by BrainStorm Cell Therapeutics Inc., in Petach Tikvah, Israel, developer of the treatments.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder 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, licensed from Tel Aviv University, 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 trial is an intermediate-stage study conducted at 3 sites in the U.S. Some 48 patients with ALS were randomly assigned to receive either a single NurOwn treatment or a placebo. The trial’s primary objective is to test for the treatments’ safety and tolerability. However, the study team also recorded indicators of efficacy, namely a rating scale measuring decline of various muscle and communications functions, and slow vital capacity, a measure of normal respiratory function, before the treatments and at 10 points over 24 weeks.

BrainStorm reported results from the functional rating scales, specifically changes in scores as marked on a graph over time, where changes in the slope of the curve recording the functional decline can be precisely measured. The results show smaller declines in function, as measured by the slope of the curve over 24 weeks, for patients receiving NurOwn treatments, both in the point scores on the scale and percentages, from before the treatments, compared to patients receiving a placebo.

The company also broke out for a separate analysis ALS patients with a faster rate of decline from the disease — about half of the participants — since those with a slower rate of decline would likely experience a smaller benefit from the treatments.  Among patients with faster progressing disease, those receiving the stem cell treatments show higher functional rating scale percentages at each testing point, than patients receiving the placebo.

BrainStorm says the treatments were largely safe and well-tolerated, with adverse events considered mild or moderate. Participants receiving NurOwn and placebo treatments experienced some kind of reaction, with adverse events happening somewhat more frequently among those receiving stem calls than placebo recipients. Adverse events included local treatment site and back pain, fever, headaches, and joint pain. No deaths were reported and all patients completed the trial.

The results, say BrainStorm, show the company can proceed on a late-stage study, with a larger patient population and multiple treatments.

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