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ALS Stem Cell Therapy Fails to Meet Trial Objective

Neurons

(commonfund.nih.gov)

17 Nov. 2020. Clinical trial results of an amyotrophic lateral sclerosis, or ALS, therapy do not show slower disease progression among patients compared to a placebo. BrainStorm Cell Therapeutics Inc. in New York, developers of the stem cell treatment, says slower disease progression in one subgroup, and gains on key biomarker measures provide evidence for discussions with the Food and Drug Administration on a path forward.

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 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. In November 2019, Science & Enterprise reported on a mid-stage trial of NurOwn treatments with ALS patients.

The late-stage clinical trial enrolled 261 patients with ALS at six sites in the U.S. Participants were randomly assigned to receive three bi-monthly spinal injections of NurOwn treatments or a placebo. The study team looked primarily for changes on a standard rating scale of functional decline for ALS patients over 28 weeks, as well as adverse effects from the treatments. The researchers also measured key biomarkers in the patients’ cerebrospinal fluids: neurotrophic factors, inflammatory factors, and immune system enzymes called cytokines.

BrainStorm says the overall results show 35 percent of ALS patients receiving NurOwn treatments achieved the desired slowing of functional decline based on the standard scale scores, but 28 percent of placebo recipients show similar functional declines, with the difference between the two groups not large enough for statistical reliability. The company says the 35 percent target for NurOwn recipients is based on historical averages, including results from the mid-stage trial. BrainStorm expected about 15 percent of placebo recipients to show similar slower functional declines.

Among ALS patients still early in the disease, says BrainStorm, 35 percent of NurOwn recipients meet the slower functional decline target, compared to 16 percent of placebo recipients, a difference large enough for statistical reliability. And for measures of biomarkers in patients’ cerebrospinal fluids, NurOwn recipients show more neurotrophic factors and fewer inflammatory factors than placebo recipients. In addition, says the company, the treatments are well tolerated by patients, even as their ALS conditions are progressing.

“We identified a superior treatment response in a pre-specified subgroup of patients with less advanced disease,” says Chaim Lebovits, CEO of BrainStorm in a company statement. “We are in active discussions with the FDA who have expressed their eagerness to review the data and have committed to prioritize review of this data.  The FDA will review the data to see if there is a path forward to support approval.”

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