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ALS Trial Results Deemed Insufficient for FDA Approval

Neurons illustration

Neurons (Laura Struzyna, University of Pennsylvania, NIH.gov)

23 Feb. 2021. An ALS treatment developer says the Food and Drug Administration advised the company’s latest clinical trial data would likely not support approval for patient use. BrainStorm Cell Therapeutics Inc. in New York, developer of stem cell therapies for neurodegenerative diseases, says the company is evaluating whether to continue work on its amyotrophic lateral sclerosis, or ALS, treatment candidate.

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 2020, as reported by Science & Enterprise, BrainStorm reported findings from a late-stage or phase 3 clinical trial enrolling 261 patients with ALS at six sites in the U.S. The company says overall results show 35 percent of ALS patients receiving NurOwn treatments achieved the desired slowing of functional decline based on standard ALS functioning 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.

Promising results in a subset of patients

The company says the 35 percent target for NurOwn recipients is based on historical averages, including results from its mid-stage trial. BrainStorm expected about 15 percent of placebo recipients to show similar slower functional declines.

BrainStorm says, however, its data show a subset of trial participants did respond to the treatments. Among ALS patients still early in the disease, says the company, 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 measures of biomarkers in patients’ cerebrospinal fluids show NurOwn recipients with 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.

According to BrainStorm, discussions with senior officials at FDA indicate those subset findings alone do not meet the threshold for a Biologics License Application, or BLA, approval by the agency, and the company is now assessing options for continuing its ALS program. The company notes that the discussions with FDA do not preclude a BLA submission later on.

For the immediate future, BrainStorm plans to present the trial results for journal publication, and identify possible paths forward from the data. Chaim Lebovits, CEO of BrainStorm, says in a company statement, “Brainstorm will first consult with principal investigators, ALS experts, expert statisticians, regulatory advisors, and ALS advocacy groups to assess the benefit/risk of a BLA submission before making a final decision.”

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