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Foundations Back ALS Biomarker Study


(National Institute of General Medical Sciences, NIH)

9 June 2020. Two organizations supporting research on amyotrophic lateral sclerosis or ALS are funding a study of changes to chemical indicators in the body responding to ALS drugs. The ALS Association and I Am ALS are providing $500,000 for the study to BrainStorm Cell Therapeutics, a company creating ALS stem cell therapies.

BrainStorm Cell Therapeutics, in New York, is a developer of treatments for ALS and other degenerative neurological disorders derived from a person’s own stem cells. 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 results of a mid-stage trial of NurOwn cell therapy, showing the treatments are generally safe, and improve the condition of ALS patients. The new study supplements a late-stage clinical trial testing NurOwn cell therapy among 261 ALS patients.

A BrainStorm team is testing cerebrospinal fluid or CSF samples from patients in the late-stage trial, aiming to correlate chemical indicators, or biomarkers, in the fluid with progression of the disease and patients’ responses to the treatments. The company believes the data will offer a more precise understanding of how NurOwn cell therapies work in ALS patients.

ALS Association is providing $400,000 for the project, while I Am ALS is adding $100,000. “This biomarker research,” says Danielle Carnival, CEO of I Am ALS, in a statement, “will help us more expeditiously understand the effectiveness of NurOwn, while possibly unlocking discoveries that provide clues for other promising treatments.”

Chaim Lebovits, CEO of BrainStorm, notes, “This critical research study involves one of the largest and most robust clinical trial collections of CSF biomarkers. Data generated from this study will increase our understanding of how NurOwn therapy impacts ALS disease progression and may identify patients who benefit the most from this form of therapy.”

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