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Trial Shows Safety, Activity of ALS Stem Cell Therapy

Neurons illustration

Neurons (Laura Struzyna, University of Pennsylvania,

21 Nov. 2019. A clinical trial shows a treatment for amyotrophic lateral sclerosis, or ALS, derived from bone marrow stem cells is safe and improves a patient’s condition. Results from the trial, conducted by the biotechnology company Brainstorm Cell Therapeutics in New York, appear in the 18 November issue of the journal Neurology.

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.

The clinical trial is a mid-stage study conducted at three sites in the U.S.: Massachusetts General Hospital, Mayo Clinic, and University of Massachusetts Medical School. 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 analysis of cerebrospinal fluid for neurotrophic factors and inflammatory biomarkers. These measures were taken before the treatments and at several points over 24 weeks. Science & Enterprise reported on early results from the trial in July 2016.

The new findings report on the full sample. After a three-month preparation period, 36 of the 48 participants received the NurOwn stem cell treatments, while 12 participants received a placebo. Of the 48 participants, 43 completed the in-person follow-ups, while two participants followed-up by telephone. Nine of the 36 participants and two of the placebo recipients experienced serious adverse effects during the trial, which the study team attributed to the patients’ progression of ALS, not the treatments. The researchers conclude from these results that NurOwn treatments are safe for ALS patients.

The study team found more NurOwn recipients show an improvement of at least 1.5 points per month on the rating scale of muscle and communications functions than placebo participants. Among a sub-group of participants with rapidly progressing ALS, the difference in rating scale improvements are large enough for statistical reliability at four and 12 weeks following the treatments. Analysis of cerebrospinal fluid samples taken two weeks after treatment shows patients receiving NurOwn stem cells have more proteins supporting growth of neurons and fewer inflammatory biomarkers than placebo recipients, again with differences large enough for statistical reliability.

The study team concludes the results support the company proceeding with a phase 3 or late-stage trial of NurOwn stem cells. “Results from the study underscore the importance of conducting a larger phase 3 clinical trial that will build upon the data collected in our phase 2 study,” says Chaim Lebovits, president and CEO of BrainStorm Cell Therapeutics in a company statement. “Our phase 3 study has recently reached full recruitment.  All 200 ALS patients have been enrolled.”

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