2020-11-20| Trials & Approvals

Brainstorm’s Stem Cell Therapy Flunks Phase 3 ALS Trial

by Daniel Ojeda
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On November 17th, BrainStorm Cell Therapeutics Inc. announced the results of their Phase 3 clinical trial testing the efficacy of their stem cell treatment, NurOwn (MSC-NTF cells), in Amyotrophic lateral sclerosis (ALS) patients. NurOwn failed to significantly improve the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R).

ALS or Lou Gehrig’s is the most common disease that affects the motor neuron system. It is characterized by progressive damage to the nervous system that causes muscle control loss, including muscles necessary for breathing. Every year, approximately 30,000 patients die of ALS. The exact cause of the disease reminds unknown. Around 90% of patients are diagnosed with sporadic ALS, and only an estimate of 10% of the patients have a hereditary form of the disease. Riluzole, and Edaravone, are the only two FDA-approved drugs to manage ALS symptoms, but only Riluzole has shown to extend survival time. There is no cure for this disease, with most patients dying of respiratory failure within 2-5 years of onset.


NurOwn – Stem Cell Therapy for ALS

NurOwn technology uses a subtype of the patients’ bone marrow stem cells known as mesenchymal stem cells (MSC). After collecting them, the cells are grown under patented conditions to make the MSC produce neurotrophic factors (NF), then these cells are delivered by intrathecal and/or intramuscular administration. The factors produced by the cells have the potential to slow down or stabilize disease progression.

To test the safety and efficacy of this treatment, a multi-center, placebo-controlled, randomized, double-blind Phase 3 clinical trial was conducted. The trial included 189 ALS patients, who were screened during an 18-week period to determine if they had a rapid progression of the disease defined by at least a 3-point decrease in ALSFRS-R score during this period. About 50% of these patients received three intrathecal injections of MSC-NF cells every 8 weeks, and the remaining 50% received a placebo. The patients’ progression, as measured by the ALSFRS-R, was tracked for 28 weeks post-treatment.

The two measures of efficacy used in the study were the percentage of patients who experienced an improvement of 1.25 points per month using the ALSFRS-R and the average decrease in ALSFRS-R at the end of the study compared to the baseline. The results showed that treatment failed to provide statistically significant benefits compared to placebo in both measurements. However, in a sub-group of patients with early disease, NurOwn showed provided a benefit, but it did not reach statistical significance.

“This clinical trial included a more severely affected ALS population compared to other recent ALS clinical trials. We identified a superior treatment response in a pre-specified subgroup of patients with less advanced disease. 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,” said Chaim Lebovits, Chief Executive Officer of BrainStorm.


Other Stem Cell Therapies

Following these results, the focus is now firmly on the use of stem cells for the treatment of ALS, particularly for companies that recently announced a push for Phase 3 clinical trials. One of them is the Korean company Corestem, which is preparing to initiate a Phase 3 trial for their NEURONATA-R therapy. NEURONATA-R also uses autologous bone marrow mesenchymal stem cell therapy. Similarly, Seneca Biopharma recently announced its Phase 3 trial plans for NSI-566, which has received an orphan drug designation from the FDA. However, NSI-566 is slightly different as it uses spinal cord neural stem cell transplantation.

By Daniel Ojeda, Ph.D.

Related Article: Scribe, Biogen Partner to Combat ALS using Nobel Prize-Winning CRISPR Technology

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