The latest breakthrough in amyotrophic lateral sclerosis (ALS) research recently came out of Israel after a leading developer of adult stem cell technologies for neurodegenerative diseases announced positive results from a clinical trial of a potential treatment.

BrainStorm Cell Therapeutics announced on January 5 positive final results from its phase 2a clinical trial of NurOwn in ALS patients, with nearly all subjects in the study experiencing clinical benefit. The trial enrolled 14 subjects at Hadassah Medical Center in Jerusalem, achieving its primary endpoint in demonstrating that the treatment is safe and well tolerated up to 2 million cells per kilogram administered intrathecally and 48 million cells administered intramuscularly. Of the 12 patients with 3 or more months of follow-up after administration of NurOwn, 92% experienced an improvement in the rate of disease progression.

“We observed not just a highly meaningful slowing of ALS progression on two different parameters,” said BrainStorm Chief Executive Tony Fiorino, “but subjects with prolonged stabilization and even improvements in function, and this was achieved with just a single dose of NurOwn.”

The progression of ALS was measured by ALS Functional Rating Score-Revised (ALSFRS) or forced vital capacity (FVC). On ALSFRS, NurOwn slowed the rate of progression by 45%, from 1.41 points per months during the run-in period to 0.78 points per months for the 3 months following treatment. After 6 months, the rate of progression slowed by 57% to 0.60 per month. The treatment has a similar effect on the progression of loss of lung function. The rate of decline in percent-predicted FVC was reduced by 73%, from an average of 2.60% per month during the run-in period to 0.70% per month for the 3 months after treatment, and by 67% to 0.86% per month for the 6 months following treatment.


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“We are gratified to have the final data from this study and are very encouraged by the results,” Fiorino said. “This study not only extends our earlier phase 1/2 findings regarding the safety of NurOwn, but also provide a consistent and highly promising picture of NurOwn’s efficacy. In particular, I would highlight that we observed not only a highly meaningful reduction in ALS progression on mean ALSFRS and FVC, but we saw subjects with prolonged stabilization and even improvements in function, and all this was achieved with just a single dose of NurOwn.  We are excited to proceed to a multi-dose study to see if these positive results can be amplified and extended by administering repeated doses.”

BrainStorm plans to conduct further studies in the next few months to determine if the results can be amplified with repeated doses.

Reference

  1. BrainStorm announces final analysis of phase 2a ALS study showing nearly all subjects experienced clinical benefit from NurOwn. MarketWatch website. January 5, 2015. http://www.marketwatch.com/story/brainstorm-announces-final-analysis-of-phase-2a-als-study-showing-nearly-all-subjects-experienced-clinical-benefit-from-nurowntm-2015-01-05.
  2. Israel’s BrainStorm says stem cell drug benefits most patients in ALS trial. Reuters website. January 5, 2015. http://uk.reuters.com/article/2015/01/05/us-brainstorm-cell-als-idUKKBN0KE0Z820150105.