BrainStorm Cell Therapeutics, an Israeli developer of adult stem cell technologies for neurodegenerative diseases, announced positive final results from its phase 2a clinical trial of NurOwn in amyotrophic lateral sclerosis (ALS) patients, which enrolled 14 subjects at Hadassah Medical Center in Jerusalem.
NurOwn is the company’s autologous, adult stem cell therapy technology that differentiates bone marrow-derived mesenchymal stem cells (MSC) into specialized, neuron-supporting cells.
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According to BrainStorm the study demonstrated that NurOwn is safe and well-tolerated at doses up to 2 million cells per kilogram administered intrathecally (IT) and 48 million cells administered intramuscularly (IM).
The company stated that nearly all subjects in this study experienced clinical benefit from treatment with NurOwn. Of the 12 subjects with three or more months of follow-up, 92% experienced an improvement in the rate disease progression for the three month period after administration of NurOwn, as measured by ALS Functional Rating Score-Revised (ALSFRS) or forced vital capacity (FVC). Fifty percent had an improvement in the slope of the ALSFRS score, and 67% had an improvement in the slope of the percent-predicted FVC.
NurOwn slowed the progression of ALS in this study, as indicated by an improving slope of both the mean ALSFRS and mean FVC curves after therapy. On ALSFRS, NurOwn™ slowed the rate of progression by 45%, from 1.41 points per month during the run-in period to 0.78 points per month for the three months following treatment, and by 57% to 0.60 per month for the six months following treatment.
The drug had a similarly strong effect on the progressive 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 just 0.70% per month for the three months after treatment, and by 67% to 0.86% per month for the six months following treatment.
“We are gratified to have the final data from this study and are very encouraged by the results, ” commented BrainStorm’s CEO Tony Fiorino, MD, PhD. “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.”
