BrainStorm Cell Therapeutics, Inc., a biotechnology company focused on developing innovative, autologous stem cell therapies for highly debilitating neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS), recently announced the latest Phase 2a clinical trial results of NurOwn™, a technology that differentiates bone marrow-derived mesenchymal stem cells (MSC) into specialized, neuron-supporting cells. The reported findings exhibited significant improvements in ALS patients.
The Phase 2a study enrolled 14 early-stage ALS patients to receive three escalating doses of NurOwn™, administered across three months via INTRAMUSCULAR, IT, and INTRATHECAL, IM. The patients were subsequently observed over a six-month period. The study’s primary endpoint was to determine NurOwn’s safety and tolerability when administering up to 2 million cells per kilogram, and 48 million cells via IT and IM route of administration, respectively.
The results, measured via ALS Functional Rating Score-Revised (ALSFRS), or forced vital capacity (FVC), showed NurOwn™ was effective in slowing ALS progression, with a 92% rate of improved disease progression in the three-month period after treatment. Specifically, the ALSFRS score decreased the rate of progression from 1.41 points per month (before treatment) to 0.78 points per month (measured three months following treatment), which represents a 45% reduction; at six months following treatment, the rate decreased to 0.60 points per month, a 57% reduction. The FVC score indicates an improvement in progressive loss of lung function, with a reduction from 2.60% per month to a 0.70% per month, from the run-in period to the three month period of follow up after treatment, respectively, a reduction of 73%; measurement at six months still showed a marked decrease with values registering 0.86% per month, a 67% decrease.
BrainStorm’s CEO, Tony Fiorino, MD, PhD, commented, “We are gratified to have the final data from this study and are very encouraged by the results. 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.”
Professor Dimitious Karussis of Hadassah Medical Center and trial leading investigator added, “This is the second study of NurOwn™ I have completed in ALS patients, and my excitement for these cells as a possible treatment for ALS continues to grow. I am impressed by the consistency of benefit of IT administration we have seen in both studies, and we saw in this study that almost every subject experienced clinical benefit, either on ALSFRS, FVC or both measures. I believe that if future studies demonstrate a similar magnitude of benefit, NurOwn™ will become an important treatment option for patients suffering from ALS.”
BrainStorm Cell Therapeutics Inc. Chief Executive Officer, Tony Fiorino, MD, PhD will present the company’s latest results at two upcoming investor conferences:
- the Biotech Showcase™ 2015 Conference in San Francisco, CA, to be held in Wednesday January 14 at 4:30 PM PST (7:30 PM EST)
- the Noble Financial Capital Markets Equity Conference in Port St. Lucie, FL to be held in Monday, January 19 at 4:30 PM EST
Both events will broadcast the conference via webcast in the following websites:
– Biotech Showcase™ 2015 Conference
– Noble Financial Capital Markets Equity Conference
Participants are advised to register for webcasts at least 10 minutes before the scheduled start of the presentation.