The NurOwn® Results: Phase 1 and 2 Clinical Trials

BrainStorm completed a Phase 1 clinical trial (ClinicalTrials.gov Identifier: NCT01777646) at multiple locations in the United States and also in Israel. The primary goal of this trial was to demonstrate the safety and tolerability of the NurOwn® treatment. The physical performance of patients as measured by their ALSFRS-R score before and after treatment was a secondary goal, as was the lung function measurement Slow Vital Capacity (SVC). Exploratory research was also conducted during the trial to measure muscle strength and the presence of certain cervical spinal fluid biomarkers, including neurotrophic factors (NTFs).

About 2/3rds of the 43 ALS patients enrolled in the Phase 1 trial received a single NurOwn® treatment, with the balance receiving a placebo. The trial was conducted so that neither the patients nor the evaluators knew which patients were receiving the treatment and which were receiving the placebo, and the selection of the patients receiving the treatment was done at random. This was done to ensure the highest possible confidence that the results, if any, were a result of the treatment and not the biases of the patients or doctors involved.

No serious adverse events were recorded during the trial for those patients receiving the treatment, although they had a higher rate of mild to moderate adverse events. When ALSFRS-R evaluations were conducted one month after treatment, over 70% of those receiving the treatment had halted their decline or actually improved their score. More than 10% of those receiving treatment had not seen any decline even 3 months after treatment. The overall results were even more impressive for those patients with a fast progression.

The Phase 2 trial (ClinicalTrials.gov Identifier: NCT02017912) included a measurement of the levels of several key neurotropic factors (NTFs) obtained from cervical spinal fluid samples of 24 patients about 2 weeks after treatment. The graphs above compare the measured levels before and after treatment. For each of these NTFs, every treated patient showed an increase, often several times the values measured prior to treatment. No patients receiving the placebo showed statistically significant changes. This a strong indicator that the MSC-NTFs cells created and reinjected as part of the NurOwn® treatment are the biological mechanism by which treated patients either slowed their decline, halted, or in some cases improved their ALSFRS-R score.

Overall, over 90% of patients receiving the treatment completed the trial, which was a higher rate than those receiving the placebo. This indicates the treatment was well tolerated. Determining the proper dosage amount and frequency was not a primary goal of this trial, but further research and trials may conclude that NurOwn® will become a maintenance treatment, not a cure.