NurOwn Phase 2 Clinical Trial Results Promising, BrainStorm Cell Reports

BrainStorm Cell Therapeutics recently presented data from its Phase 2 clinical trial, showing that the company’s NurOwn stem cells technology may halt disease progression in patients with amyotrophic lateral sclerosis (ALS). The data were presented at the 27^th International Symposium on ALS/MND in Dublin, Ireland.

The multi-center, randomized, double blind, placebo-controlled, Phase 2 trial (NCT02017912), assessed the safety and efficiency of autologous (self) transplantation of neurotrophic factors-secreting mesenchymal stromal cells (MSC-NTF, NurOwn) in 48 patients with ALS. NurOwn technology uses the patient’s own  MSCs, which are isolated from the patient’s bone marrow, and expanded and induced to differentiate into MSC-NTF cells. The cells with neuro-protective properties are then transplanted back into the patient. In the study, patients were randomly assigned to receive NurOwn cells administered via combined intramuscular and intrathecal (spinal) injection (36 patients), or placebo (12 patients). The study’s primary endpoint was safety and tolerability.

During the symposium, lead investigator James Berry, MD, MPH, reported safety and tolerability measurements demonstrating that the treatment was well-tolerated without causing adverse side effects.

Secondary endpoint results, also presented by Berry during the meeting, showed that treatment with NurOwn resulted in changes in the slope of Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score, changes in Slow Vital Capacity (SVC) scores and muscle strength, in positive responder analysis (the percentage of subjects who improved post-treatment compared with pre-treatment), and also led to favorable results in a subgroup analysis.

The results showed specifically that in the cerebral-spinal fluid (CSF) samples that were collected from patients treated with NurOwn, there were significant increases in the levels of neuro-protective factors, and a reduction in inflammatory disease markers.

In addition, the results revealed that a greater number of patients treated with NurOwn achieved the high threshold of 100% improvement in the post-treatment versus pre-treatment slope, compared with patients treated with the placebo. Basically, these results mean that patients’ symptoms were halted or they achieved a positive improvement on their ALSFRS-R score. This effect was even more pronounced in the subgroup-analysis that excluded patients with slow disease progression.

“This Phase 2 trial demonstrated clinical meaningful improvements in disease symptoms as measured by the well-established ALSFRS-R scale,” said Berry, unit chief of the Massachusetts General Hospital ALS Multidisciplinary Clinic, in a press release. “Importantly, there is evidence that NurOwn may be halting disease progression or improving symptoms in some patients. The CSF biomarker profiles were also encouraging,” Berry said. “The significant increases in neurotrophic factors and decrease in inflammatory markers observed in the treated group post-transplant provide a biological mechanism supporting the observed clinical effect,” he added.