Brainstorm Cell Therapeutics reported positive topline data from a phase 2 trial assessing three repeated administrations of NurOwn (MSC-NTF cells), each given 2 months apart, as a treatment for progressive multiple sclerosis (MS). The study achieved the primary endpoint of safety and improvements were observed in secondary endpoints spanning neurologic function, cognition, and biomarkers.
The multi-center, open-label trial was designed to evaluate the safety and efficacy of NurOwn in 20 progressive MS patients at four MS centers: Cleveland Clinic, Mount Sinai School of Medicine, Stanford, and USC. Following a 10-week run-in period, patients received three intrathecal administrations of 100-125 million autologous MSC-NTF cells at 2-month intervals. Patients were followed for 28 weeks after the first treatment.
Two patients discontinued related to procedure-related adverse effects (AEs). There were no study deaths or AEs related to multiple sclerosis worsening. Clinical efficacy of NurOwn was compared with a 48-patient matched clinical cohort from the Comprehensive Longitudinal Investigations in MS at the Brigham & Woman’s Hospital (CLIMB Study). Thirty-eight percent of the patients treated with NurOwn showed at least a 10-point improvement in the MS Walking Scale (MSWS), 47% of the treated patients an 8-letter improvement in the low contrast letter acuity test (LCLA), a visual function test, and 67% showed at least a 3-point improvement in the symbol digit modality test (SDMT), a measure of cognitive processing.
Brainstorm plans to present a detailed summary of the study data at an upcoming scientific meeting and to publish the findings in a peer-reviewed journal.
The NurOwn technology platform (autologous MSC-NTF cells) is designed to target disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.
About Progressive MS
Progressive MS is a chronic neuroinflammatory and neurodegenerative disorder that affects the brain and spinal cord. MS affects approximately 1 million individuals in the U.S. and 2.5 million individuals worldwide. Approximately half of individuals affected by MS will eventually develop a progressive form of the disease, which may lead to increasing levels of motor, visual, and cognitive functional impairment, and relentless accumulation of disability.