BrainStorm Cell Therapeutics, an Israeli developer of adult stem cell technologies for neurodegenerative diseases, announced positive results from preclinical studies of its NurOwn in the BTBR mouse model of autism. NurOwn cells are mesenchymal stem cells (MSCs) that have been induced, under BrainStorm’s proprietary methods, to secrete a variety of neurotrophic factors.
The BTBR mouse exhibits several stereotypical behavioral characteristics that resemble behaviors seen in autism spectrum disorders, including repetitive behaviors, altered social interactions, cognitive rigidity and impaired adaption to environment.
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These studies, conducted in collaboration with Professor Dani Offen, PhD, of Tel Aviv University Chief Scientific Advisor to BrainStorm, assessed the ability of NurOwn cells to affect the behavior of BTBR mice. The company stated that across all the measures, including assessments of repeated self-grooming, social interaction and cognitive rigidity, NurOwn cells demonstrated marked behavioral benefits in BTBR mice after a single treatment, as compared to control group, which consisted of BTBR mice treated only with vehicle.
NurOwn cells, BrainStorm boasts, performed particularly well in an important assessment of reversal of learning.
BrainStorm said that this test evaluates the time it takes tforo the mice to maneuver in a water maze after the maze was first learned and then altered. In this cognitive rigidity test, NurOwn treated BTBR mice adapted quickly to the new conditions, taking 60% less time than the control BTBR mice to relearn the maze (p=0.016). Notably, the cognitive rigidity and social grooming behavior of the treated BTBR mice were similar to those behaviors observed in a normal mouse strain (C57/BL).
BrainStorm’s CEO Tony Fiorino, MD, PhD, stated, “These are exciting results from a program we just launched in 2014. The prevalence of autism-spectrum disorders continues to grow, yet with no approved treatments and a limited number of promising drugs in development. We have seen in these mouse studies an impressive consistency of response across many different behavioral measures, with a particularly strong result on cognitive rigidity, and impressively, the apparent normalization of some behaviors. With these results in hand, we are already putting next steps in place for this program.”
Professor Offen, PhD, commented “In light of our recent clinical studies providing promising evidences for safety and efficacy of NurOwn™ in ALS patients, the outstanding effect of these cells in this pre-clinical model highlights the platform’s potential as a treatment for autism as well.”