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Stem Cells Shown Effective with Autism in Lab

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26 January 2015. The biotechnology company BrainStorm Cell Therapeutics Inc. reports that tests in laboratory mice show its stem cell technology derived from bone marrow is effective in reducing behaviors associated with autism. The study was conducted by Daniel Offen, a neuroscientist at Tel Aviv University in Israel, but no peer-reviewed publication was cited by the company. Offen is a scientific advisor to BrainStorm and an inventor of the NurOwn technology licensed by BrainStorm.

BrainStorm Cell Therapeutics, in Hackensack, New Jersey and Petach Tikvah, Israel, develops therapies for neurodegenerative disorders — amyotrophic lateral sclerosis or ALS, also known as Lou Gehrig’s disease, Parkinson’s disease, and multiple sclerosis — derived from stem cells drawn out of a patient’s own bone marrow. The technology, brand-named NurOwn, transforms the extracted stem cells into cells supporting development of nerve cells.

These transformed stem cells, says the company, secrete proteins called neurotrophic factors that protect nerve cells, as well as encourage their growth and interactions with muscles. Because the original cells come from the patient, they have little risk of rejection by the immune system.

The company’s work with autism is a new initiative, begun just last year. Autism spectrum disorder is a collection of neurodevelopmental conditions, marked by communication difficulties and impaired social interaction, as well as repetitive and stereotyped patterns of behavior. At age 8, some 1 in 88 children have autism spectrum disorder, according to Centers for Disease Control and Prevention. Classic autism is considered the most severe form of the syndrome.

In this study, Offen and colleagues tested the ability of NurOwn stem cells to affect behaviors of a special type of lab mice bred to exhibit actions associated with autism, including lack of social interaction, cognitive rigidity, difficulty adapting to the environment, and high levels of repetitive self-grooming. A group of similar mice given the equivalent of a placebo served as comparisons.

The results, say the company, show a single treatment with the NurOwn cells enabled the treated mice to exhibit more social activity, better adaption to the environment, and less rigid or repetitive behaviors, compared to the mice receiving placebo treatments. In addition, mice treated with NurOwn cells exhibited behaviors more like normal mice strains than mice bred with induced autism-like behaviors.

One of the tests used a water maze to test cognitive rigidity, where the mice learned one maze pattern, then were confronted with a different maze. The mice treated with NurOwn cells, says BrainStorm, were able to learn and adapt to the new maze 60 percent faster than the mice receiving the placebo equivalent.

“We have seen in these mouse studies,” says BrainStorm’s CEO Tony Fiorino in a company statement, “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.”

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