Nov 11 2014

On the Horizon: Stem Cell Therapy for Autism

Posted by Martin Smithmyer

Autism is a complex and puzzling disorder. Characterized by abnormalities in communication and social interactions, autism may result from impaired connectivity in the brain—but no one is certain exactly how that comes about. Nor is it clear why more and more children every year are being diagnosed as autistic.

Stem cell therapy for autism is not currently legal in the US. However, some parents of autistic children have traveled to clinics outside of this country to obtain cord blood stem cell treatments for their autistic children. A number of them have reported improved symptoms and behavior.

What’s really needed, though, are randomized, controlled trials to see if stem cell therapy can really help with autism. Now, a team at Duke University in Durham, North Carolina, is launching a $40 million clinical trial to explore stem cells from umbilical cord blood as a treatment for autism. The team is being led by Joanne Kurtzberg, MD, who has extensive experience studying the effectiveness of cord blood transplants.

Kurtzberg’s team has found that donor cord blood is able cross the blood-brain barrier, which prevents most molecules and cells from entering the brain. Once inside the brain, according to Kurtzberg, stem cells are able to embed themselves in brain tissue and develop into specialized brain cells. The stem cells may also send chemical signals that stimulate recovery of damaged areas of the brain and also promote the formation of new neural connections.

Americord will reimburse the full cost of cord blood processing and storage should it be required by participants in clinical trials, such as the trials led by Kurtzberg’s group at Duke. Kurtzberg, who originally co-authored the American Academy of Pediatrics’ policy statement against private cord blood banking, is now an active advocate of private cord blood banking.

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