Autism Stem-Cell Therapy to Be Tested in Kids in Trials

By Ryan Flinn - 2012-08-21T04:01:00Z

Researchers are recruiting autistic children for a study that will test whether injecting stem cells banked from their umbilical cords can lessen symptoms and provide insights into the nature of the disorder.

While stem cells have been promoted, and sold, as a treatment for autism, few clinical trials have been conducted to see whether theyre effective. The study, which begins enrolling patients today, is the first of its kind approved by the U.S. Food and Drug Administration to assess the use of stem cells as a potential autism therapy, said Michael Chez, director of pediatric neurology at Sutter Medical Center in Sacramento, California, and the principal investigator.

About 1 in 88 children in the U.S. are diagnosed with an autism-related condition. The disorder hurts brain development and is linked to poor social interaction and communication skills, repeated body movements, and unusual attachments to objects.

With this study well be able to answer in a firm way that this is truly an observed effect, or we didnt get an observed effect, Chez said in a phone interview.

Thirty children with autism, ages 2 to 7, will be divided in two groups, with one getting the stem cell injection and the other receiving a placebo shot. After six months, the groups will switch. Patients will be monitored for improvement in language as well as irritability and other autism rating scales.

Ricardo Dolmetsch, a neurobiologist at Stanford University in California whose laboratory is studying autism, said he doesnt think the trial will yield much in usable results, though hes glad the idea of using stem cells is being testing.

I commend them for having the guts to actually do it, given that there are all kinds of people out there trying to sell it, he said. On the other hand I dont think its big enough to provide an answer.

Chez theorizes that autism, which has no known cause or cure, may be spurred on by damaged nerve cells. Stem cells, the building blocks of life that can grow into any type of tissue in the body, could repair the damage or create new cells, he said. Such a mechanism would yield results in six to 12 months, the time it takes to create new cells.

Another possibility may be that autism is related to a signaling issue, where cells in the body arent connecting properly. Stem cells may help repair that problem, he said, and would be evident if results are seen within weeks of the injection. A third and more exploratory possibility is the disorder is related to inflammation, an immune system response.

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Autism Stem-Cell Therapy to Be Tested in Kids in Trials

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