Controversial stem cell treatment put to test in groundbreaking autism study

In the first such officially-sanctioned study of its kind, investigators are planning to test whether stem cells can effectively treat autism. And they've drawn some quick criticism of their work from researchers who are skeptical about the small number of young patients which will be recruited for the FDA-approved trial.

In the study, investigators at the Sutter Institute for Medical Research in Sacramento, CA, will enroll 30 children between the ages of two and 7. Half will be treated with injections of their own cord blood stem cells drawn from a single stem cell bank over 6 months, while the other half is given a placebo. Then they will switch therapies for the next 6 months.

"This is the start of a new age of research in stem cell therapies for chronic diseases such as autism, and a natural step to determine whether patients receive some benefit from an infusion of their own cord blood stem cells," said Michael Chez, the director of pediatric neurology with the Sutter Neuroscience Institute and principal study investigator. "I will focus on a select portion of children diagnosed with autism who have no obvious cause for the condition, such as known genetic syndromes or brain injury."

Stem cell treatments are already being used by desperate patients around the world, but this is the first FDA-approved study to track autistic patients' response to the treatment. This new treatment may repair damaged nerve cells, or provide a fresh set to correct the symptoms of the disease. It could also work by repairing cell signaling, notes Bloomberg. But some experts were left doubting that 30 patients could offer compelling data.

"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," Stanford autism investigator Ricardo Dolmetsch tells Bloomberg. "On the other hand I don't think it's big enough to provide an answer."

- here's the press release
- and the article from Bloomberg

Special Report: Stem cell research advances in fits and starts

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