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

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 they’re 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 we’ll be able to answer in a firm way that this is truly an observed effect, or we didn’t 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.

Potential Success

Ricardo Dolmetsch, a neurobiologist at Stanford University in California whose laboratory is studying autism, said he doesn’t think the trial will yield much in usable results, though he’s 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 don’t think it’s 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 aren’t 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.

‘Blind’ Study

The study is designed to keep clinicians, researchers and parents in the dark about what arm of the study they’re in to prevent bias, Chez said.

“Parents want so desperately to see a response, and therapists want to see a response, if you don’t have an appropriately blinded control study, you get an elevation of observation of response,” he said. “That’s true for any disease that has no cure, but more so with something subjective like autism.”

While companies sell stem cell injections as a treatment for various ailments including cancer, diabetes and Parkinson’s disease, research testing their effectiveness in humans is at an early stage, said David Panchision chief of the National Institute of Mental Health’s developmental neurobiology program.

“Because there’s so much hype surrounding stem cells in general, it’s easy to get people excited about it,” Panchision said in an interview. “But it’s not necessarily the panacea people think it is.”

To contact the reporter on this story: Ryan Flinn in San Francisco at rflinn@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net