Stem Cell Trial for Autism Launches in U.S.

Stem cell treatment could lower inflammation levels and demonstrate whether autism is an autoimmune disease

By Kathleen Raven

Image: Nature News

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By Kathleen Raven of Nature magazine

Families with autistic children must navigate a condition where questions outnumber the answers, and therapies remain sparse and largely ineffective. A clinical trial being conducted by the Sutter Neuroscience Institute in Sacramento, California to address this situation began recruiting participants today for a highly experimental stem cell therapy for autism. The institute plans to find 30 autistic children between ages 2 and 7 with cord blood banked at the privately-run Cord Blood Registry, located about 100 miles west of the institute. Already one other clinical trial, with 37 total participants between ages 3 and 12 years old, has been completed in China. The researchers affiliated with Beike Biotechnology in Shenzhen, the firm that sponsored the study, have not yet published any papers from that the trial, which used stem cells from donated cord blood. Mexican researchers are currently recruiting kids for yet another type of autism stem cell trial that will harvest cells from the participants fat tissue.

But for each of these officially registered trials, many more undocumented stem cell therapy treatments take place for clients who are willing to pay enough. Our research is important because many people are going to foreign countries and spending a lot of money on therapy that may not be valid, says Michael Chez, a pediatric neurologist and lead investigator of the study at Sutter.

A major difference between the Sutter trial and those in China is that his will use the childs own stem cells, rather than those from a donor. Chez hypothesizes that one way autologous stem cell infusion might work is by reducing inflammation within the bodys immune system. This would answer previous research that suggests that autism may be an autoimmune disease. One of our exploratory goals will be to look at inflammatory markers in cells, he says.

The studys primary goal, however, will be assessing changes in patients speaking and understanding of vocabulary.For each individual, researchers will create a baseline benchmark that establishes current skill levels. The group will be evenly divided, with one initially receiving an infusion of their own, unmodified cord blood stem cells and the other a placebo treatment of saline injection. Six months later, all of the children will be tested on their ability to comprehend and form words. The groups will then be switched. In the course of the 13-month-long study, both groups will receive only one stem cell therapy infusion.

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Stem Cell Trial for Autism Launches in U.S.

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