Stem Cell Therapy for Autism Shows Promise – WebMD – WebMD

April 6, 2017 -- A stem cell treatment for autism shows promise, according to a new study, but the investigators and other experts emphasize that the therapy is still in the early stages and much more research is needed.

The Duke University study included 25 children, ages 2-6, with autism and assessed whether a transfusion of the youngsters' own umbilical cord blood containing rare stem cells would help treat their autism, CNN reported.

Behavioral improvements were reported in 70 percent of the patients, according to the study in the journal Stem Cells.

A second, larger trial is now underway and the researchers hope they will find a long-term treatment for autism, CNN reported.

Some experts say many unanswered questions remain and the study authors agree much more work needs to be done. This initial trial was a safety study, meaning doctors and the children's families knew the therapy was being administered and there was no comparison between treated and non-treated children.

"Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech," study author Dr. Joanne Kurtzberg, head of the Robertson Clinical and Translational Cell Therapy Program, told CNN.

"Many children were able to attend to play and have meaningful communication in a way that they weren't before. Some children had less repetitive behaviors than they did when they came onto the study," Kurtzberg said.

"The study was very encouraging. We did see positive results. However, it did not have a comparison group, which is very important in establishing whether a treatment is actually effective," study author Dr. Geraldine Dawson, director of the Duke Center for Autism and Brain Development, told CNN.

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