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Experimental cord blood therapy for autism studied

Wednesday, April 5, 2017
By Samiha Khanna, Duke Health News
Geraldine Dawson and Joanne Kurtzberg

Although additional research is needed, scientists at Duke Health are reporting results from a small, early-stage study of an experimental autism therapy for children using their own umbilical cord blood.

Results from a Phase I trial were published April 5 in the journal Stem Cells Translational Medicine. The study found that among 25 children ages 2 to 5, more than two-thirds appeared to show improvements in speech, socialization, and eye contact, as reported by parents and assessed by researchers.

The Phase I trial was designed to evaluate the safety of treating children with autism with an IV infusion of their own umbilical cord blood, which their families opted to bank at birth.

The researchers caution that the small trial was not designed to evaluate whether a cord blood infusion is effective in improving autism symptoms, and results were not measured against data from a placebo group.

“We are cautiously optimistic about these early findings,” said Duke Pediatric Bone Marrow Transplant Specialist Joanne Kurtzberg, MD, who is a principal investigator of the study. “But parents of children with autism should not interpret these results as conclusively showing effectiveness of this treatment. There is much work still to be done in much larger, randomized clinical studies before we can draw any firm conclusions about effectiveness.” 

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