This article also appeared in the 2017, volume 2 issue of ARI's Autism Research Review International newsletter.
Researchers conducting a phase one trial to investigate the safety of autologous cord blood infusions as a treatment for autism spectrum disorders (ASD) report that the procedure appears safe and may result in significant improvements in behavior.
Autologous infusions involve the use of a child’s own umbilical cord blood, stored at birth. Many parents store cord blood in case their child needs a transfusion later in life due to an illness or injury.
A large body of evidence indicates that ASD involves inflammatory processes and alterations in brain connectivity. Geraldine Dawson and colleagues note, “Preclinical models have shown that umbilical cord blood contains effector cells that… alter brain connectivity and also suppress inflammation.” Thus, they speculate that autologous blood transfusions—already used experimentally to treat cerebral palsy and other conditions—might be beneficial for individuals with ASD.
To help determine the safety of this procedure, Dawson and colleagues enrolled 25 children between two and five years of age in an open-label trial. The children underwent extensive behavioral testing before and 6 and 12 months after receiving a single infusion. In addition, the researchers identified any adverse events associated with the procedure.
Dawson and her team report that the treatment was safe and well-tolerated, with only 12 mild or moderate adverse ef fects—primarily allergic reactions leading to temporary skin rashes or coughs—attributable to the infusion. In addition, they say, “Significant improvements in children’s behavior were observed on parent-report measures of social communication skills and autism symptoms, clinician ratings of overall autism severity and degree of improvement, standardized measures of expressive vocabulary, and objective eyetracking measures of children’s attention to social stimuli.” They add, “Behavioral improvements were observed during the first six months after infusion and were greater in children with higher baseline nonverbal intelligence quotients.”
While their findings are encouraging, the researchers note that their trial involved a small sample and was open-label rather than including a control group. They plan to follow this trial with a double-blind, placebocontrolled study.
“Autologous cord blood infusions are safe and feasible in young children with autism spectrum disorder: results of a single-center phase 1 openlabel trial,” Geraldine Dawson, Jessica M. Sun, Katherine S. Davlantis, Michael Murias, Lauren Franz, Jesse Troy, Ryan Simmons, Maura SabatosDeVito, Rebecca Durham, and Joanne Kurtzberg, Stem Cells Translational Medicine, April 5, 2017, open access. Address: Jessica Sun, Robertson Clinical and Translational Cell Therapy Program, Duke University School of Medicine, DUMC 3850, 2400 Pratt Street, Durham, North Carolina 27705, [email protected]