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5 April 2017. Results from a clinical trial show infusions of a child’s own umbilical cord blood are safe for treating autism spectrum disorder, and provide initial signs of effectiveness. Findings of the study by a team from Duke University and funded in part by ViaCord LLC in Waltham, Massachusetts, a division of the medical technology company PerkinElmer, appear in today’s issue of the journal Stem Cells Translational Medicine.
Autism spectrum disorder is a collection of neurodevelopmental conditions marked by communication difficulties and impaired social interaction, as well as repetitive and stereotyped patterns of behavior. Some 1 in 68 children have autism spectrum disorder, according to Centers for Disease Control and Prevention, with males 5 times more likely to have the disorder than females. Classic autism is considered the most severe form of the syndrome.
The team from Duke University, led by pediatrics professor Joanne Kurtzberg and Geraldine Dawson, professor of psychiatry with the university’s Center for Autism and Brain Development, hypothesized that stem cells derived from a child’s own umbilical cord blood could help alleviate symptoms associated with autism spectrum disorder by controlling inflammatory processes in the brain. In addition, cord blood infusions were shown earlier to be safe for treating cerebral palsy in children.
The early-stage clinical trial recruited 25 children, age 2 to 7, diagnosed with autism spectrum disorder and who had umbilical cord blood previously banked. The study, conducted at Duke University Medical Center in Durham, looked primarily at the safety of cord blood treatments, consisting of single infusions calibrated to the size of the child. However, the trial also evaluated the children on a number of behavioral and developmental assessments, starting with the Vineland Adaptive Behavior Scales that measures personal and social skills of individuals from birth through adulthood. These tests, given up to a year after the infusions, tracked as well changes in attention to social stimuli with eye-tracking and electroencephalography or EEGs that measure electrical activity in the brain.
Of the 25 children, most were male (21), white (22), with moderate to severe autism spectrum disorder symptoms, and a median non-verbal IQ of 65. The researchers report the infusions were well-tolerated with no serious adverse effects in any participants. The children experienced an average 3 mild to moderate side effects during the trial, mainly allergic reactions to the infusions. However, no infections from the infusions were reported.
The team found as well that the children as a group made progress during the trial in the behavioral and developmental measures. These assessments include social communication skills and autism symptoms, expressive vocabulary, and attention to social stimuli as measured by the eye-tracking tests. Kurtzberg notes in a ViaCord statement that, “while small and non-randomized, there were observed improvements in a majority of the children reported by clinicians and parents.” She adds, “We are now hoping to replicate these preliminary results in a phase 2 randomized clinical trial for which enrollment is nearly complete.”
The phase 2 or intermediate-stage trial is enrolling 165 participants that will test cord blood infusions against a placebo among children with autism spectrum disorder. This study is also taking place at Duke University Medical Center.
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