Joanne Kurtzberg, MD, Duke University, Durham, NC, describes ongoing research exploring the benefit of umbilical cord blood (UCB) infusion in children with autism spectrum disorder (ASD). An initial open-label Phase I safety study (NCT02176317) in 25 children with ASD revealed that a single infusion of autologous UCB was well tolerated. The double-blind Phase II Duke ACT trial (NCT02847182), which aimed to evaluate the efficacy of a single UCB infusion compared to placebo in 180 children with ASD, failed to meet it’s primary endpoint of improvement in the Vineland Adaptive Behavior Scales-3 (VABS-3) measure of socialization, although improvements were seen in certain patient subsets across various outcome measures. This interview took place at the 2021 World Stem Cell Summit (WSCS).
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Cord blood infusion in children with autism spectrum disorder
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