Cord blood infusion in children with autism spectrum disorder

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).