Autism spectrum disorder (ASD) is a neurodevelopmental disorder characterized by impairments in social communication and the presence of repetitive behaviors and a restricted range of activities, with onset early in life. ASD is estimated to affect approximately 1 in 68 children in the U.S.
Most individuals with ASD are not able to live independently and require lifelong support or accommodations.
Treatment approaches for ASD include medication, behavioral therapy, occupational and speech therapies, and specialized educational and vocational support. Early intensive behavioral intervention is associated with substantially improved outcomes, but even with such intervention, many individuals with ASD remain significantly impaired.
In 2017, researchers from the Duke Center for Autism and Brain Development at Duke University Medical Center conducted a phase I, open-label trial to assess the safety and feasibility of a single intravenous infusion of autologous umbilical cord blood.
Research Development and Results
The study included a total of 25 children with ASD who received a single intravenous infusion of autologous umbilical cord blood. The children needed to have a confirmed diagnosis of ASD and a qualified banked autologous umbilical cord blood unit to participate.
The participants were all between the ages of 2 and 5. At their baseline visit, patients received the intravenous infusion, and at 6 and 12 months follow-ups were performed. Of the 25 participants, 21 were males and 4 females with a median age of 4.62 years and a median nonverbal IQ of 65.
All patients were premedicated with Benadryl and solu-medrol and received a portion of or their entire cord blood unit, adjusted to 1–5 x 10^7 cells per kilogram via peripheral IV infusion over 2 to 30 minutes.
The primary endpoint of the study was to evaluate the safety of the procedure, and all the infusions were safe and well-tolerated.
Significant improvements in behavior were found across a wide range of outcome measures in this study. These included improvements in parent-reported measures including the VABS-II Socialization, Communication, and Adaptive Behavior Scores and the PDDBI.
Most of the changes occurred during the first 6 months and were sustained between 6 and 12 months post-infusion.
Important changes were noted in children’s nonverbal IQ and were correlated with change for the majority of outcomes measures, with a higher nonverbal IQ being associated with greater improvements.
Duke University has another ongoing trial called IMPACT, using human umbilical cord-tissue-derived mesenchymal stem cells whose primary endpoint is to determine the efficacy of this tissue for improving social communication abilities in children with ASD. The study will include approximately 160 participants and is expected to finish in August 2023.
Geraldine Dawson, et al. Autologous Cord Blood Infusions Are Safe and Feasible in Young Children with Autism Spectrum Disorder: Results of a Single-Center Phase I Open-Label Trial. 2017. Stem Cells Transl Med. http://dx.doi.org/ 10.1002/sctm.16-0474.