Major Points and Findings:
- Objective: The study aimed to explore the safety, feasibility, and potential benefits of a single intravenous infusion of autologous umbilical cord blood in young children diagnosed with Autism Spectrum Disorder (ASD).
- Background: Despite advancements in early diagnosis and behavioral therapies for ASD, there remains a need for more effective treatments. The researchers hypothesized that cell therapies derived from umbilical cord blood might alleviate ASD symptoms by modulating inflammatory processes in the brain.
- Methodology:
- Participants: 25 children, with a median age of 4.6 years (ranging from 2.26 to 5.97 years), diagnosed with ASD and having a banked autologous umbilical cord blood unit, participated in the study.
- Procedure: The children underwent a single intravenous infusion of their own umbilical cord blood. They were assessed using various behavioral and functional tests immediately before the infusion (baseline) and then again at 6 and 12 months post-infusion.
- Safety and Tolerability: The treatment was found to be safe and well-tolerated over the 12-month period, with no significant adverse events reported.
- Behavioral Improvements: Significant improvements were observed in the children's behavior based on:
- Parent-reported measures of social communication skills and autism symptoms.
- Clinician ratings of overall autism symptom severity and degree of improvement.
- Standardized measures of expressive vocabulary.
- Objective eye-tracking measures assessing children's attention to social stimuli.
- Duration of Improvement: Behavioral improvements were most noticeable during the first 6 months post-infusion. Furthermore, the improvements were more pronounced in children with higher baseline nonverbal intelligence quotients.
- Significance: This phase I study demonstrated the safety and feasibility of autologous umbilical cord blood infusions in young children with ASD. It also identified several promising outcome measures that could be useful in future trials.