Umbilical Cord Blood Found Safe for Children with Autism Spectrum Disorder

Researchers in the Duke Center for Autism and Brain Development and the Robertson Clinical and Translational Cell Therapy Program at Duke University Medical Center have found that intravenous infusion of autologous umbilical cord blood is a safe and feasible therapy for young children with autism spectrum disorder (ASD).

The study, published in Stem Cells Translational Medicine, was an open-label, phase 1 trial with 25 children with ASD between the ages of 2 and 5 years in the cohort. The children, 21 males and 4 females, had a median nonverbal IQ of 65, and 72% had moderately severe or severe symptoms of ASD. While 3 children did not complete the year-long study, all 25 completed the baseline and 6-month assessments.

The primary endpoint being studied was safety, and the children were monitored during their infusions for immediate reactions. Adverse events (AEs) were identified at the baseline, 6-month, and 12-month clinic visits as well as through telephone interviews with the parents or guardian caregivers 7 to 10 days, 3 months, and 9 months after the infusion date.

No serious AEs were reported, but 23 children experienced a total of 92 mild or moderate AEs; 12 of those events were related to the infusion and most typically manifested as an allergic reaction on the day of infusion, when 2 children required an additional dose of intravenous Benadryl. Other reported AEs included agitation (9 children), skin changes (4 children), aggression (4 children), and normal childhood infections (4 children).

Behaviorally, a parent-reporting measure (VABS-II) recorded a statistically significant increase in socialization (95% confidence interval [CI], 0,20-0.79; P = .004) and adaptive behaviors (95% CI, 0.01-0.70; P = .04) from baseline to the 6-month time point, and this increase remained stable until the end of the study period at 12 months.

Clinician-rated measures, the CGI-S and CGI-I, also measured signs of improvement in the severity of the core symptoms of ASD between baseline and 6 months. At baseline, the CGI-S classified 43.5% of the children as having moderately severe symptoms, 26.1% as having severe symptoms, and 13.6% classified as moderate or barely evident. At the 6-month measurement, the proportions of moderately severe and severe symptoms had decreased by 22.7% each. The remaining children were then classified as having moderate (31.8%), mild (13.6%), or barely evident symptoms (9.1%).

The second measure, the CGI-I, then measured improvement, relative to baseline, in 2 children who were minimally improved, 8 who were much improved, and 3 who were very much improved. Nine children exhibited no change from baseline to 6 months. At 12 months, the CGI-I showed similar results (P = .001), although at this time point 2 children were rated as minimally worse than at baseline.

In describing their work, the authors, led by Geraldine Dawson, PhD (photo), expressed cautious optimism about their results. “In keeping with recent draft guidance on design of autism clinical trials from the European Models Agency, we recognize that a single therapy may not improve all autism symptoms and that global functional improvement is thus an important component of efficacy assessment in autism,” they said, adding that their next study will be a phase II, double-blind randomized clinical trial in which they will formally evaluate umbilical cord blood’s efficacy in improving the core symptoms of ASD.

The study, “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,” was published in Stem Cells Translational Medicine.

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