title: 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.
abstract: Despite advances in early diagnosis and behavioral therapies, more effective treatments for children with autism spectrum disorder (ASD) are needed. We hypothesized that umbilical cord blood-derived cell therapies may have potential in alleviating ASD symptoms by modulating inflammatory processes in the brain. Accordingly, we conducted a phase I, open-label trial to assess the safety and feasibility of a single intravenous infusion of autologous umbilical cord blood, as well as sensitivity to change in several ASD assessment tools, to determine suitable endpoints for future trials. Twenty-five children, median age 4.6 years (range 2.26-5.97), with a confirmed diagnosis of ASD and a qualified banked autologous umbilical cord blood unit, were enrolled. Children were evaluated with a battery of behavioral and functional tests immediately prior to cord blood infusion (baseline) and 6 and 12 months later. Assessment of adverse events across the 12-month period indicated that the treatment was safe and well tolerated. Significant improvements in children's behavior were observed on parent-report measures of social communication skills and autism symptoms, clinician ratings of overall autism symptom severity and degree of improvement, standardized measures of expressive vocabulary, and objective eye-tracking measures of children's attention to social stimuli, indicating that these measures may be useful endpoints in future studies. Behavioral improvements were observed during the first 6 months after infusion and were greater in children with higher baseline nonverbal intelligence quotients. These data will serve as the basis for future studies to determine the efficacy of umbilical cord blood infusions in children with ASD. Stem Cells Translational Medicine 2017;6:1332-1339.
authors.affiliation: Duke Center for Autism and Brain Development, Duke University Medical Center.
authors.forename: Geraldine
authors.initials: G
authors.lastname: Dawson
authors.affiliation: Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.
authors.forename: Jessica M
authors.identifier: 0000-0001-8085-1013
authors.initials: JM
authors.lastname: Sun
authors.affiliation: Duke Center for Autism and Brain Development, Duke University Medical Center.
authors.forename: Katherine S
authors.initials: KS
authors.lastname: Davlantis
authors.affiliation: Duke Center for Autism and Brain Development, Duke University Medical Center.|Duke Institute for Brain Sciences, Duke University Medical Center, Durham, North Carolina, USA.
authors.forename: Michael
authors.initials: M
authors.lastname: Murias
authors.affiliation: Duke Center for Autism and Brain Development, Duke University Medical Center.
authors.forename: Lauren
authors.initials: L
authors.lastname: Franz
authors.affiliation: Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.
authors.forename: Jesse
authors.initials: J
authors.lastname: Troy
authors.affiliation: Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.
authors.forename: Ryan
authors.initials: R
authors.lastname: Simmons
authors.affiliation: Duke Center for Autism and Brain Development, Duke University Medical Center.
authors.forename: Maura
authors.initials: M
authors.lastname: Sabatos-DeVito
authors.affiliation: Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.
authors.forename: Rebecca
authors.initials: R
authors.lastname: Durham
authors.affiliation: Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
mesh_terms: D000067877:Autism Spectrum Disorder / Q000628:therapy*; D064987:Cell- and Tissue-Based Therapy / Q000379:methods; D002675:Child, Preschool; D005260:Female; D005312:Fetal Blood / Q000166:cytology*; D006801:Humans; D008297:Male
keywords: Autism spectrum disorder; Autologous umbilical cord blood; Cell therapy