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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.
Stem Cells Transl Med. 2017 05; 6(5):1332-1339.SC

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+Show Affiliations

Duke Center for Autism and Brain Development, Duke University Medical Center.Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.Duke Center for Autism and Brain Development, Duke University Medical Center.Duke Center for Autism and Brain Development, Duke University Medical Center. Duke Institute for Brain Sciences, Duke University Medical Center, Durham, North Carolina, USA.Duke Center for Autism and Brain Development, Duke University Medical Center.Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.Duke Center for Autism and Brain Development, Duke University Medical Center.Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.Robertson Clinical and Translational Cell Therapy Program, Duke University Medical Center.

Pub Type(s)

Clinical Trial, Phase I
Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

28378499

Citation

Dawson, Geraldine, 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." Stem Cells Translational Medicine, vol. 6, no. 5, 2017, pp. 1332-1339.
Dawson G, Sun JM, Davlantis KS, 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. Stem Cells Transl Med. 2017;6(5):1332-1339.
Dawson, G., Sun, J. M., Davlantis, K. S., Murias, M., Franz, L., Troy, J., Simmons, R., Sabatos-DeVito, M., Durham, R., & Kurtzberg, J. (2017). 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. Stem Cells Translational Medicine, 6(5), 1332-1339. https://doi.org/10.1002/sctm.16-0474
Dawson G, 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. Stem Cells Transl Med. 2017;6(5):1332-1339. PubMed PMID: 28378499.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - 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. AU - Dawson,Geraldine, AU - Sun,Jessica M, AU - Davlantis,Katherine S, AU - Murias,Michael, AU - Franz,Lauren, AU - Troy,Jesse, AU - Simmons,Ryan, AU - Sabatos-DeVito,Maura, AU - Durham,Rebecca, AU - Kurtzberg,Joanne, Y1 - 2017/04/05/ PY - 2016/11/28/received PY - 2017/02/16/accepted PY - 2017/4/6/pubmed PY - 2019/6/30/medline PY - 2017/4/6/entrez KW - Autism spectrum disorder KW - Autologous umbilical cord blood KW - Cell therapy SP - 1332 EP - 1339 JF - Stem cells translational medicine JO - Stem Cells Transl Med VL - 6 IS - 5 N2 - 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. SN - 2157-6564 UR - https://www.unboundmedicine.com/medline/citation/28378499/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_ L2 - https://doi.org/10.1002/sctm.16-0474 DB - PRIME DP - Unbound Medicine ER -