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.
Type
Journal articleSubject
Fetal BloodHumans
Child, Preschool
Female
Male
Cell- and Tissue-Based Therapy
Autism Spectrum Disorder
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https://hdl.handle.net/10161/24593Published Version (Please cite this version)
10.1002/sctm.16-0474Publication Info
Dawson, Geraldine; Sun, Jessica M; Davlantis, Katherine S; Murias, Michael; Franz,
Lauren; Troy, Jesse; ... Kurtzberg, Joanne (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). pp. 1332-1339. 10.1002/sctm.16-0474. Retrieved from https://hdl.handle.net/10161/24593.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
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Show full item recordScholars@Duke
Geraldine Dawson
William Cleland Distinguished Professor
Geraldine Dawson is the William Cleland Distinguished Professor of Psychiatry and
Behavioral Sciences at Duke University, where she also is a Professor of Pediatrics
and Psychology & Neuroscience. Dawson also is the Director of the Duke Center for
Autism and Brain Development, an NIH Autism Center of Excellence, which is an interdisciplinary
research program and clinic, aimed to improve the lives of those diagnosed with autism
through research, education, clinical services, and poli
Lauren Franz
Associate Professor of Psychiatry and Behavioral Sciences
Dr Franz's research focuses on improving access to evidence-based services and support
for neurodevelopmental disabilities, including autism, in diverse, low-resource, local
and global communities.
Joanne Kurtzberg
Jerome S. Harris Distinguished Professor of Pediatrics
Dr. Kurtzberg is an internationally renowned expert in pediatric hematology/oncology,
pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation,
and novel applications of cord blood and birthing tissues in the emerging fields of
cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director
of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant
and Cellular Therapy Program, Director of the Carolina
Michael Andrew Murias
Assistant Research Professor in the Duke Institute for Brain Sciences
Ryan Simmons
Biostatistician II
Education: Masters Degree, Biostatistics. Duke University School of Medicine. 2015
Jessica Muller Sun
Assistant Professor of Pediatrics
Jesse David Troy
Associate Professor of Biostatistics & Bioinformatics
I am a biostatistician supporting research in cancer therapeutics and palliative care
at the Duke Cancer Institute. Prior to this I supported research studies in cell therapies
and regenerative medicine at the Duke Marcus Center for Cellular Cures. I also teach
biostatistics in the Master of Biostatisti
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