A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder

Dawson, Geraldine; Sun, Jessica; Baker, Jennifer H.E.; Carpenter, Kimberly L. H.; Compton, Scott N.; Deaver, Megan; Franz, Lauren; Heilbron, Nicole; Herold, Brianna; Horrigan, Joseph P.; Howard, Jill; Kosinski, Andrzej S.; Major, Samantha; Murias, Michael; Page, Kristin; Prasad, Vinod K.; Sabatos‐DeVito, Maura; Sanfilippo, Fred; Sikich, Linmarie; Simmons, Ryan; Song, Allen W.; Vermeer, Saritha; Waters‐Pick, Barbara; Troy, Jesse D.; Kurtzberg, Joanne

doi:10.1016/j.jpeds.2020.03.011

Cited by 35 publications

(50 citation statements)

References 24 publications

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“…As of May 2020, 15 studies of cell therapies in ASD were registered on ClinicialTrials.gov in seven different countries. All are phase 1 or 2 studies, mostly single-arm and open-label, with cell sources including umbilical cord blood (n=4), [43][44][45] MSCs (n=3), 46,47 and autologous bone marrow (n=1) 48 (one study utilized both cord blood and MSCs). 49 Results are available for seven trials (Table 2).…”

Section: Autism Spectrum Disordermentioning

confidence: 99%

“…49 Results are available for seven trials (Table 2). [43][44][45][46][47]49,50 The single-arm studies demonstrate the relative safety of administering cellular products to children with ASD and generally report improvement on some measures of ASD symptoms in at least a portion of the treated participants. Without a control group for comparison, of course, any gains cannot be attributed to the cells themselves.…”

Section: Autism Spectrum Disordermentioning

confidence: 99%

“…At 6 months post‐treatment, the combination group demonstrated greater improvement in the Childhood Autism Rating Scale than placebo or umbilical cord blood mononuclear cells groups, and both treated groups improved more than the placebo group on the Clinical Global Impression scale. In another recently published study, 45 180 children aged 2 to 7 years were randomized to treatment with umbilical cord blood (autologous if available, allogeneic unrelated donor if not) or placebo. While there was no difference in improvement of social communication or ASD symptoms between treatment and placebo groups as a whole, children without intellectual disability who were treated with cord blood demonstrated improved communication skills on the Vineland Adaptive Behaviors Scale, increased sustained attention in eye tracking assessments, and increased in alpha and beta power on EEG.…”

Section: Autism Spectrum Disordermentioning

confidence: 99%

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Stem cell therapies in cerebral palsy and autism spectrum disorder

Sun

Kurtzberg

2021

Develop Med Child Neuro

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Across disciplines, there is great anticipation that evolving cell therapies may finally provide a therapeutic option for conditions in dire need. These conditions are typically complex and their pathophysiology incompletely understood, hindering the development of robust preclinical models and the precise assessment of therapeutic effects in human studies. This article provides an overview of the status of cell therapy investigations in two common neurodevelopmental disorders, cerebral palsy and autism spectrum disorder. Challenges facing this line of study, including inherent heterogeneity, knowledge gaps, and unrealistic expectations, are discussed. Much progress has been made in the past decade, but to definitively determine if cell therapies have a role in the treatment of neurodevelopmental disorders, both fields will need to evolve together. What this paper adds The safety profile of reported cell therapies in children with neurodevelopmental disorders is encouraging. Efficacy trials in cerebral palsy and autism spectrum disorder are ongoing in the United States and Asia. Unresolved issues pertain to the properties of the cells being studied and the characteristics of the neurodevelopmental conditions themselves.

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Section: Autism Spectrum Disordermentioning

confidence: 99%

Section: Autism Spectrum Disordermentioning

confidence: 99%

Section: Autism Spectrum Disordermentioning

confidence: 99%

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Stem cell therapies in cerebral palsy and autism spectrum disorder

Sun

Kurtzberg

2021

Develop Med Child Neuro

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“…Randomized controlled trials using stem cells as investigational products administered to persons with ASD have not demonstrated convincing evidence of efficacy. 6,7 There are no licensed stem cell therapies for ASD, and stem cell interventions are not part of the evidence-based standard of care for persons with ASD. Despite the lack of robust safety and efficacy data supporting the use of stem cell-based therapies in individuals with ASD, many parents pursue such interventions for their children with ASD.…”

Section: Introductionmentioning

confidence: 99%

Ethical issues concerning a pay-to-participate stem cell study

Turner¹,

Snyder

2021

Stem Cells Translational Medicine

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In our critique of a pay‐to‐participate study, we address how the failure to disclose study‐related payments appears to have violated STEM CELLS Translational Medicine's editorial policies concerning conflict‐of‐interest and financial disclosure. Our analysis also identifies broader ethical issues and scientific concerns related to pay‐to‐participate studies conducted by businesses with a record of selling purported stem cell treatments before determining whether the products they sell are safe and efficacious. Authors of peer‐reviewed articles have a responsibility to comply with journal policies and disclose financial conflicts of interest to editors, reviewers, and readers. Authors should also disclose when stem cell interventions being tested in clinical trials have already been sold on a direct‐to‐consumer basis as “stem cell treatments” by authors' affiliate institutions. Financial conflicts of interest and other forms of possible bias must be disclosed to put clinical studies in context and facilitate the critical assessment of research methods, findings, and conclusions. The apparent failure to comply with journal editorial policies and disclose such financial conflicts warrants careful investigation.

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“…Highly effective immunotherapies including allogeneic CAR‐NK and cytotoxic T‐cells (CTLs) have been manufactured from cord blood and are in development for commercialization 13,14 . Derivatives of cord blood CD14 monocytes are in clinical trials for treatment of hypoxic and demyelinating diseases 15‐18 and cord blood is being investigated as a therapy for children with autism spectrum disorder, a severely disabling disease with increasing prevalence in our society 19,20 . Finally cord and placental tissues are robust sources of mesenchymal stromal cells which are undergoing testing in many diseases as immune modulators and suppressors of pro‐inflammatory states, 21 including as a treatment for complications of COVID‐19 22 …”

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confidence: 99%

The view for cord blood is “cup half full” not “cup half empty”

Kurtzberg

2020

Stem Cells Translational Medicine

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Ballen present a targeted overview of the advantages, disadvantages, and milestones of cord blood transplantation over the past 30 years. 1 First they highlight the advantages of cord blood as a source of donor cells for hematopoietic transplantation, including off the shelf availability, increased tolerance for HLA mismatching, lower incidence of acute and chronic GvHD, lower risk of infectious disease transmission, and improved protection against leukemic relapse in high risk patient populations.

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A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder

Cited by 35 publications

References 24 publications

Stem cell therapies in cerebral palsy and autism spectrum disorder

Stem cell therapies in cerebral palsy and autism spectrum disorder

Ethical issues concerning a pay-to-participate stem cell study

The view for cord blood is “cup half full” not “cup half empty”

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