10 Feb Umbilical Cord Blood Stem Cells May Improve Symptoms in Children with Autism
MedicalResearch.com Interview with:
Michael G. Chez, M.D.
Director of Pediatric Neurology Sutter Memorial Hospital
Director of the Pediatric Epilepsy and Autism Programs
Sutter Neuroscience Group
MedicalResearch.com: What is the background for this study? What are the main findings?
Response: The study looked at possible use of autologous cord blood as a source of stem cells in patients with autism. The patients had to have fairly good genetic screening per protocol and had confirmation of autism to participate.
The use of cord blood was a pilot cross over double blind study with hypothesis that a post natal factor or immune dysregulation may add to the autism clinical phenotype.
Cord blood ( the baby’s own from birth) is a safe source of mixed stem cell types and should be safe from rejection or autoimmune reaction in theory.
Infusion /placebo or placebo/infusion was randomized and observed and tested every 3 months with switch to other wing of treatment at 0 and 6 months. Total observation was over 1 year.
MedicalResearch.com: What should readers take away from your report?
Response: That cellular cord blood therapy using your own supply of cord blood is safe. There may be trends for language and social improvement but due to pilot study smaller number of participant true statistically significant changes over placebo were hard to achieve, but subgroups with strong response were seen, and socialization scores on standardized Vineland social subtesting was positive.
Post hoc analysis is in progress now that the paper is published and the principal investigator is allowed per protocol to be unblended and actually look at responder and non- responders and see if further medical benefit may occur in some subtypes in the study. This work begins now and hopefully will add to our experience using cellular interventions for autism. This type of work opens new potential therapy interventions for autism and parallels current ongoing work at Duke University using umbilical cord blood in autism by Dr Krissberg.
MedicalResearch.com: What recommendations do you have for future research as a result of this work?
Response: To better classify subgroups and pick better language testing and perhaps longer observation periods.
Perhaps standardize dosing, and look into future cellular products that use matched pooled sources of cord blood or enriched stem cell products that are deemed safe by our FDA or similar agencies for study.
MedicalResearch.com: Is there anything else you would like to add?
Response: The study was sponsored by a research grant form CBR (Cord Blood Registry) based on my investigator initiated designed protocol. I have no personal conflicts of interest to disclose.
I would add many thanks to patients and their dedicated famllies helping to do this type of research.
Safety and Observations from a Placebo-Controlled, Crossover Study to Assess Use of Autologous Umbilical Cord Blood Stem Cells to Improve Symptoms in Children with Autism
Chez M, Lepage C, Parise C, Dang-Chu A, Hankins A, Carroll M.
Stem Cells Transl Med. 2018 Feb 6. doi: 10.1002/sctm.17-0042.
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