More news about stem cell therapy in children with autism spectrum disorder

Recently more news about stem cell therapy in children with autism spectrum disorder (ASD) came from the the medical research team led by Dr. Michael Chez from Pediatric Neuroscience Department of Sutter Institute for Medical Research, Sacramento, California.

Safety of the infusion of own umbilical cord have been proven once more by the results of this clinical trial.

  • Introduction

Recently  have been published in "Stem Cells Translational Medicine"* journal, the results of a new study addressed to children with autism spectrum disorder.

"The objective of this exploratory study was to
assess the safety of a single infusion of own cord blood in subjects with ASD and document changes in language, social behavior, and learning."*

Autism spectrum disorder is currently considered to be a heterogeneous disorder, more mechanisms and risk factors being incriminated for its etiology.

Systemic immune dysfunction is one of the main mechanism proven to be involved in autism spectrum disorder.

Cord blood by its mixture of cells may have the potential to regulate an abnormally activated immune system and thus may potentially improve neuronal function.

Stem cell therapy using own cord blood has been already investigated by some clinical trials performed in children with  acquired neurological diseases.

Although encouraging results have been reported,this is the first randomized, placebo-controlled, double-blinded, cross over trial performed in the United States to assess the feasibility of treating autistic children.

  • Main steps of the clinical study addressed to children with autism spectrum disorder 

According to the study protocol 29 children ages 2 to 6 years with a diagnosis of autism spectrum disorder (ASD) based on Diagnostic and Statistical Manual of Mental Disorders, (DSM-IV-TR) and the Autism Diagnostic Observational Schedule (ADOS) were enrolled in the clinical trial.

Children assessment was applied by neuropsychologist and corroborated by the Principal Investigator.

To exclude any gene variations or chromosome abnormalities,  subjects were required to have documentation of performed genetic tests (e.g. Chromosome analysis or karyotyping, Fragile X DNA testing).

After baseline evaluation the eligible children were divided by chance in two separate groups and subsequently their results were compared. Being a double-blinded study neither the parents of enrolled children nor the investigators have known who is receiving the real treatment or the placebo. This procedure is applied to prevent bias in research results.

First group (A) were infused with their own cord blood, while the other group (B) received placebo solution (a 0.9% saline placebo).

A placebo is a substance/compound with not known therapeutic activity, a neutral substitute for a treatment or intervention, but similar from aspect point of view to the active substance under evaluation.

After the first infusion, children from both of these groups were evaluated at 12, and 24 weeks by applying the same assessment tests which were used at the baseline.

Then, children from both groups received a second infusion with the opposite product, and subsequently evaluated again at 12 and 24 weeks post infusion.

For a better understanding, see below chart*:

Autism study chart

  • Objectives of the clinical trial addressed to children with autism spectrum disorder

The aim of this exploratory study was to assess the safety and clinical effects of the infusion of own cord blood in children with idiopathic (unknown cause) autism spectrum disorder.

Safety was proven in all cases no serious adverse events being reported.

From clinical effectiveness point of view as secondary endpoint of this study, there were reported only minimal evidence. These results are not in line with the findings from the previous clinical study conducted at Duke University Hospital, by dr. Kurtzberg and dr. Dawson.

Therefore the most important conclusion of this study is the confirmation of safety and the necessity to conduct future more tightly controlled trials orientated on eficcacy and effectiveness.

These may prove the potentially  impact which own cord blood infusion may have on socialization for children with autism.


*Michael Chez & al. "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" , Stem Cells Translational Medicine Journal, 2018;00:00–00

2 Responses

  1. Thanks. To add there is still no scientific evidence that stem cell therapy is effective for treating autism. Here is an article/response about stem cells therapy written by<a href="//”" rel="nofollow">The Association for Science in Autism Treatments</a>:
    • admin
      Indeed. These are the results of a phase I clinical study, the first step for any new medical drug or medical device proposed to be clinically tested. First it is necessary to be proven the safety of the new formula of a medical drug or medical device. Only after it is possible to move on to the next stages of clinical studies, evaluating the efficacy and efficiency of the new treatment. Until we don't have statistically proven results of efficacy and/or efficiency of a new treatment, it is not possible to draw clear conclusions about them. It is possible only to mention cautiously about some positive outcomes, as individual observational results, which definitely can't be translated to other individuals.

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