Autism spectrum disorder (ASD) is a group of a neurodevelopmental disorders associated with significant social, communication and behavioral challenges. The spectrum refers to the wide range of symptoms and levels of disability among those affected. The rate of Autism has steadily increased over the past few decades and now affects 1 in 59 children with over half classified as having an intellectual or borderline intellectual disability. ASD occurs in all racial, ethnic and socioeconomic groups and is 4 times more common in boys.

Although there is no cure for autism early intervention and treatment can improve a child’s development and quality of life. Individuals with autism often have other medical conditions such as allergies, asthma, epilepsy, digestive disorders, ADHD, OCD, persistent viral infections, sleep disorders and more. ASD has also been shown to be associated with immune dysregulation and neuro-inflammation.

Current conventional management of ASD includes interventions such as psychological, behavior, cognitive and speech therapies and symptom management using medications such as anti-depressants, anti-psychotics, etc., which often have significant side effects. Considering the current approach is rather limited and does not always confer with significant benefits, patients, care givers, clinicians and researchers alike continue to search for more effective therapies.

Numerous research studies demonstrated stem cells not only reduced inflammatory markers and influence change to the central nervous system, but also improve behavior, socializing, nonverbal communication, and lethargy in children with ASD.

Year Author No. of Patients Delivery and Cell Type Follow-up Safety Outcome Efficacy Outcome
2019 Riordan et al 20 IV UC-MSCs 12 Months No treatment-related SAEs were observed during the course of this trial. AEs related to treatment were mild or moderate and short in duration. 40% of children showed improvements of symptoms achieving lower ASD symptom categories as measured by CARS and ATEC. Inflammatory cytokines MDC and TARC decreased in 20%.
2018 Chez et al 29 IV UCB 24 weeks No SAEs. 17/86 AEs were ‘probable’ or possible’ related to treatment. No AEs required treatment. Improvement trends on the Socialization Subscale of the VABS. Minimal evidence of clinical effectiveness due to lack of statistical significant results.
2017 Dawson et al 25 IV UCB 12 months No SAEs. 13% of AEs deemed related to infusion. AEs over 12 months indicate treatment was safe and well tolerated. All behavioral endpoints and EGT measurements improved at 6 months and sustained at 12 months. Significant improvement in behavior.
2013 LV et al 37 IV UC-MSCs and CB-MNCs 24 weeks No allergic, immunological reactions or other SAEs. No significant changes in lab tests. Significant improvements in behavior, nonverbal communication and lethargy (CARS, ABC and CGI). Better outcomes in combined treatment group.

* UC-MSCs = umbilical cord derived mesenchymal stem cells, CB-MNCs = cord blood derived mononuclear cells, UCB = umbilical cord blood, AEs = Adverse Events, SAEs = Serious Adverse Events, ASD = autism spectrum disorder, CARS = Childhood Autism Rating Scale, ATEC = Autism Treatment Evaluation Checklist, MDC = monocyte-derived chemokine, TARC = thymus and activation regulated chemokine, VABS = Vineland Adaptive Behavior and Socialization Subscales, EGT = an objective eye gaze tracking measurement, CARS = Childhood Autism Rating Scale, CGI = Clinical Global Impression, ABC = Aberrant Behavior Checklist.

Early Trials of Stem Cells in ASD

  • Reduction of inflammatory markers in blood (Riordan et al)
  • Significant improvements in behavior, socialization and communication (Dawson et al)
  • Trends toward improved socialization (Chez et al)
  • Improvement in visual, emotional and intellectual responses (Lv et al)
  • Improvement in lethargy/social withdrawal, hyperactivity and inappropriate speech (Lv et al)
  • Improvement in ASD symptom score with patients achieving less severe symptom category (Riordan et al)

Specific Conclusions from Selected Studies

“The administration of repeated-dose UC-MSC infusions is safe and tolerable for patients with ASD. Although this phase I study included a small number of subjects without a placebo arm, the trends observed in this study are indicative of potential therapeutic benefits reflected in lower CARS and ATEC scores that may be associated with decreases in TARC and MDC levels.”

  • Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for the Treatment of Autism Spectrum Disorder in Children: Safety Profile and Effect on Cytokine Levels (Riordan et al 2019)

“This research suggests that stem cells from autologous cord blood are safe and potentially have an impact on socialization for children with autism.”

  • 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 et al 2018)

“We demonstrated in an open label, phase I trial that intravenous infusion of autologous umbilical cord blood in young children with ASD is safe and feasible. We describe significant improvements in behavior observed in the first 6 months post-infusion and sustained at 12 months.”

  • 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 (Dawson et al)

“In summary, CBMNC and UCMSC transplantation may improve some behavioral symptoms and function observed in children with autism. This study presents for the first time a safety and efficacy analysis of using allogeneic CBMNCs and UCMSCs to treat behaviors in addition to conventional behavioral therapy in autism.”

  • Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism (Lv et al)

For additional information and details regarding these studies please visit our Research Archive.

Exosomes have potential significant therapeutic effects in regenerative medicine, anti-aging and chronic disease. Click here to learn more about Exosome Therapy.

Brought to you by:

Ahvie Herskowitz, MD, President of ACAM
Director of Anatara Medicine
Clinical Professor of Medicine at UC San Francisco (2014)
(Read Dr. Herskowitz’s Bio Here)

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