Stem Cell Therapy for Autism

What is the autism spectrum? 

Autism is a spectrum of disorders characterized by marked abnormalities in communication and social interactions. Two common consistent findings are associated in children with this disorder are diminished oxygenation in specific areas of the brain and a chronic immunologically mediated inflammatory condition in the gut. Autism spectrum disorder (ASD) usually is not diagnosed until the child is a few years old, based on difficulties with language and social skills, and behaviors that are rigid or repetitive. The percentage of children with autism has been rising in developed countries; over the span of one decade in the United States the prevalence of autism has gone from 1 in 125 to the latest statistic of 1 in 59 children (CDC). 


What stem cell research exists for autism?

Stem cells are an effective form of treatment because, in a way, they are ultimate “spare” or “blank cells.” they can be “programmed” to become any cell that is required. Autism is a good candidate for stem cell therapy because evidence exists that some types of stem cells, given intravenously, yet clinical trials for autism are lagging. Between the years 2011 to 2018, worldwide there were 14 stem cell trials for autism. Dr. Kurtzberg’s team at Duke (is researching autism therapy with both CB-MNC and UC-MSC) launched their first autism clinical trial in 2014 treating children with their own cord blood (NCT02176317). Although the study did not have a control group, the published results show significant improvements on the Vineland Adaptive Behavior Scale (VABS) as well as clinician-measured scales. Improvements were much better in children that started with a non-verbal IQ at or above 70. A similar study at Sutter Health in California (NCT01638819), using a lower cell dose, did not find significant improvements. Since then, both Duke University and the Panama Stem Cell Institute have completed clinical trials that treat autism with a different source of cells, the mesenchymal stromal cells (MSC) from cord tissue. The Panama trial was conducted in 2015 and the results were published in June 2019, while the Duke trial was conducted in 2017 and the data is still in preparation.

Why treat autism with Mesenchymal Stem/Stromal Cells (MSC)? 

The primary sources of the MSC in clinical trials are bone marrow, fat tissue, and umbilical cord tissue. MSC are the most popular cell type in regenerative medicine because, on top of their potential to treat any type of inflammation, they have also been considered to be “immune privileged”. Being immune privileged means the cells do not trigger the patient’s immune system to recognize them as foreign. When MSC from a donor are given to a patient, no testing for donor-patient matching is required. This has the enormous practical advantage that a clinic can obtain MSC from unrelated donors and use them to treat all their patients.  

On Other Studies 

An Autism study in China reported that the combination of cord blood mononuclear stem cells (CBMNCs) and mesenchymal stromal stem cells (UCMSCs) showed larger therapeutic effects than the CBMNC transplantation alone. Stem cell therapy for autism is still an experimental treatment that is under research. When a clinic gives you promises that you will see gains, and/or claims that the same stem cell therapy can cure everything, run! In real medicine, results are not guaranteed and there are no cure-alls.