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What is stem cell therapy for Autism?

The efficacy of stem cell therapy for Autism Spectrum Disorder (ASD) is not as well studied as Cerebral Palsy. There are reports of individual patients with dramatic improvements, such as Nicoloz, but so far no published studies in major medical journals have established a clear long-term benefit. Just as with cerebral palsy, the “mechanism of action” of how the cells benefit the brain is not understood.

Nonetheless, stem cell therapy is very popular with parents because autism is a growing problem that now impacts 1 in 59 children in the US and other developed countries. There are Facebook groups for parents who seek stem cell therapy for autism at Duke University or elsewhere.  Within the US, the FDA only allows stem cell therapy for autism as part of a clinical trial.  Any clinic inside the US that offers stem cell therapy for autism and cannot provide a trial registration on ClinicalTrials.gov is operating outside the law and could be shut down by the FDA.

As with cerebral palsy, researchers do not know what type of stem cells are “best” for autism and parents are often confused by the advertising claims made by for-profit clinics. Here are the key differences between therapy with cells from cord blood versus cord tissue:

  • Cord blood stem cells require HLA matching. You cannot receive cord blood from a random donor. Cord blood therapy for autism is only possible using the child’s own cord blood or cord blood from a sibling.
  • Mesenchymal stem/ stromal cells (MSC) from umbilical cord tissue or from bone marrow do not require any matching. Numerous clinics offer autism therapy with MSC from anonymous donors. The donors should be screened for communicable diseases.

If parents do visit a for-profit clinic to obtain “stem cell therapy” for autism, they should try to investigate what is being injected into their child: Exactly what type of cells are being used, how are the cells prepared, and does the laboratory maintain cGMP conditions or have any quality certifications? Please remember that the website of Parent’s Guide to Cord Blood Foundation is not a substitute for medical advice from a physician.

References:

  • Lv Y-T, Zhang Y, Liu M, ....  Xiang Hu X. Transplantation of human cord blood mononuclear cells and umbilical cord-derived mesenchymal stem cells in autism. 2013; Journal of Translational Medicine 11:196
  • Dawson G, Sun JM, Davlantis KS, .... Kurtzberg J. 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. 2017; Stem Cells Translational Medicine 6(5):1332-1339
  • Chez M, Lepage C, Parise C. et 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. 2018; Stem Cells Translational Medicine 7(4):333-341