Stem cell therapy for autism.

Clinics give children cells from cord blood, cord tissue or their own bone marrow, by drip or by injection into the spine. The two largest placebo-controlled trials, in 317 children, found it did not change autism.[1][2]

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What to expect

The procedure

Cells from donor cord blood, cord tissue or the child's own bone marrow are given through a drip, or injected into the fluid around the spinal cord.[1][2][3] A spinal injection means putting the child under anaesthetic.[4]

How many sessions

The blinded trials gave a single infusion.[1][2] Clinics usually sell a course: parents describe several infusions over one trip, then three to five trips in total.[4]

When parents see a change

Parents rarely report a change in the first month, and most who report one settle on about six months.[4] Many say they cannot separate it from the speech and behaviour therapy their child was having anyway.[4]

Risks

Infusion reactions were more common with cells than placebo: 13 of 68 children against 2 of 69.[2] In an earlier Duke study, 5 of 12 children made antibodies against the donor tissue.[5] Seizures have followed spinal injection in two Indian series,[6][3] and one study found bacteria in a thawed cord blood unit.[7]

The two largest blinded trials found no effect

Does it work? Not in the trials that compared it with a placebo. Clinic studies report that most children improve, but none of them had a placebo group, and the people running them were usually selling the treatment.[3][8]

No effecton social communication, autism symptoms or vocabulary at six months after one cord blood infusionDuke randomised placebo-controlled trial, 180 children[1]
1.32 pointsdifference from placebo on a social and communication score, too small and uncertain to count as an effectDuke cord tissue trial, 137 children[2]
RetractedThe Panama study that reported benefit was withdrawn because families had been charged to take part without this being disclosedJournal retraction, 2021[9]
12 of 27parents who described their child's treatment online reported a clear improvement. 7 saw no change.Our review of patient accounts on Reddit and forums[4]

Where it is allowed

United States. No stem cell product is FDA-approved for autism.[10] Duke's open-access cord blood programme expressly excludes autism.[11]

Malaysia. Donor and lab-grown cells must be registered with safety and efficacy data before a clinic can use them.[12]

Mexico. Cord stem cells are approved only for children with blood diseases.[13] COFEPRIS has said stem cell treatments lack authorisation.[14]

Specialists. The American Academy of Pediatrics recommends evidence-based behavioural and other therapies, and cell therapy is not among them.[15]

Common questions

Why do clinic studies show 90% improvement when the Duke trials show nothing?

Clinic studies have no placebo group, so everyone knows the child was treated, and most use questionnaires filled in by parents.[3][8] In the Duke trial, the placebo group's scores also moved over six months.[1]

Will treatment abroad stop my child joining a trial later?

It can. Duke's programme excludes children who have had donor cell therapy outside a Duke trial.[11]

Is cord blood better than bone marrow?

No trial has compared them. Duke compared the child's own cord blood with donor cord blood and found neither did better than placebo.[1]

Questions to ask a clinic

  1. Which cells, from where, and can I see the certificate for this batch?
  2. Is it a drip, or an injection into the spine under anaesthetic?
  3. How many trips and infusions will you recommend, and at what total cost?
  4. Is this a registered clinical trial, and am I being charged to take part?
  5. Can I speak to families who saw no change, not only those who did?
  6. Will this rule my child out of a clinical trial later?

Sources

15 references
  1. Dawson G, et al. A Phase II Randomized Clinical Trial of the Safety and Efficacy of Intravenous Umbilical Cord Blood Infusion for Treatment of Children with Autism Spectrum Disorder. The Journal of Pediatrics, 2020. Link
  2. Kurtzberg J. A Phase II Study of hCT-MSC, an Umbilical Cord-Derived Mesenchymal Stromal Cell Product, in Children With Autism Spectrum Disorder (IMPACT). ClinicalTrials.gov, U.S. National Library of Medicine, 2024. Link
  3. Sharma A, et al. Autologous bone marrow mononuclear cell administration in a large cohort of 1,011 patients with autism spectrum disorder: a retrospective observational study. Clinical Transplantation and Research, 2026. Link
  4. StemFinder review of first-hand patient accounts on Reddit and patient forums, 2026. Method
  5. Sun JM, et al. Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. Stem Cells Translational Medicine, 2020. Link
  6. Sharma A, et al. Autologous bone marrow mononuclear cell therapy for autism: an open label proof of concept study. Stem Cells International, 2013. Link
  7. Wong CM, et al. Autologous umbilical cord blood infusion for the treatment of autism in young children: A within-subjects open label study on safety (assessed via caregiver report) and efficacy. Autism Research, 2024. Link
  8. Kobinia GS, et al. Intrathecal injection of bone marrow concentrate in children with autism spectrum disorder: a retrospective chart analysis. Frontiers in Medicine, 2025. Link
  9. STEM CELLS Translational Medicine Editors. Retraction: Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for the Treatment of Autism Spectrum Disorder in Children: Safety Profile and Effect on Cytokine Levels. Stem Cells Translational Medicine, 2021. Link
  10. U.S. Food and Drug Administration. Approved Cellular and Gene Therapy Products. U.S. Food and Drug Administration, Center for Biologics Evaluation and Research, 2026. Link
  11. Kurtzberg J. Expanded Access Protocol: Umbilical Cord Blood Infusions for Children With Brain Injuries. ClinicalTrials.gov, U.S. National Library of Medicine, 2026. Link
  12. National Pharmaceutical Regulatory Agency, Ministry of Health Malaysia. Guidance Document and Guidelines for Registration of Cell and Gene Therapy Products (CGTPs) in Malaysia, Second Edition (September 2025). 2025. Link
  13. Secretaría de Salud, Gobierno de México. 318. Uso de células madre contará con marco regulatorio. Gobierno de México, Secretaría de Salud (press Release), 2018. Link
  14. Comisión Federal para la Protección contra Riesgos Sanitarios (COFEPRIS). La COFEPRIS suspendió clínica del D.F. que ofrecía tratamientos con Células Madre sin aval científico. Gobierno de México, COFEPRIS (press release), 2015. Link
  15. Hyman SL, et al. Identification, Evaluation, and Management of Children With Autism Spectrum Disorder. Pediatrics, 2020. Link

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