How strong is the evidence for stem-cell therapy in Autism Spectrum Disorder?
ClinicalTrials.gov registrations alone cannot establish how strong the evidence is. Registry activity is active but still early, with limited late-phase registration: the registry holds 25 records, 10 completed, 1 at Phase 3 or 4, and 2 stopped early (8%). MSC-specific work accounts for 10 of those records. These counts describe research activity and maturity, not benefit, safety or certainty. Snapshot 2026-08-29.
What counts as a real result in Autism Spectrum Disorder
Meaningful evidence requires blinded assessors using validated instruments, a control group and follow-up long enough to separate treatment effect from development and from concurrent therapy changes. Parent-rated improvement after a costly trip is the classic expectancy signal, and it is what most testimonials contain.
What we can say from registrations alone
- Volume: 25 registered studies since 2009.
- Maturity: 1 confirmatory-phase records.
- Delivery: 10 completed; 2 terminated, withdrawn or suspended.
- Scale: median planned enrolment 37 participants, largest 900.
The completed studies
Completed does not mean published, and published does not mean positive. These are the finished records — the starting point for checking whether results ever appeared.
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT04089579 — hCT-MSC in Children With Autism Spectrum Disorder | Phase 2 | Completed | 137 | Joanne Kurtzberg, MD | 2020-10-12 |
| NCT01343511 — Safety and Efficacy of Stem Cell Therapy in Patients With Autism | Phase 1, Phase 2 | Completed | 37 | Shenzhen Beike Bio-Technology Co., Ltd. | 2009-03 |
| NCT03225651 — Autologous Bone Marrow Stem Cell Therapy Combined With Psychological Therapy and Rehabilitation for Autism | Phase 2 | Completed | 30 | Vinmec Research Institute of Stem Cell and Gene Technology | 2017-07-18 |
| NCT01740869 — Autologous Bone Marrow Stem Cells for Children With Autism Spectrum Disorders | Phase 3 | Completed | 30 | Hospital Universitario Dr. Jose E. Gonzalez | 2012-11 |
| NCT04710810 — UCB Stem Cells for Autism Spectrum Disorders | Phase 1 | Completed | 30 | St. Petersburg Bekhterev Research Psychoneurological Institu | 2019-11-04 |
| NCT01638819 — Autologous Cord Blood Stem Cells for Autism | Phase 2 | Completed | 30 | Sutter Health | 2012-08 |
What registrations cannot tell you
Whether results were positive. Whether they were published at all — a large share of registered studies never report outcomes, which biases what the literature appears to show. And whether the product a commercial clinic sells resembles the product studied: cell source, dose, processing and delivery route vary enormously between a registered protocol and an off-protocol injection.
How to use this when someone is selling you a treatment
Ask which specific registered study supports the offer, then compare its intervention description with your quote. If the answer is a general gesture at "Autism Spectrum Disorder research", you are being sold the field's activity rather than evidence about the product. The Provider Question Builder turns that gap into questions.
Frequently asked
How strong is the evidence for stem-cell therapy in Autism Spectrum Disorder?
ClinicalTrials.gov registrations alone cannot establish how strong the evidence is. Registry activity is active but still early, with limited late-phase registration: 25 registered records, 1 at Phase 3/4 and 2 stopped early (2026-08-29).
Has any stem-cell product been approved for this condition?
Look it up at source: regulators publish authorised products and indications. Registration of a trial, a licence to operate a clinic and product approval are three different things.
Why do clinics cite hundreds of studies?
It converts activity into the appearance of proof. A hundred early-phase registrations do not add up to one adequately controlled result.
Registry and literature counts retrieved 2026-08-29 (ClinicalTrials.gov API v2; PubMed E-utilities). Registration or publication volume measures research activity, not effectiveness or approval. Educational information — not medical advice; verify product, indication, legal pathway and evidence with an independent qualified physician. Page generated 2026-08-29. See our editorial standards.
More on this condition
- Autism Spectrum Disorder: cell-therapy evidence and registered trials
- How much does stem cell therapy for Autism Spectrum Disorder cost? (2026)
- Autism Spectrum Disorder stem cell therapy — your questions answered (2026)
- Autism Spectrum Disorder: cell-therapy evidence by therapy class
- MSC vs exosomes for Autism Spectrum Disorder: what the registries show