Effects of stem cell therapy for autism in children and adults

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SBU Enquiry Service

Consists of structured literature searches to highlight studies that can address questions received by the SBU Enquiry Service from Swedish healthcare or social service providers. We assess the risk of bias in systematic reviews and when needed also quality and transferability of results in health economic studies. Relevant references are compiled by an SBU staff member, in consultation with an external expert when needed.

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Published: Report no: ut202516 Registration no: SBU 2025/496

Question

What systematic reviews have been published regarding the effects of stem cell therapy for autism in children and adults?

PICO

  • Population: Individuals diagnosed with autism spectrum disorder (ASD)
  • Intervention: Stem cell therapy
  • Comparison: Other treatment or no treatment
  • Outcome: Autism symptoms, such as changes in social interaction, communication, or behavioral patterns

Method

A systematic literature search was performed using the following databases: Medline (Ovid), Scopus och Embase.  

Two authors independently assessed the abstracts of all identified studies.

Risk of bias in relevant systematic reviews was assessed using an assessment tool based on AMSTAR.

Identified literature

One relevant systematic review with low to moderate risk of bias were identified [1]. The results and conclusions are presented in Table 1. In six relevant systematic reviews, the risk of bias was considered to be high, therefore the results and conclusions are not reported [2-7].

Table 1 Included systematic reviews with low/moderate risk of bias.
CCT = controlled clinical study that is not randomized; RCT = randomized controlled trial; RR = risk ratio; WMD = weighted mean difference
Included studies  Population, Intervention, Control  Outcome and Results
Qu et al, 2022 [1]
Efficacy and Safety of Stem Cell Therapy in Children with Autism Spectrum Disorders: A Systematic Review and Meta-Analysis
Relevant studies:
3 RCT
2 CCT

Setting:
China: 2 studies
Iran: 1 study
USA: 2 studies		 Population:
Children 0–18 years old, diagnosed with autism (2–15 years old in included studies) 

Intervention:
Multiple kinds of stem cells interventions that vary in administration method and dosage, including methods such as intrathecally injection, intravenous infusion and lumbar puncture

Control: Rehabilitation therapy, for example behavioral therapy, speech and language therapy or sensory integration therapy		 Autism symptoms:
Significant difference in favour of stem cell group (less symptoms) on the Childhood Autism Rating Scale:

WMD: –5.96

(95% CI, –8.87 to –3.06)

n=111 

No significant difference between stem cell group and control group for the Clinical Global Impression Scale:
RR=1.01
(95% CI, 0.87 to 1.18)
n=313 

No significant difference in the incidence of adverse reactions between the stem cell group and the control group  

RR=1.55

(95% CI, 0.60 to 3.98)

n=247
Authors' conclusion:
“The use of stem cells to treat children with autism may be effective and safe, but the current evidence is in-sufficient. The conclusions are based on studies that do not have a uniform treatment protocol and the evidence was compromised by the limitations in current study size as well as shortages in diagnostic tools and long period follow-up studies.Hence, the current state of knowledge calls for more studies to systematically demonstrate the efficacy and safety of stem cell therapy for children with autism spectrum disorders.”

References

  1. Qu J, Liu Z, Li L, Zou Z, He Z, Zhou L, et al. Efficacy and Safety of Stem Cell Therapy in Children With Autism Spectrum Disorders: A Systematic Review and Meta-Analysis. Front Pediatr. 2022;10:897398. Available from: https://doi.org/10.3389/fped.2022.897398
  2. Adnan M, Motiwala F, Trivedi C, Chaudhari G, Mansuri Z, Jain S. Human Umbilical Cord Blood Infusions in the Management of Autism Spectrum Disorder. Prim Care Companion CNS Disord. 2022;24(3). Available from: https://doi.org/10.4088/PCC.21r03042
  3. Akat A, Karaöz E. Cell therapies for autism spectrum disorder: a systematic review of clinical applications. Middle East Current Psychiatry. 2023;30(1). Available from: https://doi.org/10.1186/s43045-023-00363-9
  4. Paprocka J, Kaminiow K, Kozak S, Sztuba K, Emich-Widera E. Stem Cell Therapies for Cerebral Palsy and Autism Spectrum Disorder-A Systematic Review. Brain sci. 2021;11(12):03. Available from: https://doi.org/10.3390/brainsci11121606
  5. Paton MCB, Wall DA, Elwood N, Chiang KY, Cowie G, Novak I, et al. Safety of allogeneic umbilical cord blood infusions for the treatment of neurological conditions: a systematic review of clinical studies. Cytotherapy. 2022;24(1):2-9. Available from: https://doi.org/10.1016/j.jcyt.2021.07.001
  6. Saisa D, Pradhani A, Shen JC, Atthariq MR, Khodavian W, Pawitan JA. Hematopoietic Stem Cell Therapy in Improving Clinical Outcomes for Children with Autism Spectrum Disorder: A Systematic Review and Meta-Analysis. Regen Eng Transl Med. 2025;11(1):209-18. Available from: https://doi.org/10.1007/s40883-024-00355-z
  7. Villarreal-Martinez L, Gonzalez-Martinez G, Saenz-Flores M, Bautista-Gomez AJ, Gonzalez-Martinez A, Ortiz-Castillo M, et al. Stem Cell Therapy in the Treatment of Patients With Autism Spectrum Disorder: a Systematic Review and Meta-analysis. Stem Cell Rev Rep. 2022;18(1):155-64. Available from: https://doi.org/10.1007/s12015-021-10257-0

Literature search

Upplysningstjänsten har gjort sökningar i databaserna Medline (Ovid), Scopus och Embase.

Sökningen har begränsats till systematiska översikter.

Nedan redovisas sökningen som gjorts i de vetenskapliga databaserna.

Medline via OvidSP 07 Month 25

Title: Stem cell treatment and autism
/ = Term from the MeSH controlled vocabulary; .sh = Term from the MeSH controlled vocabulary; exp = Term from MeSH including terms found below this term in the MeSH hierarchy; .ti,ab = Title or abstract;.tw = Title or abstract; .kf = Keywords; .kw = Keywords, exact; .bt = Book title. NLM Bookshelf; .pt = Publication type; .ja = Journal abbreviation; .af = All fields; adjn = Adjacent. Proximity operator retrieving adjacent words, adj3 retrieves records with search terms within two terms from each other; * or $ = Truncation; “ “ = Citation Marks; searches for an exact phrase
Search terms Items found
Population: autism
1. Exp Autism spectrum disorder/ 48 513
2. (autism or autistic or "Rett syndrome" or Asperger or "childhood disintegrate disorder" or "pervasive developmental disorder").ab, bt,kw,ti. 81 167
3. 1 OR 2 85 077
Intervention: stem cell
4. Exp Stem cell transplantation/ 106 729
5. ("stem cell" or "cell therap*" or "regenerative medicine").ab,bt,kw,ti. 263 935
6. ((mesenchymal or stromal or cord or mononuclear) adj3 cell).ab,bt,kw,ti. 57 033
7. 4 or 5 or 6 339 954
Study types: systematic reviews and meta-analysis
8. ((Systematic Review/ or Meta-Analysis/ or Cochrane Database Syst Rev.ja. or ((systematic adj4 review) or "meta analys*" or metaanalys*).ti,bt,ab.) not (editorial/ or letter/ or case reports/)) 539 494
Final result
9. 3 AND 7 AND 8 10

Scopus via scopus.com 08 Month 25

Title: Stem cell transplantation and autism
TITLE-ABS-KEY = Title, abstract or keywords (including indexed keywords and author keywords); ALL = All fields; W/n = Within. Proximity operator retrieving terms within n words from each other; PRE/n = Precedes by. Proximity operator, the first term in the search must precede the second by n words; LIMIT-TO (X) ;= Includes only results of specified type, e.g., publication type or time range; DOCTYPE = Publication type; “re” = review; “le” = letter; “ed” = editorial; “ch” = book chapter; “cp” = conference proceedings; * = Truncation; “ “ = Citation Marks; searches for an exact phrase
Search terms Items found
Population: autism
1. (TITLE-ABS-KEY(autism or autistic or "Rett syndrome" or Asperger or "childhood disintegrate disorder" or "pervasive developmental disorder") 141 460
Intervention: stem cell transplantation
2. (TITLE-ABS-KEY("stem cell" OR "cell therap*" or "regenerative medicine") OR TITLE-ABS-KEY ((mesenchymal OR stromal OR cord OR mononuclear ) W/3 cell)) 938 874
Study types: systematic reviews and meta-analysis / randomized controlled trials
3. (TITLE-ABS-KEY ( ( systematic W/2 review ) OR "meta analy*" OR metaanaly* ) AND (EXCLUDE (DOCTYPE, “le”) OR EXCLUDE (DOCTYPE, “ed”) OR EXCLUDE (DOCTYPE, “ch”) OR EXCLUDE (DOCTYPE, “cp”))) 803 486
Final result
4. 1 AND 2 AND 3 86

Embase via Elsevier 08 Month 25

Title: Stem cell transplantation and autism
/de = Term from the EMTREE controlled vocabulary; /exp = Term from the EMTREE controlled vocabulary, including all narrower terms in the hierarchy; ti = Title; ab = Abstract; kw = Author keywords; it = Publication type; NEAR/n = Near. Proximity operator retrieving terms within n words from each other; * = Truncation; ‘ ‘ = Citation Marks; searches for an exact phrase
Search terms Items found
Population: autism
1. ('autism'/exp OR (autism or autistic or 'Rett syndrome' or Asperger or 'childhood disintegrate disorder' or 'pervasive developmental disorder'):ab,ti,kw) 127 186  
Intervention: stem cell
2. ('stem cell transplantation'/exp OR ('stem cell' OR 'cell therap*' OR 'regenerative medicine' OR ((mesenchymal OR stromal OR cord OR mononuclear) NEAR/3 cell)):ab,ti,kw) 550 552
Study types: systematic reviews and meta-analysis / randomized controlled trials
3. ('systematic review'/de OR 'meta analysis'/de OR [cochrane review]/lim OR ((systematic NEAR/4 review) OR 'meta analys*' OR metaanalys*):ti,ab NOT ('case report'/de OR 'editorial'/it OR 'letter'/it OR 'note'/it)) 784 596
Combined sets:
4. 1 AND 2 AND 3 25
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