Stem Cell Therapy for Autism: A Comprehensive Review of Clinical Trials

Published on [28 September 2025] | Medical Research

Autism Spectrum Disorder (ASD) affects millions of children worldwide, presenting challenges in social communication, behavior, and sensory processing. While traditional therapies focus on behavioral interventions, emerging research explores innovative treatments including stem cell therapy. This comprehensive review examines seven groundbreaking clinical trials conducted between 2013-2025, investigating various stem cell approaches for treating autism in children.

The Promise of Regenerative Medicine​

Stem cell therapy represents a paradigm shift in autism treatment, targeting potential neurobiological mechanisms underlying ASD. These cellular therapies aim to:

  • Reduce neuroinflammation
  • Promote neural repair and connectivity
  • Modulate immune system dysfunction
  • Support brain development and plasticity

Clinical Trial Landscape: Seven Key Studies​

Early Pioneers: Safety-First Approach

The Bradstreet Study (2013) marked one of the first explorations into fetal stem cell transplantation for autism. This open-label pilot study demonstrated that cellular therapy could be administered safely to children with ASD, establishing a foundation for future research. The study found no significant adverse events, including no transmitted infections or immunological complications.

Breakthrough Phase I Results

Dawson’s 2017 Phase I trial represented a major milestone, testing autologous umbilical cord blood in 25 young children with autism. The results were encouraging: approximately 70% of children showed improvement in one or more core autism symptoms. Notably, children with higher cognitive abilities (non-verbal IQ >70) demonstrated greater improvements, suggesting that patient selection may be crucial for treatment success.

The Definitive Phase II Trial

The 2020 Phase II randomized controlled trial by Dawson et al. stands as the most rigorous study to date. This double-blind, placebo-controlled trial involving 180 children with ASD tested whether a single intravenous umbilical cord blood infusion could improve social communication skills.

Key Findings:

  • Treatment was safe and well-tolerated
  • No significant improvement in primary outcomes compared to placebo
  • Results challenged earlier optimistic findings
  • Highlighted the complexity of treating ASD with single interventions

Exploring Different Cell Sources

Riordan’s 2019 study investigated allogeneic (donor-derived) umbilical cord mesenchymal stem cells, focusing on safety profiles and immune system effects. This research provided crucial data on using donor cells rather than the patient’s own cells, potentially making treatment more accessible.

Sun’s 2020 research explored umbilical cord tissue-derived mesenchymal stromal cells, representing yet another cellular source. This diversification of cell types reflects researchers’ efforts to identify the most effective therapeutic approach.

Alternative Approaches

Sharifzadeh’s 2021 randomized controlled trial took a different approach, using intrathecal (direct spinal) injection of autologous bone marrow stem cells. This method aimed to deliver cells directly to the central nervous system, potentially increasing therapeutic efficacy while using the patient’s own bone marrow as the cell source.

Latest Developments

The 2025 review by Kabataş et al. highlighted advances in Wharton’s jelly mesenchymal stem cell transplantation, representing the newest frontier in ASD cellular therapy. Wharton’s jelly, found in umbilical cord matrix, offers another promising cell source with unique regenerative properties.

Stem Cell Therapy Benefits for Autism Spectrum Disorder(ASD)

Autism Spectrum Disorder (ASD) is a complex neurodevelopmental condition characterized by challenges in social communication, restricted interests, and repetitive behaviors. While established interventions such as behavioral therapy, speech therapy, and occupational therapy remain the standard of care, there has been growing global interest in stem cell–based therapies as a potential adjunctive treatment.

Understanding Stem Cell Therapy

Stem cells are undifferentiated cells with the unique capacity to self-renew and differentiate into specialized cell types. In autism research, mesenchymal stem cells (MSCs) and umbilical cord blood–derived stem cells are most commonly studied. These cells are thought to provide therapeutic benefits through immune regulation, anti-inflammatory action, and neuroprotection.

Potential Benefits in Autism

1. Modulation of Neuroinflammation

Several studies have demonstrated that children with autism often exhibit increased markers of inflammation in the brain and abnormal immune responses. Stem cells possess strong immunomodulatory properties, which may help normalize this imbalance and improve neurological function.

2. Enhancement of Neural Connectivity

Autism has been linked to atypical brain connectivity and impaired neuronal signaling. Stem cells secrete growth factors and neurotrophic factors that may support repair of damaged neural networks and improve brain plasticity.

3. Improvement in Communication and Social Interaction

Preliminary clinical trials and observational reports suggest that children who undergo stem cell therapy show measurable improvements in language development, social awareness, and eye contact. These improvements, while variable, are among the most encouraging outcomes reported by families.

4. Reduction in Behavioral Challenges

Hyperactivity, irritability, and repetitive behaviors are common concerns for parents of children with autism. Some studies indicate that stem cell therapy may help reduce these behavioral symptoms, potentially enhancing daily functioning and overall quality of life.

5. Support for Cognitive Development

By reducing inflammation and enhancing neural repair, stem cell therapy may positively influence cognitive processes such as attention, memory, and learning, which are often affected in children with ASD.

Safety Considerations

By reducing inflammation and enhancing neural repair, stem cell therapy may positively influence cognitive processes such as attention, memory, and learning, which are often affected in children with ASD.

Stem cell therapy represents a promising and innovative approach in the management of Autism Spectrum Disorder. While it should not be regarded as a definitive cure, the therapy offers potential benefits in improving communication, social interaction, and behavior, alongside reducing neuroinflammation. Parents and caregivers considering this option should consult with qualified healthcare professionals and rely on evidence-based information before making treatment decisions.

Reference

  1. Dawson, G., Sun, J. M., Baker, J., Carpenter, K., Compton, S., Deaver, M., Franz, L., Heilbron, N., Herold, B., Horrigan, J., Howard, J., Kosinski, A., Major, S., Murias, M., Page, K., Prasad, V. K., Sabatos-DeVito, M., Sanfilippo, F., Sikich, L., . . . Kurtzberg, J. (2020). 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, 222, 164-173.e5. https://doi.org/10.1016/j.jpeds.2020.03.01 
  2. Riordan, N. H., Hincapié, M. L., Morales, I., Fernández, G., Allen, N., Leu, C., Madrigal, M., Paz Rodríguez, J., & Novarro, N. (2019). 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, 8(10), 1008-1016. https://doi.org/10.1002/sctm.19-0010 
  3. Sharifzadeh, N., Ghasemi, A., Afshari, J. T., Moharari, F., Soltanifar, A., Talaei, A., Pouryousof, H. R., Nahidi, M., Fayyazi Bordbar, M. R., & Ziaee, M. (2021). Intrathecal autologous bone marrow stem cell therapy in children with autism: A randomized controlled trial. Asia-Pacific Psychiatry, 13(2), e12445. https://doi.org/10.1111/appy.12445 
  4. Kabatas S, Civelek E, Savrunlu EC, Karaaslan U, Yıldız Ö, Karaöz E. Advances in the treatment of autism spectrum disorder: Wharton jelly mesenchymal stem cell transplantation. World J Methodol 2025; 15(1): 95857 [PMID: 40115399 DOI: 10.5662/wjm.v15.i1.95857]
  5. Bradstreet, J. J., Sych, N., Antonucci, N., Klunnik, M., Ivankova, O., Matyashchuk, I., Demchuk, M., & Siniscalco, D. (2013). Efficacy of Fetal Stem Cell Transplantation in Autism Spectrum Disorders: An Open-Labeled Pilot Study. Cell Transplantation. https://doi.org/10.3727/096368914X684916 
  6. Sun, J. M., Dawson, G., Franz, L., Howard, J., McLaughlin, C., Kistler, B., Meadows, N., Troy, J., & Kurtzberg, J. (2020). Infusion of human umbilical cord tissue mesenchymal stromal cells in children with autism spectrum disorder. Stem Cells Translational Medicine, 9(10), 1137-1146. https://doi.org/10.1002/sctm.19-0434 
  7. Dawson, G., Sun, J. M., Davlantis, K. S., Murias, M., Franz, L., Troy, J., Simmons, R., Durham, R., & Kurtzberg, J. (2017). 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. Stem Cells Translational Medicine, 6(5), 1332-1339. https://doi.org/10.1002/sctm.16-0474 
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