ABSTRACT:
Background: Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder affecting approximately 1 in 100 children worldwide. Existing pharmacological interventions targeting ACE2 or other immune-related agents have not yet undergone largescale analysis. Umbilical cord-derived mesenchymal stem cells (UC-MSCs) are a novel, ethically supported, immunomodulatory therapeutic that may engage these central biological mechanisms. Objective: To perform a review of the safety, clinical efficacy, mechanistic basis and routes of administration of UC-MSC therapy and associated cell-free products in children with ASD. Methods and materials: A detailed literature search was conducted using PubMed/MEDLINE, Scopus, Web of Science, the Cochrane Library, and ClinicalTrials.gov. gov through the first half of November 2025. Clinical and preclinical studies assessing UCMSCs or products derived from these cells (including exosomes) in paediatric ASD and similar neurological disorders were identified. Eligible randomised controlled trials (RCTs) and observational studies published in English, reporting safety and/or efficacy measures, were narratively synthesised by safety profile, clinical efficacy, the mechanistic data on which they relied, and route of administration. Results: Safety profiles were favourable in all phase I/II trials and case reports, with no serious adverse events observed with the treatment; transient, self-limited reactions were observed. Clinically meaningful improvements, defined as such based on standardised measures CARS, ATEC and adaptive behaviour scales, were noted in approximately 40–50% of treated subjects. Mechanistic evidence supports systemic immunomodulation, reduced neuroinflammation, restoration of the excitatory–inhibitory balance, and paracrine neurotrophic signalling. Novel cell-free candidates have emerged, among them UC-MSC-derived exosomes. Conclusions: UC-MSC therapy is feasible and well-tolerated with promising early efficacy signals. Nevertheless, validating these findings in rigorously designed double-blind randomised controlled trials with biomarker-based patient stratification will be essential before wider clinical application.
Cite this article:
Kovardini Anandhi Jayavelu, Venkateswaramurthy N. Umbilical Cord-Derived Mesenchymal Stem Cell Therapy in Autism Spectrum Disorder: A Mini Review. Research Journal of Pharmacology and Pharmacodynamics. 2026;18(4):389-4. doi: 10.52711/2321-5836.2026.00052
Cite(Electronic):
Kovardini Anandhi Jayavelu, Venkateswaramurthy N. Umbilical Cord-Derived Mesenchymal Stem Cell Therapy in Autism Spectrum Disorder: A Mini Review. Research Journal of Pharmacology and Pharmacodynamics. 2026;18(4):389-4. doi: 10.52711/2321-5836.2026.00052 Available on: https://rjppd.org/AbstractView.aspx?PID=2026-18-4-8
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