Introduction: Autism Spectrum Disorder (ASD) is a neurodevelopmental condition characterized by impaired social interaction, communication challenges, and repetitive behaviors. Current ASD treatments focus on behavioral and symptomatic management, lacking curative options. This study explored the safety and efficacy of umbilical cord blood-derived mesenchymal stem cells (UC-MSCs) as a potential therapeutic intervention for ASD in children.
Methods: This single-arm Phase I/II clinical trial included 27 children aged 2.5 to 12 years diagnosed with ASD based on DSM-5 criteria. Participants received four subcutaneous injections of UC-MSCs at three-month intervals, with a dosage of 2 million neurocells per kilogram of body weight per injection, administered in a sterile suspension around the umbilical area. Stem cells were isolated from screened and donated umbilical cords following normal deliveries and prepared under GMP conditions.
Outcome measures were evaluated at baseline, 3, 6, and 12 months post-treatment, with final Follow-ups at 21 months. Safety assessments included adverse event monitoring, complete blood counts, metabolic panels, and inflammatory markers (MDC, TARC). Efficacy was evaluated using standardized ASD-specific tools (CARS, ATEC, ADOS), cognitive scales (WPPSI/WISC), behavioral measures (VABS, ABC), and quality-of-life assessments (PedsQL). Statistical analyses employed repeated measures MANOVA to identify trends in symptom changes across time points. Missing data were analyzed using Little’s MCAR test and replaced with the EM algorithm where appropriate.
Results: UC-MSC therapy was generally safe, with no severe treatment-related adverse events. Of 324 infusions, 135 adverse events were reported, with 20.4% deemed related to the treatment, including mild injection site inflammation and transient behavioral increases (e.g., tics, anxiety). These events were mild and self-resolving.
Efficacy assessments revealed significant reductions in autism severity over time. At the 12-month follow-up, mean CARS scores decreased by 7 points (baseline mean: 38.50; 12-month mean: 32.00), and ATEC scores improved by 25 points (baseline mean: 62.80; 12-month mean: 40.00). These improvements corresponded with clinical observations, with 41% of participants demonstrating a meaningful reduction in CARS scores that placed them in a lower diagnostic category for autism severity. Among these responders, 63.6% transitioned from mild-to-moderate autism to below the diagnostic threshold, while 36.4% improved from severe autism to below threshold levels.
Serum inflammatory markers MDC and TARC decreased by 150.00 pg/mL and 85.00 pg/mL, respectively. TARC levels followed a similar trend. These reductions were associated with corresponding behavioral and cognitive improvements in many participants, suggesting an immunomodulatory mechanism of action.
By 21 months, some regression in symptoms and inflammatory marker levels was noted, with scores stabilizing but remaining improved compared to baseline. Notably, 11 participants (41%) continued to show sustained gains, maintaining reduced ASD severity scores.
Conclusion: UC-MSC therapy demonstrated a favorable safety profile and potential efficacy in reducing core ASD symptoms and improving associated comorbidities. While results are promising, the small sample size and lack of a control group necessitate further research in larger, randomized trials. This study underscores the potential of stem cell therapies as innovative interventions for ASD management.
Keywords: Autism Spectrum Disorder, Cord Blood-Derived Stem Cells, Mesenchymal Stem Cells, Neuroinflammation, Pediatric Therapy.
Citation: Islam Al Dababsekh et.al., (2025). Management of Autism Spectrum Disorder in Children Using Cord Blood-Derived Mesenchymal Stem Cells. J Psychol Neurosci; 7(2):1-7. DOI : https://doi.org/10.47485/2693-2490.1111