Evaluating Wharton’s jelly-derived stem cell therapy in autism: Insights from a case study

Abstract

Autism spectrum disorder (ASD) is a complex neurodevelopmental disorder affecting over 2% of the global population, marked by social communication deficits and repetitive behaviors. Kabatas et al explored the efficacy and safety of Wharton’s jelly-derived mesenchymal stem cell (WJ-MSC) therapy in a 4-year-old child with ASD. Using the childhood autism rating scale and Denver II developmental screening test, significant improvements were seen after six WJ-MSC sessions, with no adverse events over 2 years. Despite promising results, the study’s single-case design limits generalizability. Larger, multi-center trials are needed to validate the findings and assess long-term effects of WJ-MSC therapy in ASD.

Key Words: Autism spectrum disorder; Stem cell therapy; Neuroinflammation; Neurodevelopmental disorders; Wharton jelly derived mesenchymal stem cell

Core Tip: This letter highlights the groundbreaking study by Kabatas et al, which demonstrates the efficacy and safety of Wharton’s jelly-derived mesenchymal stem cell (WJ-MSC) therapy in improving developmental outcomes for a child with autism spectrum disorder. Despite limitations such as the single-case design and lack of a control group, the study suggests WJ-MSC therapy as a promising treatment option, emphasizing the need for larger, controlled trials to validate these findings and develop standardized treatment protocols.

TO THE EDITOR

Neurodevelopmental disorders impact over 2% of the global population, with autism spectrum disorder (ASD) representing a prominent and heterogeneous condition characterized by challenges in social communication and interaction, as well as restricted and repetitive behaviors that typically manifest in early childhood[1,2]. Despite substantial research, the precise etiology of ASD remains elusive, with onset varying across individuals. Proposed mechanisms include advanced parental age, pregnancy-related complications, drug use during pregnancy, toxin exposure, epigenetic factors, oxidative stress, hypoxic damage, neurotransmitter anomalies, and neuroinflammation[3]. Recent literature has emphasized neuroinflammation and altered cytokine levels within the central nervous system as key contributors to the neurobiological changes observed in ASD, suggesting potential therapeutic targets[4]. Given the variability of ASD, assessment and treatment require a multidisciplinary approach. Early interventions are critical in mitigating symptoms and improving the quality of life for young children with ASD. Traditionally, behavioral and psychosocial therapies have been the mainstay of treatment; however, stem cell therapy has recently emerged as a promising alternative[5]. Notably, stem cell therapy has shown significant improvements in social skills and reductions in autistic symptoms, with a meta-analysis indicating no serious adverse effects[6]. A recent study by Kabatas et al[7] further investigates the efficacy and safety of Wharton’s jelly-derived mesenchymal stem cell (WJ-MSC) transplantation in ASD patients.

MSC TRANSPLANTATION IN ASD

The study by Kabatas et al[7] is a detailed single-case assessment of WJ-MSC therapy in ASD. The study involved a 4-year-old child diagnosed with ASD, who presented with impaired eye contact, frequent crying spells, and severe social interaction difficulties. Initial assessments included a childhood autism rating scale (CARS) score of 37 and Denver II developmental screening test results indicating developmental delays. The child underwent six sessions of intravenous and intrathecal WJ-MSC transplantation under sedation. Post-treatment assessments demonstrated improvements in both CARS score and Denver II developmental screening test results, with no adverse events reported during a 2-year follow-up. These findings provide promising evidence for the potential benefits of WJ-MSC therapy in treating ASD symptoms and enhancing developmental outcomes while highlighting its safety profile.

The comprehensive evaluation using both the CARS score and Denver II developmental screening test ensures a robust assessment of developmental progress. Additionally, the use of WJ-MSCs, known for safety and low risk of immune rejection, lends credibility to the findings. Nonetheless, the study has notable limitations. As a single-patient case report, the generalizability of the findings is limited. While the 2-year follow-up is commendable, longer-term studies are necessary to fully assess the enduring effects and safety of WJ-MSC therapy. The study also did not evaluate specific neurobiological markers or mechanisms, which could offer deeper insights into treatment efficacy. Furthermore, the reliance on subjective measures, such as parental reports, may introduce variability in developmental assessments.

Despite its limitations, the study offers significant clinical implications for ASD management. The promising results of WJ-MSC therapy suggest it could be a viable treatment option to improve developmental outcomes in children with ASD. Improvements in social skills and developmental milestones underscore the potential of WJ-MSC therapy to address core ASD symptoms, such as impaired social interaction and communication difficulties. These findings advocate for the development of standardized protocols for WJ-MSC application, including optimal dosing, administration routes, and patient selection criteria. Moreover, interdisciplinary collaboration among neurologists, pediatricians, and stem cell researchers is essential to integrate this emerging therapy into comprehensive ASD management plans. Future research should focus on larger, multi-center trials to confirm these preliminary results and refine treatment approaches.

CONCLUSION

The study by Kabatas et al[7] provides valuable initial evidence supporting the use of WJ-MSCs in treating ASD. The observed improvements in developmental assessments and the absence of severe adverse effects during the 2-year follow-up period highlight the potential of WJ-MSC therapy to address core ASD symptoms. Although the study’s single-case design limits its generalizability, it paves the way for future research. The promising outcomes emphasize the need for larger, controlled, and longitudinal studies to validate these findings and explore the long-term effects of WJ-MSC therapy. This research contributes to the growing body of evidence on stem cell therapies for neurodevelopmental disorders, and underscores the importance of continued innovation and interdisciplinary collaboration in advancing ASD treatment.