Clinical Trials Study
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World J Methodol. Mar 20, 2025; 15(1): 95857
Published online Mar 20, 2025. doi: 10.5662/wjm.v15.i1.95857
Advances in the treatment of autism spectrum disorder: Wharton jelly mesenchymal stem cell transplantation
Serdar Kabatas, Erdinç Civelek, Eyüp Can Savrunlu, Umut Karaaslan, Özlem Yıldız, Erdal Karaöz
Serdar Kabatas, Erdinç Civelek, Eyüp Can Savrunlu, Department of Neurosurgery, University of Health Sciences Turkey, Gaziosmanpaşa Training and Research Hospital, Istanbul 34255, Türkiye
Serdar Kabatas, Erdinç Civelek, Department of Pediatric Allergy-Immunology, Institute of Health Sciences Marmara University, Istanbul 34865, Türkiye
Serdar Kabatas, Center for Stem Cell & Gene Therapy Research and Practice, University of Health Sciences Turkey, Istanbul 34255, Türkiye
Umut Karaaslan, Department of Child and Adolescent Psychiatry, University of Health Sciences, Gaziosmanpaşa Training and Research Hospital, Istanbul 34255, Türkiye
Özlem Yıldız, Department of Child and Adolescent Psychiatry, Kocaeli University Faculty of Medicine, Kocaeli 41001, Türkiye
Erdal Karaöz, Center for Regenerative Medicine and Stem Cell Research & Manufacturing, Liv Hospital, Istanbul 34340, Türkiye
Erdal Karaöz, Department of Histology and Embryology, Istinye University, Faculty of Medicine, Istanbul 34010, Türkiye
Erdal Karaöz, Center for Stem Cell and Tissue Engineering Research and Practice, Istinye University, Istanbul 34340, Türkiye
Author contributions: Kabataş S and Civelek E contributed to the concept; Kabataş S, Civelek E, and Savrunlu EC, contributed to the design; Kabataş S and Karaöz E contributed to the supervision; Civelek E, Kabataş S, Savrunlu EC, Karaaslan U, and Yıldız Ö contributed to the analysis and/or interpretation; Kabataş S, Civelek E, and Savrunlu EC contributed to the literature search; Kabataş S, Civelek E, Savrunlu EC, Karaaslan U, Yıldız Ö, and Karaöz E contributed to the writing; Kabatas S, Civelek E, and Savrunlu EC contributed to the critical reviews.
Informed consent statement: There is human subject in this article and written informed consents were obtained from the patient for their anonymized information to be published in this article and before the stem cell therapies.
Clinical trial registration statement: Our research constitutes a pilot study rather than a randomized controlled trial. Consequently, it was not formally registered. Our intention is for this pilot study to pave the way for future randomized studies.
Conflict-of-interest statement: All authors declare no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.
CONSORT 2010 statement: The authors have read the CONSORT 2010 statement, and the manuscript was prepared and revised according to the CONSORT 2010 statement.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Serdar Kabatas, MD, Full Professor, Department of Neurosurgery, University of Health Sciences Turkey, Gaziosmanpaşa Training and Research Hospital, Karayolları Mahallesi, Osmanbey Caddesi 616. Sokak No. 10, Gaziosmanpaşa, Istanbul 34255, Türkiye. kabatasserdar@hotmail.com
Received: April 20, 2024
Revised: June 23, 2024
Accepted: August 2, 2024
Published online: March 20, 2025
Processing time: 161 Days and 21.5 Hours
Abstract
BACKGROUND

Autism spectrum disorder (ASD) is a complex neurodevelopmental disorder with multifaceted origins. In recent studies, neuroinflammation and immune dysregulation have come to the forefront in its pathogenesis. There are studies suggesting that stem cell therapy may be effective in the treatment of ASD.

AIM

To evolve the landscape of ASD treatment, focusing on the potential benefits and safety of stem cell transplantation.

METHODS

A detailed case report is presented, displaying the positive outcomes observed in a child who underwent intrathecal and intravenous Wharton’s jelly-derived mesenchymal stem cells (WJ-MSCs) transplantation combined with neurorehabilitation.

RESULTS

The study demonstrates a significant improvement in the child’s functional outcomes (Childhood Autism Rating Scale, Denver 2 Developmental Screening Test), especially in language and gross motor skills. No serious side effects were encountered during the 2-year follow-up.

CONCLUSION

The findings support the safety and effectiveness of WJ-MSC transplantation in managing ASD.

Keywords: Autism spectrum disorder; Neurorehabilitation; Stem cell transplantation; Wharton jelly mesenchymal stem cells; Inflammation

Core Tip: According to data of the World Health Organization, autism spectrum disorder (ASD) is observed in approximately 1 in every 100 children. Recent studies have revealed that immune factors and inflammation are effective in the development of ASD. In this study, we applied six doses of Wharton’s jelly-derived mesenchymal stem cell therapy to a 4-year-old patient diagnosed with autism spectrum disorder. After the applications, we observed the patient for 2 years. We did not encounter any serious side effects. According to the Childhood Autism Rating Scale values and Denver II Developmental Screening Test values, we detected significant improvements.