TEHRAN – Researchers from Royan Institute, in a study, have managed to lower the Childhood Autism Rating Scale (CARS) scores and certain inflammatory biomarkers in children with autism spectrum disorder (ASD) using allogeneic mesenchymal stromal cells (MSCs) derived from umbilical cord Wharton’s jelly.

The study was conducted as part of the ongoing research by Royan researchers in the field of regenerative medicine and in the investigation of the potential applications of MSCs for cell-based therapeutic approaches across a range of diseases.

According to the Public Relations Department of Royan Institute, five children with Level 2 ASD participated in the study and received three intrathecal administrations of MSCs derived from umbilical cord Wharton’s jelly. Each administration consisted of 20 million cells, with a one-month interval between administrations, royan.org reported.

The preliminary findings of a Phase I clinical trial evaluated the safety and tolerability of three intrathecal administrations of allogeneic MSCs derived from umbilical cord Wharton’s jelly in children with ASD. No serious adverse events or Grade 3 or higher adverse events were reported during the study period, and the intervention was considered acceptable in terms of safety and tolerability.

These findings are of particular relevance from the perspective of Phase I study design, as a primary objective of such early-phase trials is to assess the safety and tolerability of an investigational intervention and to determine whether further clinical investigation is warranted.

The researchers assessed the severity of autism-related symptoms using the CARS and reported a reduction in CARS scores in all participants. Analysis of cerebrospinal fluid (CSF) also demonstrated reductions in certain inflammatory biomarkers, including tumor necrosis factor-alpha (TNF-a) and interleukin-6 (IL-6). Changes in patterns of cerebral electrical activity were additionally reported based on electroencephalographic (EEG) assessments.

These observations may provide a basis for further investigation into the potential relationships among cellular and molecular alterations, neurophysiological parameters, and selected clinical manifestations of autism spectrum disorder in future studies.

According to the investigators, rigorous evaluation of the potential efficacy of this approach, the durability of the observed effects, and its benefit–risk profile will require further clinical trials involving larger numbers of participants, randomized study designs, and appropriate control groups.

The findings of this study, published in the international journal ‘Stem Cell Research & Therapy’, should not be interpreted as evidence that autism has been treated or cured through cell therapy. Rather, the findings provide preliminary data regarding safety and certain clinical and biological changes observed following the intervention, which may inform the design and scientific rationale of future studies.