Researchers from the Royan Institute conducted a study that successfully reduced scores on the Childhood Autism Rating Scale (CARS) and certain inflammatory biomarkers in children with Autism Spectrum Disorder (ASD) using allogeneic mesenchymal stem cells (MSCs) derived from umbilical cord Wharton's jelly.
The work is part of Royan researchers' ongoing investigations into regenerative medicine and the study of possible applications of MSCs for cellular therapeutic approaches in various diseases.
According to the Royan Institute's PR department, five children with Level 2 ASD participated in the study. They received three intrathecal injections of MSCs obtained from umbilical cord Wharton's jelly. Each injection contained 20 million cells, with a one-month interval maintained between procedures.
The preliminary results of Phase I clinical trials assessed the safety and tolerability of three intrathecal injections of allogeneic MSCs from umbilical cord Wharton's jelly in children with ASD. No serious adverse events or Grade 3 or higher adverse events were recorded throughout the study period, allowing the intervention to be considered acceptable in terms of safety and tolerability.
These findings are particularly significant for Phase I design, as the primary goal of such early trials is to evaluate the safety and tolerability of the investigational intervention and determine the need for further clinical investigation.
Researchers evaluated the severity of autism-related symptoms using CARS and recorded a decrease in CARS scores in all participants. Analysis of cerebrospinal fluid (CSF) also showed a reduction in some inflammatory biomarkers, including tumor necrosis factor-alpha (TNF-a) and interleukin-6 (IL-6). Furthermore, changes in brain electrical activity patterns were noted based on electroencephalography (EEG) examinations.
These observations may form the basis for further study of potential links between cellular and molecular changes, neurophysiological parameters, and specific clinical manifestations of autism spectrum disorder in future research.
In the opinion of the researchers, a thorough assessment of the potential effectiveness of this method, the sustainability of the observed effects, and the benefit-risk ratio will require additional clinical trials involving a larger number of volunteers, the use of randomized study designs, and appropriate control groups.
The conclusions of this study, published in the international journal 'Stem Cell Research & Therapy', should not be interpreted as proof of treatment or cure for autism using cell therapy. Rather, they represent preliminary data regarding the safety and certain clinical and biological changes observed after the intervention, which may influence the development and scientific basis of future work.
