Preliminary findings from a Phase I clinical trial involving researchers from Royan Institute have been published, evaluating the safety and tolerability of three intrathecal administrations of allogeneic mesenchymal stromal cells (MSCs) derived from umbilical cord Wharton’s jelly in children with autism spectrum disorder (ASD).
Five children with Level 2 ASD received three administrations of 20 million cells each at one-month intervals.
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.
As a Phase I study, the primary objective was to assess the safety and tolerability of the investigational intervention and determine whether further clinical investigation is warranted.
The researchers assessed autism-related symptoms using the Childhood Autism Rating Scale (CARS) and reported a reduction in CARS scores in all participants.
Analysis of cerebrospinal fluid (CSF) showed reductions in certain inflammatory biomarkers, including tumor necrosis factor-alpha (TNF-α) and interleukin-6 (IL-6).
Electroencephalographic (EEG) assessments also indicated changes in patterns of cerebral electrical activity.
These findings may provide a basis for further investigation of potential relationships among cellular and molecular changes, neurophysiological parameters and selected clinical manifestations of ASD.
However, the investigators emphasized that the efficacy-related findings are preliminary.
The study involved only five participants and was conducted as an open-label trial without a control group, so the findings are insufficient to establish the efficacy of the therapeutic approach or determine its comparative effectiveness relative to existing treatments.
Further evaluation of potential efficacy, the durability of observed effects and the benefit-risk profile will require larger clinical trials with randomized designs and appropriate control groups.
Mahdi Lotfipanah, Director of Public Relations and International Affairs at Royan Institute, said the study is part of the institute’s ongoing research into regenerative medicine and the potential applications of mesenchymal stromal cells in cell-based therapeutic approaches for a range of diseases.
Lotfipanah said the findings, 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.
He said the study instead provides preliminary data on safety and certain clinical and biological changes observed following the intervention, which may inform the design and scientific rationale of future studies.