Review of Cell Therapy Approaches to Autism: A Review of Clinical Trial

Abstract

Autism Spectrum Disorders (ASD) are characterized by persistent social communication and interaction difficulties, as well as repetitive behavioral patterns, often accompanied by abnormalities of the immune system and inflammatory reactions. Stem cell-based therapies, particularly those involving stem cells, are being investigated as potential treatments. This review examines cellular and molecular ASD abnormalities, stem cell types, animal models, ongoing clinical trials, and therapy limitations. ASD, a complex neurodevelopmental disorder with early onset social communication challenges and repetitive behaviors, lacks effective treatments. New research links ASD to neuroinflammation, microglia and glucose metabolism. Ten clinical trials of cell therapy show generally positive results. Although cell therapies show promise, extensive research is needed. This abstract discusses the potential of cell therapy for conditions lacking effective treatments, including cerebral palsy and ASD. It acknowledges complexity and incomplete understanding of pathophysiology. Disparate results in ASD cell therapy trials raise concerns about therapeutic targets and approaches, which require refined preclinical studies. Global ASD prevalence has increased, but treatments for core symptoms remain inadequate. Stem cell therapies show potential. Non-pharmacological interventions, such as diet and supplements, can address comorbidities. This brief abstract summarizes the aim, methodology, key results and conclusions of the full article. This review analyzes the role of miRNAs in ASD onset and progression. It is exploring miRNAs as regulators of neural development, diagnostic biomarkers and therapeutic targets. Findings from in vivo and in vitro models, including induced pluripotent stem cells (iPSCs), highlight the miRNA impact on ASD phenotypes. miRNAs are critical to understanding ASD pathogenesis. This review discusses neural stem cell-derived extracellular vesicles (NSC-EVs) as a treatment for CNS disorders. It highlights the mechanisms, therapeutic roles and advantages of cell-free therapy of NSC-EVs. The prevalence of Neuropsychiatric Disorders (NPD) is increasing globally, without full understanding of pathophysiology and effective treatments. Human induced pluripotent stem cells (hiPSCs) offer patient-specific neuropharmacological study potential. Guidelines and best practices for hiPSC-based NPD studies, with emphasis on donor selection, experimental design, and translational relevance, aim to improve drug discovery and clinical translation.