Therapeutic potential of stem cells in pediatric neurology: Insights from clinical trials

Pediatric neurological disorders comprise diverse conditions that impair nervous system function in children and contribute substantially to global disease burden. Stem cell therapy has become a promising treatment in neurology due to the cells' ability to self-renew, ensuring a continuous supply of cells. Cells are harvested from various origins, notably embryonic tissues and adult sources such as bone marrow, adipose tissue, and umbilical cord. Therapeutic effects arise from cell or enzyme replacement, trophic support, immunomodulation, and paracrine actions of the secretome. This review summarizes clinical applications of stem cell therapies across pediatric neurological diseases-including autism spectrum disorder, cerebral palsy, traumatic brain and spinal cord injury, epilepsy, neuromuscular disorders, and lysosomal storage diseases-and appraises evidence from preliminary descriptive studies that update the field and reveal methodological limitations. Reported therapeutic effect differs markedly by cell type, disease biology, timing of intervention, dose, and delivery method, producing inconsistent clinical results. Positive functional or developmental improvements have been documented in selected reports, but safety concerns, heterogeneity in study design, short follow-up, and variable potency assays limit conclusions. Because stem cell populations share phenotypic features but vary in therapeutic capacity, a universal, one-size-fits-all strategy is unlikely to succeed. Critical gaps remain regarding long-term safety, durability, standardized manufacturing, and optimal clinical endpoints. Continued rigorous translational research, standardized clinical trials, and expanded long-term surveillance are essential to optimize these therapies and improve outcomes for affected children and to ensure equitable access for diverse pediatric populations worldwide and sustainable implementation.