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

Several human studies show positive signals, while research methods and sample sizes continue to develop.

• Level A · Stronger Clinical Evidence
• Level B · Emerging clinical evidence with positive signals
• Level C · Early human research exploring benefits
• Level D · Scientific groundwork from lab and animal studies
• Emerging · Emerging topic under active research

Abstract (original English)

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 featur

Evidence level

Several human studies show positive signals, while research methods and sample sizes continue to develop.

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