Regenerative medicine approaches for children with cerebral palsy: a systematic review of clinical safety and effectiveness

Background: Cerebral palsy (CP) is a leading cause of childhood disability, and current interventions limited to symptomatic management. Regenerative medicine has been proposed as a promising to enhance neuroplasticity and improve outcomes. This systematic review aimed to synthesize clinical evidence regarding the effectiveness and safety of regenerative medicine interventions in children with CP.

Methods: A systematic search of PubMed, Web of Science, Cochrane Library, and ScienceDirect [2010–2025] identified studies evaluating regenerative therapies in pediatric CP. Eligible studies included randomized controlled trials (RCTs), interventional studies, cohort studies, and case series ≥3 participants. Risk of bias was assessed using RoB-2 and ROBINS-I. Due to heterogeneity, findings were synthesized narratively.

Results: Eighteen studies including 1,087 children were analyzed. Most interventions involved umbilical cord (UC)-derived products (73%), followed by bone marrow-derived cellular therapies and granulocyte colony-stimulating factor-based protocols. Cord-derived therapies demonstrated moderate improvements in motor outcomes in selected trials, combination therapies (cord blood with erythropoietin) demonstrated the greatest motor and cognitive gains, while bone marrow approaches showed more variable effects. Adverse events were primarily mild and transient; however, long-term safety data were limited.

Conclusions: Regenerative medicine represents a promising adjunctive strategy for treating CP in children, particularly with regard to products derived from UC blood. However, the variability in study design, small sample sizes, and short follow-up periods limit the possibility of drawing definitive conclusions.