Abstract / Summary
Pediatric patients with genetic disorders, neurological problems, and congenital defects continue to face significant therapeutic challenges. Despite advances in treatment, many pediatric diseases remain incurable due to the limited availability of effective therapeutic options. Stem cells with their unique regenerative properties are promising candidates for the treatment of various pediatric diseases. Numerous clinical studies are currently investigating the safety and efficacy of stem cell-based treatments in the pediatric population. This narrative review explores the diverse sources of stem cells utilized in pediatric therapeutic applications and critically appraises their clinical relevance across a range of pediatric conditions. Furthermore, it distinguishes established hematopoietic stem cell transplantation (HSCT) from emerging approaches such as mesenchymal stem cells (MSCs), extracellular vesicles (EVs), induced pluripotent stem cells (iPSCs), and tissue engineering strategies, focusing on evidence strength, safety, clinical progress, translational challenges, risks, and ethical considerations. In addition, this review highlights the specific limitations and discusses future perspectives associated with stem cell-based therapies in pediatric patients. While current preclinical and clinical studies of stem cell–based therapies show encouraging results, further progress requires clear product classification, pediatric-specific trial designs, standardized manufacturing protocols, and robust ethical frameworks to ensure appropriate protection of children. Importantly, HSCT remains the only stem cell-based modality with established clinical benefit for several pediatric indications, whereas most regenerative stem cell applications remain investigational.