Potential applications of umbilical cord blood-derived cells in neonatal diseases.

Abstract

Umbilical cord blood (UCB) has garnered attention as a potentially valuable resource in neonatal medicine. Blood from the cord contains a rich population of stem and progenitor cells, including hematopoietic stem cells, mesenchymal stromal cells, and endothelial progenitor cells, each offering unique therapeutic potential. Early clinical studies and preclinical trials have suggested that these cells may be able to promote tissue repair, provide neuroprotection, and modulate the immune system, potentially providing promising therapeutic avenues for neonates with limited treatment options. Current research has focused on the safety, feasibility, and preliminary efficacy of UCB-based therapies, particularly in neonates with brain injuries, lung diseases, and hematological disorders. Despite positive early results, the field is still evolving, and large-scale clinical trials are underway to further evaluate the potential of UCB-derived cells in neonates. In this review, we examine the diverse roles of UCB-derived cells in potentially treating neonatal diseases such as anemia, intraventricular hemorrhage, hypoxic-ischemic encephalopathy, and bronchopulmonary dysplasia. We also discuss future directions for the application of UCB-derived cells in transfusion and regenerative medicine.Copyright © 2025 by the American Academy of Pediatrics.