Cell therapy in pediatric blood diseases

Abstract

The fight against mortality in pediatric oncohematological diseases is a story of human rights, academia, innovation, and technological advancement—one in which women in science have played a pivotal role. With talent, perseverance, and sensitivity, they greatly contributed to research in new cellular therapies, expanding the frontiers of treatment and giving the most vulnerable patients the gift of more years of life. This review explores multipotent and unipotent cell therapies, focusing on hematopoietic stem cell transplantation (HSCT), mesenchymal stromal cell transplantation (MSCT), and chimeric antigen receptor (CAR)-T cell and natural killer (NK) cell therapy. HSCT remains the gold standard for high-risk and relapsed cases, with graft sources including bone marrow (BM), mobilized peripheral blood (MPB), and umbilical cord blood (UCB). Advances in MSCT highlight its role in hematopoiesis support and immunomodulation, reducing graft-versus-host disease (GVHD) risks. CAR-T cell therapy has revolutionized leukemia treatment, although challenges such as antigen escape, T-cell exhaustion, and treatment resistance persist. Emerging strategies, including CAR-NK cells, seek to enhance efficacy while minimizing toxicity. Despite these advancements, cell therapy remains complex and resource-intensive, necessitating further innovations for broader accessibility, particularly in developing regions.