# Cell therapy in pediatric blood diseases

## TL;DR

This review discusses cell therapy advancements in treating pediatric blood diseases, highlighting the role of women in science and the potential of new therapies like CAR-T and CAR-NK cells.

## Contribution

The paper provides a comprehensive review of current and emerging cell therapies for pediatric blood diseases, emphasizing their impact and challenges.

## Key findings

- HSCT is the gold standard for high-risk and relapsed pediatric blood diseases.
- CAR-T cell therapy has transformed leukemia treatment but faces challenges like antigen escape and T-cell exhaustion.
- CAR-NK cells are emerging as a promising strategy to improve efficacy and reduce toxicity.

## 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.