IMPLEMENTATION AND DEVELOPMENT OF CAR-T THERAPY IN PEDIATRIC ONCOHEMATOLOGY: GLOBAL EXPERIENCE AND PROSPECTS FOR EMERGING HEALTHCARE SYSTEMS

Abstract

Chimeric antigen receptor (CAR) T-cell therapy has revolutionized the treatment of pediatric B-cell acute lymphoblastic leukemia (ALL), offering hope in relapsed or refractory (r/r) cases that previously had dismal outcomes. Pediatric ALL is a global health challenge – it is the most common childhood cancer, and while cure rates exceed 85–90% in high-income countries, the majority of children with ALL live in low- and middle-income countries (LMICs) where survival remains much lower[1][2]. The emergence of CAR-T therapy, marked by the first FDA approval in 2017 for r/r pediatric ALL, represents a game-changer in bridging this survival gap. This review (2020–2025 literature) examines worldwide CAR-T implementation, including FDA/EMA-approved therapies and their dissemination in North America, Europe, and Asia, as well as indigenous CAR-T programs in countries like China and India. We discuss the major challenges impeding global adoption: high treatment costs (often >$400,000 per infusion), complex logistics (requirement for specialized cell manufacturing and accredited centers), regulatory hurdles, and ethical considerations of equity. Case studies of CAR-T introduction in emerging healthcare systems are highlighted, such as academic point-of-care manufacturing in Kazakhstan and collaborative platforms like the WHO–St. Jude Global initiative. Strategies for building capacity in resource-limited settings are reviewed – including technology transfer, regional centers of excellence, and exploration of allogeneic “off-the-shelf” CAR immune cells to reduce infrastructure demands. In conclusion, we outline a roadmap for integrating CAR-T into pediatric oncology networks of Central Asia and similar regions, emphasizing sustainable approaches to ensure this innovative therapy benefits children worldwide.