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Research Article Open access CC BY 4.0

CAR-T Cell Therapy in Pediatric Acute Lymphoblastic Leukemia: From Innovations to Continuing Challenges and Future Perspectives

Masco M, Shabnam F, Ramesh K, Swathi G, Kalaivani R, Nepolean R

Journal of Pharmaceutical Research International · pp. 99–113 · Published 7 Nov 2025

10.9734/jpri/2025/v37i117769

Abstract

The most prevalent cancer in children is acute lymphoblastic leukemia (ALL), and relapsed or refractory illness still poses a significant therapeutic challenge. Because chimeric antigen receptor T-cell (CAR-T) therapy reroutes autologous T cells to identify and eliminate cancerous cells, it has revolutionized the way hematologic malignancies are treated. The mechanics, production, and clinical results of CAR-T-cell therapy in pediatric ALL are highlighted in this review, along with its effectiveness, safety, and new approaches to dealing with toxicity and resistance. The therapeutic promise has been further increased by recent advancements such as in vivo CAR engineering, dual-target CARs, and biologic prophylaxis. CAR-T-cell therapy is still developing as a customized and potentially curative treatment for kids with relapsed or refractory ALL, despite obstacles such cytokine release syndrome, neurotoxicity, and production costs. Conclusion: CAR-T-cell therapy represents a groundbreaking step in pediatric leukemia management, offering renewed hope and paving the way for safer, more accessible next-generation immunotherapies.

CAR-T-cell therapy pediatric leukemia immunotherapy cytokine release syndrome neurotoxicity

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