Immunotherapies for childhood cancer Review


Authors: Park, J. A.; Cheung, N. K. V.
Review Title: Immunotherapies for childhood cancer
Abstract: Children are surviving cancer in greater numbers than ever. Over the last 50 years, substantial advancements in pediatric cancer treatment have resulted in an 85% 5-year survival rate. Nonetheless, a notable 10%-15% of patients encounter relapse or develop refractory disease, leading to significantly lower survival. Recent attempts to further intensify cytotoxic chemotherapy have failed due to either severe toxicities or ineffectiveness, highlighting the need for new treatment strategies. Immunotherapies are emerging and expanding their clinical application to a wide array of cancers, including those affecting children. In pediatric cancers, monoclonal antibodies targeting GD2 have demonstrated durable radiographic and histologic responses in neuroblastoma (NB), and CD19-targeted bispecific antibodies (BsAbs) and chimeric antigen receptor (CAR) T cells have likewise changed the outlook for refractory acute lymphoblastic leukemia (ALL) in children. This review discusses the clinical development of immunotherapies for pediatric cancers, focusing on pediatric ALL and NB, two major pediatric cancers transformed by immunotherapy, updates on the recent advancements in immunotherapies, and further discusses the future directions of immunotherapy for pediatric cancers. Copyright © 2025 Cold Spring Harbor Laboratory Press; all rights reserved.
Keywords: child; neoplasm; neoplasms; immunology; immunotherapy; neuroblastoma; chimeric antigen receptor; precursor cell lymphoblastic leukemia-lymphoma; therapy; adoptive immunotherapy; immunotherapy, adoptive; procedures; antibodies, bispecific; bispecific antibody; humans; human; b cell acute lymphoblastic leukemia; receptors, chimeric antigen
Journal Title: Cold Spring Harbor Perspectives in Medicine
Volume: 15
Issue: 6
ISSN: 2157-1422
Publisher: Cold Spring Harbor Laboratory Press  
Date Published: 2025-06-01
Start Page: a041574
Language: English
DOI: 10.1101/cshperspect.a041574
PUBMED: 39009446
PROVIDER: scopus
PMCID: PMC12128874
DOI/URL:
Notes: Review -- MSK Cancer Center Support Grant (P30 CA008748) acknowledged in PubMed and PDF -- MSK corresponding author is Nai-Kong Cheung -- Source: Scopus
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  1. Nai-Kong Cheung
    654 Cheung