Park Jeong A, Cheung Nai-Kong V
Department of Pediatrics, Inha University Hospital, Inha University College of Medicine, Incheon, Korea.
Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA
Cold Spring Harb Perspect Med. 2025 Jun 2;15(6):a041574. doi: 10.1101/cshperspect.a041574.
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.
如今，患癌后存活下来的儿童数量比以往任何时候都多。在过去50年里，儿科癌症治疗取得了重大进展，5年生存率达到了85%。尽管如此，仍有10%-15%的患者出现复发或发展为难治性疾病，导致生存率显著降低。最近试图进一步强化细胞毒性化疗的尝试，要么因严重毒性，要么因无效而失败，这凸显了新治疗策略的必要性。免疫疗法正在兴起，并将其临床应用扩展到包括儿童癌症在内的多种癌症。在儿科癌症中，靶向GD2的单克隆抗体在神经母细胞瘤（NB）中显示出持久的影像学和组织学反应，而靶向CD19的双特异性抗体（BsAbs）和嵌合抗原受体（CAR）T细胞同样改变了儿童难治性急性淋巴细胞白血病（ALL）的前景。本综述讨论了儿科癌症免疫疗法的临床发展，重点关注儿科ALL和NB这两种因免疫疗法而发生转变的主要儿科癌症，介绍了免疫疗法的最新进展，并进一步探讨了儿科癌症免疫疗法的未来方向。
