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GD2:中枢神经系统肿瘤患儿治疗的希望与挑战

英文原题:GD2: hopes and challenges for the treatment of pediatric patients with tumors of the central nervous system.

PubMed 2025/08/21(内容时间) NPJ Precis Oncol Q1 · IF 9.9(JCR 2025)

研究概要

儿童中枢神经系统(CNS)肿瘤是儿童癌症相关死亡的首要原因,但有效的治疗选择仍然有限。

中文摘要

儿童中枢神经系统(CNS)肿瘤是儿童癌症相关死亡的首要原因,但有效治疗选择仍有限。肿瘤相关抗原 GD2 是治疗儿童 CNS 癌症的有前景免疫治疗靶点;然而,肿瘤内及肿瘤间表达异质性会增加制定有效策略的难度。本文综述 GD2 生物学的不同方面,包括其结构、合成途径及细胞和组织表达,重点关注儿童 CNS 肿瘤。作者详细概述了评估新鲜解离肿瘤样本、组织切片或肿瘤来源细胞系中 GD2 表达,以及将其作为液体活检循环标志物的研究和诊断方法。此外,综述全面介绍基于 GD2 的治疗策略,包括单克隆抗体、CAR-T、适配体、疫苗和多模式方法,涵盖临床前研究至近期临床应用,并强调靶向这些癌症中 GD2 的前景与挑战。

展开英文摘要原文

Pediatric Central Nervous System (CNS) tumors are the leading cause of cancer-related death in children, yet effective treatment options remain limited. The tumor-associated antigen GD2 is a promising target for immunotherapeutic approaches aimed at treating pediatric CNS cancers; however, its heterogeneous expression within and between tumors can complicate the development of effective strategies. Here we review different aspects of GD2 biology, including its structure, synthesis pathway, and cellular and tissue expression, focusing on pediatric CNS tumors. We provide a detailed overview of the investigational and diagnostic methods for evaluating GD2 expression on freshly dissociated tumor samples, tissue sections, or tumor-derived cell lines, and as a circulating marker in liquid biopsy. Furthermore, we provide a comprehensive overview of GD2-based therapeutic strategies, such as monoclonal antibodies, CAR T-cells, aptamers, vaccines, and multimodal approaches, from preclinical studies to recent clinical applications, highlighting both the promise and challenges of targeting GD2 in these cancers.

论文信息

作者
Fernández-Rilo AC、de Billy E、Del Baldo G、De Angelis B、Rossi S、Del Bufalo F、Quintarelli C、Locatelli F
第一作者单位
Research Area of Onco-haematology and Pharmaceutical GMP Facility, Bambino Gesù Children's Hospital-IRCCS, Rome, Italy.Italy
通讯作者单位
Research Area of Onco-haematology and Pharmaceutical GMP Facility, Bambino Gesù Children's Hospital-IRCCS, Rome, Italy. maria.vinci@opbg.net.Italy
文献类型
综述
期刊
NPJ precision oncology2025 Aug 21
原文标识
PubMed 40835729 · DOI 10.1038/s41698-025-01079-1