肿瘤细胞治疗研究
英文原题：CAR T cell therapy for central nervous system solid tumors: current progress and future directions.
对于高危 CNS 肿瘤患者，标准治疗往往无效，生存率低于 10%。
中枢神经系统（CNS）肿瘤是儿童中第二常见的癌症，也是儿科肿瘤患者死亡的首要原因。对于高危CNS肿瘤患者，标准治疗往往无效，生存率低于10%。因此，亟需为这类患者开发替代治疗策略。全球有大量临床试验正在积极研究多种新型治疗方法，包括药物和免疫疗法，以及直接作用于肿瘤的物理疗法。在这些新兴疗法中，CAR-T细胞疗法显示出巨大潜力，已报告首批客观临床应答。本综述评估儿童CNS肿瘤CAR-T细胞疗法的现状，重点关注临床疗效、全身和局部给药的毒性特征、抗原异质性及临床应用的关键挑战。我们全面分析已报告的临床试验，包括CAR-T细胞研究，以及TIL（肿瘤浸润淋巴细胞）、NK细胞和淋巴因子激活的杀伤（LAK）细胞研究，从而更广泛地呈现CNS恶性肿瘤的免疫治疗方法。
Central nervous system (CNS) tumors are the second most common type of cancer in children and remain the leading cause of mortality in pediatric oncology. For patients with high-risk CNS tumors, standard treatments often prove ineffective, with survival rates being less than 10%. Hence, there is an urgent need to develop alternative treatment strategies for this patient population. Globally, numerous clinical trials are actively investigating a range of novel therapeutic approaches, from pharmacological and immunological therapies to physical modalities targeting the tumor. Among these emerging therapies, CAR T cell therapy has shown great promise, with the first objective clinical responses already reported. This review aims to evaluate the current landscape of CAR T cell therapy for pediatric CNS tumors, focusing on clinical efficacy, toxicity profiles of systemic and locoregional delivery, antigen heterogeneity, and key challenges in clinical implementation. We provide a comprehensive analysis of reported clinical trials, including not only CAR T cell studies but also investigations involving tumor-infiltrating lymphocytes (TILs), NK and lymphokine-activated killer (LAK) cells, offering a broader perspective on immunotherapeutic approaches for CNS malignancies.
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