Background Brain tumors are the most common solid tumors and the leading cause of cancer-related deaths in children. Incidence in the US has been on the rise for the last 2 decades. While therapeutic advances in diagnosis and treatment have improved survival and quality of life in many children, prognosis remains poor and current treatments have significant long-term sequelae. Summary There is a substantial need for the development of new therapeutic approaches, and since the introduction of immunotherapy by immune checkpoint inhibitors there has been an exponential increase in clinical trials to adopt these and other immunotherapy approaches in children with brain tumors. In this review, we summarize the current immunotherapy landscape for various pediatric brain tumor types including choroid plexus tumors, embryonal tumors (medulloblastoma, AT/RT, PNETs), ependymoma, germ cell tumors, gliomas, glioneuronal and neuronal tumors, and mesenchymal tumors. We discuss the latest clinical trials and noteworthy preclinical studies to treat these pediatric brain tumors using immune checkpoint inhibitors, cellular therapies (CAR-T, NK, T cell), oncolytic virotherapy, radioimmunotherapy, tumor vaccines, immunomodulators, and other targeted therapies. Key Messages The current landscape for immunotherapy in pediatric brain tumors is still emerging, but results in certain tumors have been promising. In the age of targeted therapy, genetic tumor profiling, and many ongoing clinical trials, immunotherapy will likely become an increasingly effective tool in the neuro-oncologist armamentarium.


中文翻译：

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儿科脑和脊柱肿瘤的免疫治疗、现状和未来方向。

背景 脑肿瘤是最常见的实体瘤，也是儿童癌症相关死亡的主要原因。过去 20 年来，美国的发病率一直在上升。虽然诊断和治疗方面的治疗进展提高了许多儿童的生存率和生活质量，但预后仍然很差，并且目前的治疗方法会产生严重的长期后遗症。摘要 迫切需要开发新的治疗方法，并且自从免疫检查点抑制剂引入免疫疗法以来，在患有脑肿瘤的儿童中采用这些和其他免疫治疗方法的临床试验呈指数增长。在这篇综述中，我们总结了当前各种儿科脑肿瘤类型的免疫治疗前景，包括脉络丛肿瘤、胚胎肿瘤（髓母细胞瘤、AT/RT、PNET）、室管膜瘤、生殖细胞肿瘤、神经胶质瘤、胶质神经元和神经元肿瘤以及间充质肿瘤。我们讨论使用免疫检查点抑制剂、细胞疗法（CAR-T、NK、T 细胞）、溶瘤病毒疗法、放射免疫疗法、肿瘤疫苗、免疫调节剂和其他靶向疗法治疗这些儿童脑肿瘤的最新临床试验和值得注意的临床前研究。关键信息 目前儿科脑肿瘤免疫治疗的前景仍在不断显现，但某些肿瘤的结果令人鼓舞。在靶向治疗、遗传肿瘤分析和许多正在进行的临床试验的时代，免疫疗法可能会成为神经肿瘤学家的武器库中越来越有效的工具。