Siegel Benjamin I, Patil Prabhumallikarjun, Prakash Akul, Klawinski Darren M, Hwang Eugene I
Brain Tumor Institute and Gilbert Family Neurofibromatosis Institute, Children's National Hospital, Washington, DC, United States.
Division of Oncology, Children's National Hospital, Washington, DC, United States.
Front Oncol. 2025 Feb 28;15:1504803. doi: 10.3389/fonc.2025.1504803. eCollection 2025.
Central nervous system tumors represent the leading cause of cancer-related mortality in children. Conventional therapies of surgery, radiation, and cytotoxic chemotherapy have insufficient efficacy for some pediatric CNS tumors and are associated with significant morbidity, prompting an ongoing need for novel treatment approaches. Identification of molecular alterations driving tumorigenesis has led to a rising interest in developing targeted therapies for these tumors. The present narrative review focuses on recent progress in targeted therapies for pediatric CNS tumors. We outline the key implicated cellular pathways, discuss candidate molecular therapies for targeting each pathway, and present an overview of the clinical trial landscape for targeted therapies in pediatric CNS tumors. We then discuss challenges and future directions for targeted therapy, including combinatorial approaches and real-time drug screening for personalized treatment planning.
中枢神经系统肿瘤是儿童癌症相关死亡的主要原因。手术、放疗和细胞毒性化疗等传统疗法对某些儿童中枢神经系统肿瘤疗效不足，且伴有明显的发病率，这促使人们持续需要新的治疗方法。对驱动肿瘤发生的分子改变的识别，引发了人们对开发这些肿瘤靶向治疗的兴趣日益浓厚。本叙述性综述重点关注儿童中枢神经系统肿瘤靶向治疗的最新进展。我们概述了关键的相关细胞途径，讨论了针对每条途径的候选分子疗法，并概述了儿童中枢神经系统肿瘤靶向治疗的临床试验情况。然后，我们讨论了靶向治疗的挑战和未来方向，包括联合治疗方法和用于个性化治疗规划的实时药物筛选。
