Medulloblastoma (MB) is a major pediatric malignant brain tumor that arises in the cerebellum. MB tumors exhibit highly heterogeneous driven by diverse genetic alterations and could be divided into four major subgroups based on their different biological drivers and molecular features (Wnt, Sonic hedgehog (Shh), group 3, and group 4 MB). Even though the therapeutic strategies for each MB subtype integrate their pathogenesis and were developed to focus on their specific target sites, the unexpected drug non-selective cytotoxicity, low drug accumulation in the brain, and complexed MB tumor microenvironment still be huge obstacles to achieving satisfied MB therapeutic efficiency. This review discussed the current advances in modern MB therapeutic strategy development. Through the recent advances in knowledge of the origin, molecular pathogenesis of MB subtypes and their current therapeutic barriers, we particularly reviewed the current development in advanced MB therapeutic strategy committed to overcome MB treatment obstacles, focusing on novel signaling pathway targeted therapeutic agents and their combination discovery, advanced drug delivery systems design, and MB immunotherapy strategy development.

髓母细胞瘤（MB）是一种主要的小儿恶性脑肿瘤，发生于小脑。MB 肿瘤表现出由不同遗传改变驱动的高度异质性，并且根据其不同的生物驱动因素和分子特征可分为四个主要亚组（Wnt、 Sonic Hedgehog (Shh)、第 3 组和第 4 组 MB）。尽管每种MB亚型的治疗策略都整合了其发病机制并针对其特定靶位点而开发，但意想不到的药物非选择性细胞毒性、脑内低药物蓄积以及复杂的MB肿瘤微环境仍然是实现满意治疗的巨大障碍。 MB治疗效率。本综述讨论了现代 MB 治疗策略开发的当前进展。通过对MB亚型的起源、分子发病机制及其当前治疗障碍的了解的最新进展，我们特别回顾了致力于克服MB治疗障碍的先进MB治疗策略的当前发展，重点关注新型信号通路靶向治疗药物及其组合发现、先进药物输送系统设计和 MB免疫治疗策略开发。