Medulloblastoma (MB) is the most common childhood brain tumor, which occurs in the posterior fossa. MB tumors are highly heterogeneous and have diverse genetic make-ups, with differential microRNA (miRNA) expression profiles and variable prognoses. MB can be classified into four subgroups, each with different origins, pathogenesis, and potential therapeutic targets. miRNA and small-molecule targeted therapies have emerged as a potential new therapeutic paradigm in MB treatment. However, the development of chemoresistance due to surviving cancer stem cells and dysregulation of miRNAs remains a challenge. Combination therapies using multiple drugs and miRNAs could be effective approaches. In this review we discuss various MB subtypes, barriers, and novel therapeutic options which may be less toxic than current standard treatments.

髓母细胞瘤（MB）是儿童期最常见的脑肿瘤，好发于后颅窝。MB肿瘤高度异质，具有多种遗传组成，具有不同的microRNA（miRNA）表达谱和可变的预后。MB可分为四个亚组，每个亚组具有不同的起源，发病机理和潜在的治疗靶标。miRNA和小分子靶向疗法已成为MB治疗中潜在的新治疗范例。然而，由于存活的癌症干细胞和miRNA的失调而引起的化学抗药性的发展仍然是一个挑战。使用多种药物和miRNA的联合疗法可能是有效的方法。在这篇综述中，我们讨论了各种MB亚型，屏障和新颖的治疗选择，它们的毒性可能低于当前的标准治疗方法。