Samkari Ayman, White Jason C, Packer Roger J
Department of Pediatrics, Drexel University College of Medicine, Philadelphia, PA; Section of Oncology, St Christopher׳s Hospital for Children, Philadelphia, PA.
Department of Pediatrics, Drexel University College of Medicine, Philadelphia, PA.
Semin Pediatr Neurol. 2015 Mar;22(1):6-13. doi: 10.1016/j.spen.2014.12.010. Epub 2014 Dec 24.
Medulloblastoma is the most common malignant brain tumor in children and, as such, has been the focus of tremendous efforts to genomically characterize it. What was once thought to be a single disease has been divided into multiple, molecularly unique subgroups through gene expression profiling. Each subgroup is not only unique in its origin and pathogenesis but also in the prognosis and potential therapeutic options. Targeted therapy of malignancies has long been the goal of clinical oncology. The progress made in the classification of medulloblastoma should be used as a model for future studies. With the evolution of epigenetic and genomic sequencing, especially when used in tandem with high-throughput pharmacologic screening protocols, the potential for subgroup-specific targeting is closer than ever. This review focuses on the development of the molecular classification system and its potential use in developing prognostic models as well as for the advancement of targeted therapeutic interventions.
髓母细胞瘤是儿童最常见的恶性脑肿瘤，因此一直是对其进行基因组特征描述的大量研究的重点。曾经被认为是单一疾病的髓母细胞瘤，通过基因表达谱分析已被分为多个分子特征独特的亚组。每个亚组不仅在起源和发病机制上独特，在预后和潜在治疗选择方面也各有不同。恶性肿瘤的靶向治疗长期以来一直是临床肿瘤学的目标。髓母细胞瘤分类方面取得的进展应作为未来研究的典范。随着表观遗传学和基因组测序的发展，尤其是当与高通量药理学筛选方案联合使用时，亚组特异性靶向治疗的潜力比以往任何时候都更接近现实。本综述重点关注分子分类系统的发展及其在开发预后模型以及推进靶向治疗干预方面的潜在用途。
