Cancer Program, Broad Institute, Cambridge, Massachusetts, USA.
Semin Liver Dis. 2010 Feb;30(1):35-51. doi: 10.1055/s-0030-1247131. Epub 2010 Feb 19.
Hepatocellular carcinoma (HCC) is one of most lethal cancers worldwide. Strategic decisions for the advancement of molecular therapies in this neoplasm require a clear understanding of its molecular classification. Studies indicate aberrant activation of signaling pathways involved in cellular proliferation (e.g., epidermal growth factor and RAS/mitogen-activated protein kinase pathways), survival (e.g., Akt/mechanistic target of rapamycin pathway), differentiation (e.g., Wnt and Hedgehog pathways), and angiogenesis (e.g., vascular endothelial growth factor and platelet-derived growth factor), which is heterogeneously presented in each tumor. Integrative analysis of accumulated genomic datasets has revealed a global scheme of molecular classification of HCC tumors observed across diverse etiologic factors and geographic locations. Such a framework will allow systematic understanding of the frequently co-occurring molecular aberrations to design treatment strategy for each specific subclass of tumors. Accompanied by a growing number of clinical trials of molecular targeted drugs, diagnostic and prognostic biomarker development will be facilitated with special attention on study design and with new assay technologies specialized for archived fixed tissues. A new class of genomic information, microRNA dysregulation and epigenetic alterations, will provide insight for more precise understanding of disease mechanism and expand the opportunity of biomarker/therapeutic target discovery. These efforts will eventually enable personalized management of HCC.
肝细胞癌(HCC)是全球最致命的癌症之一。为了推进该肿瘤的分子治疗,需要明确其分子分类。研究表明,细胞增殖(如表皮生长因子和 RAS/丝裂原活化蛋白激酶途径)、存活(如 Akt/雷帕霉素靶蛋白途径)、分化(如 Wnt 和 Hedgehog 途径)和血管生成(如血管内皮生长因子和血小板衍生生长因子)等信号通路的异常激活在每个肿瘤中都呈现异质性。对累积的基因组数据集的综合分析揭示了 HCC 肿瘤的分子分类的总体方案,该方案在不同的病因和地理位置都有观察到。这样的框架将有助于系统地了解经常同时发生的分子异常,为每个特定的肿瘤亚类设计治疗策略。随着越来越多的分子靶向药物临床试验的开展,诊断和预后生物标志物的开发将得到促进,特别关注研究设计和专门针对存档固定组织的新检测技术。一类新的基因组信息,即 microRNA 失调和表观遗传改变,将为更精确地了解疾病机制提供依据,并为生物标志物/治疗靶点的发现提供更多机会。这些努力最终将能够实现 HCC 的个体化管理。