Division of Hematology/Oncology, Geffen School of Medicine at the University of California at Los Angeles, Los Angeles, California, USA.
Clin Cancer Res. 2010 Jan 15;16(2):390-7. doi: 10.1158/1078-0432.CCR-09-2084. Epub 2010 Jan 12.
Hepatocellular carcinoma (HCC), once considered an orphan disease in the West, has become a global health concern. It is the third leading cause of cancer death worldwide, and its incidence continues to increase. Historically, the development of new systemic agents for advanced HCC has been lacking despite no clear benefit with traditional cytotoxic therapies. Although two randomized studies with sorafenib for the treatment of HCC patients have recently been completed, survival benefits have been modest and highlight the unmet medical need among patients with HCC. Given the clear need, clinical development of novel systemic agents in HCC has begun in earnest. These clinical studies are founded on a growing body of basic and translational science that has identified several potential molecular targets in HCC. The successful development of such targeted agents in the future will be linked to our ability to appropriately select patients for treatment based on their clinical stage (including extent of liver disease and extent of tumor) and on potential predictive markers of response. Here, we review these data in the context of rational drug development in HCC in the front-line setting and in previously treated patients.
肝细胞癌（HCC）曾被认为是西方的一种罕见疾病，但现已成为全球关注的健康问题。它是全球癌症死亡的第三大主要原因，其发病率持续上升。尽管传统细胞毒性疗法没有明显获益，但历史上晚期 HCC 的新型全身治疗药物的发展一直缺乏。尽管最近完成了两项索拉非尼治疗 HCC 患者的随机研究，但生存获益有限，突显了 HCC 患者存在未满足的医疗需求。鉴于这种明显的需求，HCC 的新型全身治疗药物的临床开发已认真开展。这些临床研究建立在日益增多的基础和转化科学研究基础上，这些研究确定了 HCC 中的几个潜在分子靶点。未来这些靶向药物的成功开发将取决于我们根据患者的临床分期（包括肝脏疾病的严重程度和肿瘤的范围）和反应的潜在预测标志物，适当选择接受治疗的患者的能力。在这里，我们根据一线治疗和既往治疗患者的 HCC 合理药物开发的背景来审查这些数据。
