Suh Sang Jun, Yim Hyung Joon
Department of Internal Medicine, Korea University College of Medicine, Seoul, Korea.
Korean J Gastroenterol. 2013 Mar 25;61(3):136-46. doi: 10.4166/kjg.2013.61.3.136.
Hepatocellular carcinoma (HCC) is one of the leading causes of cancer death in Korea. Curative treatment is only possible when the disease is diagnosed at the early stage. The prognosis of patients with HCC is even dismal in advanced stages. No systemic cytotoxic chemotherapy has proven to be beneficial in overall survival. Recently, the understanding of the molecular pathogenesis led to the development of new therapies. With the evidence of dysregulation of critical genes associated with cellular proliferation, growth factor signaling, cell cycling, apoptosis, and angiogenesis in HCC, a number of molecular target agents are under clinical trials. Sorafenib is the first systemic anticancer drug which has proven to gain survival benefit in the global as well as Asia-Pacific trials. However, the survival gain is still modest, and further efforts to improve outcomes in patients with HCC are necessary by developing novel drugs or combining other forms of therapies. This article will review signaling pathways in HCC and introduce molecular target agents under investigation currently.
肝细胞癌（HCC）是韩国癌症死亡的主要原因之一。只有在疾病早期被诊断出来时，才有可能进行根治性治疗。HCC患者在晚期的预后甚至很糟糕。没有任何全身性细胞毒性化疗已被证明对总体生存有益。最近，对分子发病机制的了解促使了新疗法的开发。鉴于有证据表明与HCC中细胞增殖、生长因子信号传导、细胞周期、细胞凋亡和血管生成相关的关键基因失调，许多分子靶向药物正在进行临床试验。索拉非尼是第一种在全球以及亚太地区试验中已被证明能带来生存益处的全身性抗癌药物。然而，生存获益仍然有限，有必要通过开发新药或联合其他形式的疗法来进一步努力改善HCC患者的治疗效果。本文将综述HCC中的信号通路，并介绍目前正在研究的分子靶向药物。
