Experience and Expectation for Molecular Target Therapy in Hepatocellular Carcinoma

Kim, Do Young

Abstract

Sorafenib, the first-approved molecular targeted agent (MTA), is actively used in patients with advanced hepatocellular carcinoma (HCC) worldwide. Accumulating experiences suggest that the efficacy of this drug is modest and the adverse events are not minimal, although those are manageable. Given that newer drugs targeting various molecules involved in hepatocarcinogenesis have been failed to show efficacy compared with sorafenib, the perspectives regarding development of novel MTA appear to be gloomy. Nonetherless, it is necessary to maximize the efficacy of sorafenib by combining this drug with locoregional therapies and by finding biomarkers predicting the outcomes of patients treated with sorafenib. A series of data indicate that combining sorafenib and locoregional therapies including transarterial chemoemolization could improve patients outcome. Unfortunately, there has been no biomarkers identified which could predict the response to sorafenib. Global investigation of therapeutic decisions in HCC and of its treatment with sorafeNib (GIDEON) is a global, non-interventional, observational study to evaluate the safety and efficacy of sorafenib, particularly in patients with Child-Pugh B. The final report of GIDEON study is going to be published soon, and the Korean GIDEON data suggest that sorafenib in patients with decreased liver function might be safe and effective as in patients with Child-Pugh A. The way to conquer HCC using molecular approach is still long and the era of immune-oncology seems to get started.