Novel therapeutic targets and predictive markers for hepatocellular carcinoma.

INTRODUCTION: Following the approval of sorafenib as the first systemic agent for treatment of advanced hepatocellular carcinoma (HCC), there have been an increasing number of targeted treatments under testing for the cancer. However, most of the recently published drug trials in HCC failed to produce remarkable results. The researchers are actively pursuing novel therapeutic targets as well as predictive biomarker for treatment of HCC. AREAS COVERED: This review discusses a number of potential novel targets for drug development of HCC. Focus is put on the underlying rationale for therapeutic development of the target and the possibility of using a predictive biomarker to select patients for drug testing. EXPERT OPINION: Future direction of drug development will be discussed. Notably, a clinical trial on drug testing in HCC should be shifted from all-comers approach to selected populations based on underlying viral etiology and molecular targets. A study to evaluate predictive biomarker is crucial to the development of targeted agents for HCC. Design of clinical trials on HCC should introduce measures to encourage acquisition of tumor and plasma samples for biomarker development.