Sorafenib and the medical treatment of hepatocellular carcinoma

Abstract

Hepatocellular carcinoma (HCC) is the most frequent form of primary liver tumor. HCC is characterized by its occurrence in a context of chronic liver disease. The burden of HCC is expected to increase in the coming decade, principally because of its rising incidence. Although radical treatments are available when a unique tumor of small size is found, HCC is more often diagnosed at an advanced stage. Classical schemes for the treatment of advanced HCC have been recently challenged with the introduction of sorafenib (Nexavar^®), a new reference treatment for advanced HCC. The report of the SHARP clinical trial, showing for the first time the efficacy of sorafenib in advanced HCC, became one of the most cited reports in the cancer literature (Highly cited cancer papers, 2008-2010, Nature Medicine 2011: 17: 280). Sorafenib is an inhibitor of oncogenic kinases that has recently been approved as the reference treatment for advanced HCC. Sorafenib affords a modest gain in survival by delaying the progression of HCC. In the absence of validated biomarkers that would predict its efficacy, several questions pertaining to its indications and the follow-up of this treatment remain unanswered. Here, we review the essential facts about sorafenib and its use in HCC. We also put emphasis on the actual research perspectives that could improve HCC treatment based on sorafenib.