Hepatocellular carcinoma: Will novel targeted drugs really impact the next future?

Core Tip

Core tip: Hepatocellular carcinoma (HCC) is a tumor with increasing incidence and epidemiologic relevance. Advanced hepatocellular carcinoma that is not amenable to radical treatments (i.e., transplantation or surgical resection) has a dismal prognosis (1-2 mo). Sorafenib, a tyrosine kinase inhibitor which targets multiple pro-angiogenic factors, is a cornerstone in the history of HCC treatments. Since the introduction of sorafenib, novel biological drugs have been investigated in hepatocellular carcinoma patients, but no monotherapy or combination therapy has significantly improved outcomes in clinical trials. Insights into tumor gene profile are critical in recognizing various classes of hepatocellular carcinoma in order to help determine which therapeutic approaches will be beneficial. Well-designed clinical trials may disclose differences in efficacy end-points, thus leading the way to clinical use.