Introduction: Sorafenib is a novel oral bis-aryl urea compound that has proven survival benefit in patients with advanced hepatocellular carcinoma (HCC), for which several therapies are currently available with unsatisfactory results. Sorafenib is the first compound to demonstrate a significant effect on survival in HCC. Areas covered: Our experience in the clinical application of sorafenib is reviewed in conjunction with relevant publications in the literature.
Based on ex vivo and in vivo experiments, we conclude that sorafenib plays an important role in blocking tumor cell proliferation, inducing apoptosis, and reversing multidrug resistance. Antitumor activities were observed in multiple tumor types in ongoing randomized Phase III studies. Toxicity was observed as tolerable. On 16 November 2007, sorafenib was approved by the United States Food and Drug Administration for the treatment of unresectable HCC, based on the results of an international, multicenter, randomized, double-blind, placebo-controlled trial in patients with unresectable, biopsy-proven HCC.
Expert opinion: With the approval of sorafenib being given the significance of a milestone, systemic treatment of HCC is no longer regarded as ineffective. The tyrosine kinase inhibitor, sorafenib, gives us new hope for successful cancer therapy.