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Hepatocellular carcinoma (HCC) is the most frequent liver cancer and one of the most common causes of cancer-related death. The rates of incidence and mortality of HCC are increasing worldwide. Unfortunately, both figures are very similar mainly due to the longer survival of patients with cirrhosis, the aggressiveness of HCC and the lack of efficient therapies.1 ,2 Since 2008, sorafenib, a multikinase (BRAF/vascular endothelial growth factor receptor/platelet-derived growth factor receptor) inhibitor is the only approved systemic therapy for advanced HCC. Sorafenib offers modest survival benefit, and recently some concerns about its cost-effectiveness have been raised.3 In the last years, many other molecular targeted agents have been tested for the treatment of HCC in different clinical trials with negative results.2 ,4
The limited efficacy of sorafenib and the rest of the molecular therapies assayed against HCC can be linked to the existence of primary and the emergence of acquired drug resistance mechanisms.5 However, it is becoming evident that the main reason for the failure of targeted therapies in HCC is their administration to an unselected population of patients due to the lack of specific biomarkers.
Precision medicine is characterised by targeting a specific molecular alteration, which in the case of cancer is supposed to dominate the malignant phenotype. The primary success of a targeted therapy for a particular tumour will rely mainly on a correct process of identification and …
Competing interests None declared.
Provenance and peer review Commissioned; internally peer reviewed.