Decoding hepatocellular carcinoma: the promise of microRNAs

Hepatocellular carcinoma (HCC) is the most common primary liver cancer with rising incidence and mortality worldwide. While advances in surgical, transplantation, and regional therapies have made an impact in treatment, the highly vascular nature of the tumor and tendency to metastasize make systemic treatment necessary for the majority of patients. In the past five years, molecularly targeted therapies have been developed with modest success. Most notably, sorafenib (Nexavar), an oral multi-kinase inhibitor of the vascular endothelial growth factor receptor (VEGFR) demonstrated improved survival (1). Unfortunately, the benefit of sorafenib is short-lived, and despite numerous clinical trials, no other VEGF-targeting agent has clearly shown efficacy in randomized studies (2-4). Therefore, it is essential to understand drivers of HCC other than the classic VEGF pathway