Translational medicine in hepatocellular carcinoma

Abstract

Hepatocellular carcinoma (HCC) is a highly complex disease that is generally resistant to commonly used chemotherapy and radiotherapy. Consequently, there is an urgent need for the development of new treatment strategies for this devastating disease. In the past decade, tremendous progress has been achieved in the molecular stratification of HCCs for diagnosis, prognosis, and therapeutic decision-making. To date, the molecular classification of HCCs has been carried out through transcriptomic, genetic and epigenetic profiling of tumors. Such research has led to identification of several potential molecular targets in HCC, and subsequently, development of novel systemic agents for the treatment of HCC has begun in earnest. In this article, we review the current knowledge of the molecular pathogenesis of HCC and outline potential areas for application of this knowledge in a clinical setting. As a typical virus and inflammation-associated cancer, both host immune response and tumor microenvironment have crucial roles in HCC pathogenesis. In addition, we examine the potential of immunotherapy and strategies targeting various components of the tumor microenvironment, as well as novel molecular and cellular targets in HCC such as cancer stem cells.

Keywords

hepatocellular carcinoma / molecular classification / molecular targeted therapies / tumor microenvironment / immunotherapy

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Acknowledgements

This work was supported by the Major Program of National Natural Science Foundation of China (No. 81030038), National Key Sci-Tech Project (No. 2008ZX10002-019), FANEDD (No. 201183), Shanghai Rising-Star Program (No. 10QA1401300), National Natural Science Foundation of China (No. 81071992 & No. 30901432), and “Chen Guang” project supported by Shanghai Municipal Education Commission and Shanghai Education Development Foundation (No. 11CG02).

We thank Dr. Yizhou He (Zhongshan Hospital, Fudan University) for his expertise in drawing the Fig. 1.

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2014 Higher Education Press and Springer-Verlag Berlin Heidelberg