HCC Translational Research

The strategic position of Mount Sinai and its partners in the US and Europe has enabled the establishment of a translational program which is intended to: 1. Identify gene expression profiles and genetic alterations in hepatocellular carcinoma in HCV-cirrhotic patients, and elucidate their diagnostic and prognostic implications. 2. Identify the role of signal transduction pathways in the pathogenesis of HCC, and assess the effect of molecular targeted therapies. To that end, a tissue bank containing ~280 samples was established and six translational research projects were initiated by using the resources of the co-investigators involved in the studies and new personnel incorporated through private funding grants (the Samuel Waxman Cancer Research Foundation and the Bendheim Family Trust).

1. Identify gene expression profiles and genetic alterations in HCC in HCV-cirrhotic patients, and elucidate their diagnostic and prognostic implications. The hypothesis of the project is that the knowledge of gene expression profiles and the somatic genetic alterations of HCC will enable the identification of tumor suppressor genes or oncogenes involved in the initiation/progression of the disease, and will provide markers of early detection and targets for the prevention and treatment of this neoplasm.
2. Identify the role of signal transduction pathways in the pathogenesis of HCC, and assess the effect of molecular targeted therapies.

HCC Clinical Research

A key goal of the Program is to attract to Mount Sinai the key worldwide clinical trials (particularly phase III studies) conducted in HCC, and to establish a strong clinical research team with a collaborative effort among the surgeons, hepatologists, oncologists and the Clinical Trials Office of the Department of Medicine. To that end, the largest randomized controlled trial conducted worldwide (sorafenib vs placebo) has been led from Mount Sinai, which has been the leading center in patient recruitment in the US. In addition, other studies funded by the Pharmaceutical Industry and led by Mount Sinai are currently underway. Finally, a randomized controlled trial to assess the effect of EGFR-inhibition through a tyrosine kinase inhibitor (erlotinib) in the prevention of recurrence after surgical resection has been submitted for NIH funding.

Education and Mentorship

A regular HCC research meetings review progress and research opportunities for the HCC program. These meetings have served as a forum to present new proposals and review results of clinical and translational research. The Program has also provided training opportunities for several international postdoctoral fellows, students and visiting scientists.

A highly successful Postgraduate Course in recognition of the first anniversary of the Mount Sinai Liver Cancer Program was held in June 2006, featuring a range of outstanding international speakers covering the gamut of topics from basic research to new directions in diagnosis and therapy.