Temsirolimus is a mTOR inhibitor. It is an FDA approved for renal cell carcinoma. The mTOR pathway is activated in 40-50% of patients with hepatocellular cancer (HCC). In different models (i.e., hepatoma cell lines and implanted HCC tumors in rats), mTOR inhibitors (mTORIs) were effective in reducing cell growth and tumor vascularity. Synergistic effects were observed for mTORIs and chemotherapeutic agents in these studies. Clinical data are, as yet, only preliminary and are mainly derived from retrospective studies in patients who underwent liver transplantation for HCC. Those patients had received sirolimus thereafter for immunosuppression, and a much lower rate of tumor recurrence than with calcineurin inhibitors alone was noted. Current prospective trials of MTOR inhibitors are ongoing for treatment of advanced HCC .
A similar drug is in a current trial: Randomized Phase I/II of RAD001 in Advanced Hepatocellular Carcinoma (HCC), NCT00390195
Treiber, Gerhard, mTOR inhibitors for hepatocellular cancer: a forward-moving target
Expert Review of Anticancer Therapy, Volume 9, Number 2, February 2009 , pp. 247-261(15)
P. Newell, A. Villanueva, J. Llovet, Molecular targeted therapies in hepatocellular carcinoma: From pre-clinical models to clinical trials, Journal of Hepatology, Volume 49, Issue 1, Page 1
Josep M. Llovet Novel advancements in the management of hepatocellular carcinoma in 2008
Journal of Hepatology Volume 48, Supplement 1, 2008, Pages S20-S37 Management of Liver Diseases 2008