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Treatment of advanced pancreatic neuroendocrine tumors: potential role of everolimus

Authors Cen P, Amato R

Received 17 September 2011

Accepted for publication 26 October 2011

Published 3 April 2012 Volume 2012:5 Pages 217—224

DOI https://doi.org/10.2147/OTT.S16289

Review by Single-blind

Peer reviewer comments 4


Putao Cen, Robert J Amato
Division of Oncology, Department of Internal Medicine, University of Texas Health Science Center at Houston (Medical School), Houston, TX

Abstract: Pancreatic neuroendocrine tumors (PanNETs) are frequently diagnosed at unresectable stage and remain a medical challenge. Everolimus (RAD001, Afinitor®, Novartis, Basel, Switzerland), an orally administered inhibitor of mammalian target of rapamycin (mTOR), was recently approved by the Food and Drug Administration to treat patients with advanced PanNETs. This review will examine the mechanism of action of everolimus, the function of mTOR and its inhibition, PanNETs and the mTOR pathway, and clinical trials of everolimus in PanNETs. Future investigations will focus on everolimus combination therapy to treat PanNETs and the discovery of predictive biomarkers for response to everolimus.

Keywords: pancreatic neuroendocrine tumor, PanNETs, everolimus, RAD001, mTOR inhibitor, biomarker

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