Molecular cancer prevention: Phase IIa trial of Erlotinib for IPMNs and the prevention of pancreatic cancer.

CS04-03 Intraductal Papillary Mucinous Neoplasms (IPMNs) are a distinct type of precursor lesions that can progress to pancreatic adenocarcinoma. IPMNs are grossly visible, mucin-producing, predominantly papillary or rarely flat epithelial neoplasms arising from the main pancreatic duct or branch ducts. Recently there has been a sharp increase in the number of IPMN cases although the reasons for this increase are not clear. Erlotinib is an Epidermal Growth Factor Receptor (EGFR) inhibitor that prolongs survival in combination with gemcitabine in patients with advanced pancreatic cancer. Based on the observation that there is a significant pancreatic adenocarcinoma regression with Erlotinib therapy, Erlotinib may analogously cause IPMN regression. We are conducting a one arm Phase IIa trial of Erlotinib in IPMNs. This exploratory study is designed to test whether Erlotinib treatment can effectively decrease expression of EGF dependent biomarkers in IPMNs. If successful, this study will provide a firm rationale for future randomized trials to test whether Erlotinib can cause clinical regression of IPMNs before they progress to pancreatic cancer and decrease the overall number of patients developing frank pancreatic adenocarcinoma.