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Case Report

Case Study Case Rep. 2011; 1(3): 137-145

Long-Term Stable Disease of Stage IV Pancreatic Neuroendocrine Tumors and Without Significant Adverse Effect by CPI-613, an Investigational Novel Anti-Cancer Agent

King Lee; Divis Khaira; Robert Rodriguez; Claudia Maturo; Kathleen O&’Donnell; Robert Shorr.

Introduction: The current case study describes the safety and prolonged therapeutic effect of an investigational drug (CPI-613) in a patient (Patient LRW) with Stage IV pancreatic neuroendocrine tumors.

Case presentation: Patient LRW was an 88-year old Caucasian woman who was diagnosed with pancreatic neuroendocrine tumors, and had been treated with Sandostatin followed by Enzastaurin. Prior to treatment with CPI-613, she was suffering from progressive Stage IV disease, with lesions located in multiple areas including the pancreas, liver, lung, uterine, stomach, thyroid nodule, etc. During the one-year treatment with CPI-613, only two incidents of mild (Grade 1) and transient adverse events (metallic taste and hyperuricemia) were observed. The Progression-Free Survival was >15 months (Patient LRW still alive as of June 2011), which is consistent with the reported median Progression-Free Survival of 11.4 months with Everolimus treatment and 11.4-12.6 months with Sunitinib treatment. Since the disease remained stable for a year during treatment with CPI-613, Patient LRW was switched to maintenance therapy with CPI-613 and is currently doing well.

Conclusion: In Patient LRW, who had Stage IV pancreatic neuroendocrine tumor with Disease Progression, CPI-613 therapy provided long-term Stable Disease and with only 2 minor transient incidents of adverse events during a one-year treatment. This is an uncommon case where chemotherapy with an investigational drug is providing long-term stabilization of advanced metastatic disease without significant adverse events.

Key words: pancreatic neuroendocrine tumors;CPI-613

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