Telotristat Ethyl and First-Line Chemotherapy Combination Investigated for Advanced Biliary Tract Cancer

December 2020, Vol 1, No 3

The metabolism of serotonin is dysregulated in cholangiocarcinoma (CCA) cell lines compared with normal cholangiocytes and in tissue and bile from patients with CCA.1 Telotristat ethyl (Xermelo) is a tryptophan hydroxylase inhibitor that is indicated for the treatment of patients with carcinoid syndrome–related diarrhea not well-controlled by somatostatin analog. Telotristat ethyl is currently being investigated for the treatment of patients with biliary tract cancer, including CCA tumors that express serotonin.

Richard Kim, MD, Section Chief, Gastrointestinal Medical Oncology, Moffitt Cancer Center, Tampa, FL, presented early results of a phase 2, multicenter, open-label clinical trial in patients with biliary tract cancer. Dr Kim and colleagues are studying the safety of telotristat ethyl (500 mg 3 times daily) plus standard chemotherapy with gemcitabine and cisplatin for the treatment of advanced, unresectable, or recurrent metastatic biliary tract cancer in patients whose tumors express serotonin.

“Based on the first 6 patients, there were no other safety signals with regard to dose-limiting toxicity that will preclude us from continuing on with the trial. Therefore, our conclusion is that the combination of telotristat ethyl and standard chemotherapy was very well-tolerated,” Dr Kim stated.


  1. Alpini G, Invernizzi P, Gaudio E, et al. Serotonin metabolism is dysregulated in cholangiocarcinoma, which has implications for tumor growth. Cancer Res. 2008;68:9184-9193.

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