The Lynx Group
Cholangiocarcinoma News

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.

Related Items

The Latest Research in Biliary Tract Cancers Presented at ASCO GI 2021
March 2021, Vol 2, No 1
At the CCA Summit held during the 2021 ASCO Gastrointestinal (GI) Cancers Symposium, Rachna T. Shroff, MD, MS, Chief, Section of GI Medical Oncology, University of Arizona Cancer Center, Tucson, discussed 15 clinical trials that were presented at the ASCO GI Cancers Symposium on cholangiocarcinoma (CCA) and hepatobiliary diseases. She highlighted key advances related to chemotherapy, targeted therapies, and biomarkers in the management of biliary tract cancers, including CCA.
What’s Next in Immunotherapy in Biliary Tract Cancers?
March 2021, Vol 2, No 1
The rationale for immunotherapy in biliary tract cancers is strong, because these cancers are inflammatory in nature, according to Abby Siegel, MD, MS, Executive Director and PDT Lead, Hepatobiliary Oncology, Global Clinical Development at Merck. Inroads are being made into biomarkers predictive of response to immunotherapy in biliary cancers, Dr Siegel said at the 2020 Annual Cholangiocarcinoma Summit.
Periadjuvant Approaches to Biliary Tract Cancers Offer Hope for Improved Outcomes
March 2021, Vol 2, No 1
A periadjuvant, or perioperative, approach to the treatment of biliary tract cancers, including cholangiocarcinoma (CCA), has several potential advantages that may offer an opportunity to improve outcomes in these diseases, said James Harding, MD, Regional Director, Early Drug Development, Memorial Sloan Kettering Cancer Center, New York, NY, at the 2020 Annual Cholangiocarcinoma Summit.
The Updated Staging System for Cholangiocarcinoma: Implications for Hilar and Intrahepatic Disease
March 2021, Vol 2, No 1
Flavio Rocha, MD, Hepatopancreatobiliary Surgeon, Virginia Mason Medical Center, Director of Research, Digestive Disease Institute, and Associate Medical Director of the Floyd and Delores Jones Cancer Institute, Seattle, WA, provided an overview of the changes made in the 8th edition of the AJCC’s tumor, node, metastasis (TNM) staging system at the 2020 Annual Cholangiocarcinoma Summit. These changes have direct implications for the management of patients with hilar or intrahepatic CCA.
Targeted Therapies in Cholangiocarcinoma: Next-Generation Sequencing Is a Must
December 2020, Vol 1, No 3
The recent FDA approval of the first FGFR inhibitor, pemigatinib (Pemazyre), and the positive results from the phase 3 study of the first IDH1 inhibitor, ivosidenib (Tibsovo), represent major breakthroughs in the treatment of patients with cholangiocarcinoma (CCA), a rare cancer associated with poor outcomes. However, the duration of response with these agents is still relatively short.
Gallbladder Cancer: Novel Approaches to Therapy Are Needed
December 2020, Vol 1, No 3
The current standard of care for the systemic treatment of patients with advanced gallbladder cancer is the chemotherapy combination of gemcitabine and cisplatin, which is suboptimal, yielding only modest benefit, said Richard Kim, MD, Section Chief, Gastrointestinal Medical Oncology, Moffitt Cancer Center, Tampa, FL, at the 2020 Cholangiocarcinoma Summit.
FGFR Inhibition in Cholangiocarcinoma: Overcoming Acquired Resistance
December 2020, Vol 1, No 3
In April 2020, the FDA granted accelerated approval to pemigatinib (Pemazyre), the first targeted therapy for cholangiocarcinoma (CCA). The FGFR inhibitor was approved for adults with CCA and FGFR2 fusion.
Emerging Molecular Targets in Cholangiocarcinoma: IDH1, HER2, BRAF, and Beyond
December 2020, Vol 1, No 3
Targeted therapy has improved survival for patients with cancer across a broad spectrum of disease sites, but until recently, progress has been slow in the treatment of patients with cholangiocarcinoma (CCA).
Improving Chemotherapy Combinations and Novel Treatments Are Needed in Advanced Biliary Tract Cancer
December 2020, Vol 1, No 3
New cytotoxic chemotherapy regimens and novel combinations are being evaluated in clinical trials in an effort to improve the outcomes of chemotherapy in the adjuvant and palliative settings in patients with biliary tract cancer, including cholangiocarcinoma (CCA), said Angela Lamarca, MD, PhD, MSc, Consultant, Medical Oncology, the Christie NHS Foundation Trust, Manchester, United Kingdom. She discussed the future of cytotoxic chemotherapy for this patient population at the 2020 Cholangiocarcinoma Summit.
Role of Liver Transplant in Patients with Cholangiocarcinoma
December 2020, Vol 1, No 3
Liver transplant is emerging as a treatment option in patients with unresectable hilar or intrahepatic cholangiocarcinoma (CCA), with accumulating data supporting transplant protocols in patients with early-stage CCA.

Subscribe to CCA News

Stay up to date with personalized medicine by subscribing to receive the free CCA News print publication or weekly e‑Newsletter.

I'd like to receive: