The Lynx Group
Cholangiocarcinoma News

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.
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“You have cancer.” These are the 3 little words that everyone dreads hearing and that doctors wish they never had to utter. Yet according to the American Cancer Society, an estimated 1.8 million people will be diagnosed with cancer in the United States this year, and more than 600,000 people will die of it.
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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.
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In this last issue for the year, we are highlighting the proceedings of the Second Annual Cholangiocarcinoma (CCA) Summit, which was conducted on October 22-23, 2020. This summit attracted a wide, multidisciplinary audience and was very interactive, despite being a virtual event. An important focus for this meeting was the emergence of molecular targets in CCA and early successes seen with precision medicine in this disease. Many of the key presentations from the summit are included in the current issue and are highlighted below.
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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.
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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).
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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.
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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.
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Despite the onslaught of the COVID-19 pandemic, researchers are hard at work to develop innovative therapies that will make a difference in the lives of patients with cancer.
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At the 2020 Cholangiocarcinoma Summit, Nabeel El-Bardeesy, PhD, Associate Professor, Medicine, Harvard Medical School, and Associate Geneticist, Center for Cancer Research, Massachusetts General Hospital Cancer Center, Boston, MA, moderated a session titled “Molecular Biomarkers in CCA: Focus on Current and Emerging Technologies.” The session focused on how best to integrate the array of genomic testing platforms into clinical practice. The session included 2 presenters who discussed this topic.
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