Cholangiocarcinoma

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).
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.
Patient-derived tumor organoids may have clinical application to predict drug responses in a personalized treatment setting; they have shown concordance with actionable genomic anchors and retrospective treatment outcomes.
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.
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.
Targeted therapy has improved survival for patients with cancer across a broad spectrum of disease sites, but until recently, progress has been slow in applying the use of targeted therapies in the treatment of patients with cholangiocarcinoma (CCA).
Of the approximately 18,000 individuals who are diagnosed annually in the United States with biliary cancer, an estimated 8000 are diagnosed with cholangiocarcinoma (CCA), and the 5-year survival rate is less than 20%. CCA is often diagnosed at advanced stages when treatment is only minimally effective.
Barcelona, Spain—Alterations in the fibroblast growth factor receptor (FGFR2)2 gene have been identified as driver mutations in cholangiocarcinoma (CCA). Durable objective responses were observed in >33% of patients with locally advanced or metastatic CCA and FGFR2 rearrangements or fusions who received treatment with pemigatinib, a selective oral inhibitor of FGFR1, FGFR2, and FGFR3. Data from the single-arm, open-label phase 2 clinical trial FIGHT-202, which was presented at the ESMO Congress 2019, revealed that investigational pemigatinib induced a response in 35.5% of the 107 patients with FGFR2 fusions or rearrangements (cohort A), with a median duration of response of 7.5 months.
Barcelona, Spain—Ivosidenib (Tibsovo), an oral therapy that targets isocitrate dehydrogenase-1 (IDH1) mutation, significantly improved pro­gression-free survival (PFS) in patients with advanced cholangiocarcinoma (CCA) and an IDH1 mutation, in a phase 3 clinical trial reported lead investigator Ghassan K. Abou-­Alfa, MD, Medical Oncologist, Gastrointestinal Oncology Service, Memorial Sloan Kettering Cancer Center, New York City, at the ESMO Congress 2019.
Diagnosis of cholangiocarcinoma (CCA) is often identified at a late stage. Analyzing the tumor­specific mutation profile of a patient with CCA can improve the diagnosis and treatment for the individual patient.

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