Cholangiocarcinoma

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.

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.

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.

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).

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.

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.

At diagnosis, the majority of patients with intrahepatic cholangiocarcinoma (CCA) present with advanced disease and a poor prognosis. Comprehensive genomic profiling (CGP) of intrahepatic CCA has revealed multiple potential therapeutic targets, including FGFR2, ERBB2 (HER2), and IDH1. Therefore, performing CGP early in the disease course is critical to increasing first-line clinical trial enrollment and access to treatment with FGFR inhibitors.

The first confirmed case in the United States of the 2019 novel coronavirus (COVID-19) was reported on January 21, 2020, and as of November 27, 2020, there were >63 million confirmed cases and >1.5 million deaths globally. Despite the global impact, certain populations have been identified as having a higher risk of developing severe COVID-19 infection, including patients with cancer. Various studies have shown that patients with cancer experience particularly poor outcomes after COVID-19 infection. As a result of the pandemic, care delivery has been disrupted to some degree based on the need for prioritization and limitations on resources. Therefore, healthcare providers and patients have been continually reassessing the balance between the benefits and risks of anticancer interventions in the context of the added risk of COVID-19 infection.

As a result of the COVID-19 pandemic, year 2020 has witnessed unprecedented changes in the practice of medicine. The effect on medical conferences has been equally dramatic, and several major meetings have been canceled or altered. Fortunately, the oncology community adapted quickly!