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.
Imane El Dika, MD, of Memorial Sloan Kettering Cancer Center, discussed the targeting of IDH1, HER2, and BRAF mutations, among other genetic signatures, as potential targeted treatment options for patients with CCA. In April 2020, the FDA granted accelerated approval to pemigatinib (Pemazyre), the first targeted therapy for CCA. The FGFR inhibitor was approved for the treatment of CCA and FGFR2 fusion in adults. However, acquired resistance has been a limiting factor with all FGFR inhibitors.
Lipika Goyal, MD, MPhil, of Massachusetts General Hospital, discussed the mechanisms of resistance to FGFR inhibition and strategies for improving the current approaches to treatment. Ghassan Abou-Alfa, MD, MBA, Attending Physician, Memorial Sloan Kettering Cancer Center, discussed the growing list of molecular targets for CCA and highlighted the clinical relevance of tumor mutation profiling in the management of this disease.
Angela Lamarca, MD, PhD, MSc, of the Christie NHS Foundation Trust in the United Kingdom, focused on the continued role of cytotoxic chemotherapy and pointed out that more is not always better; for example, FOLFIRINOX has not been proved to be more efficacious than gemcitabine and cisplatin.
John Fung, MD, PhD, Professor of Surgery and Director, Transplantation Institute, University of Chicago, addressed the emerging role for transplantation in hilar and intrahepatic CCA. Dr Fung emphasized the importance of having a multidisciplinary team to apply the newest tools, including imaging, molecular profiling, and bridging to prepare patients for transplant and to manage them appropriately afterward, and stressed the potential of targeted therapies to address disease recurrence.
This summit was novel in incorporating a special session on gallbladder cancer. Richard Kim, MD, of Moffitt Cancer Institute, indicated that there are very few clinical trials that focus exclusively on gallbladder cancer; most studies tend to group gallbladder cancer and CCA together, thereby limiting a comprehensive understanding of gallbladder cancer. Moving forward, Dr Kim said, targeting the molecular alteration in gallbladder cancer will be the key to managing patients appropriately. Fortunately, this pattern may be changing.
Shubham Pant, MD, Associate Professor at M.D. Anderson Cancer Center, presented the results of the phase 1 clinical trial of zanidatamab (a bispecific antibody) in an innovative virtual poster discussion session. It was gratifying to learn that soon after the CCA Summit, on November 30, 2020, the FDA granted zanidatamab a breakthrough therapy designation for patients with HER2-amplified biliary tract cancer.
In the same session, Kimberly McGregor, MD, Director of Clinical Development at Foundation Medicine, noted a changing pattern of comprehensive genomic profiling in CCA, which coincides with a dramatic increase in clinical trials for therapy trials.
Filip Janku, MD, Associate Professor at M.D. Anderson Cancer Center, discussed the next generation of targeted agents, particularly IDH1 and IDH2 inhibitors, several of which are already in phase 2 clinical trials for CCA.
Finally, in an eloquent speech, Melinda Bachini, Director of Advocacy, Cholangiocarcinoma Foundation, described patients’ journey through this disease, pointing out that, “Patients and providers are partners in hope.”
We are extremely grateful to the presenters, participants, and organizers of the 2020 CCA Summit for providing an important avenue for discussing the recent developments in this disease.
If you have any questions, suggestions, or concerns please address them to me at This email address is being protected from spambots. You need JavaScript enabled to view it.
Sincerely,
Milind Javle, MD Professor, Department of Gastrointestinal Medical Oncology Division of Cancer Medicine The University of Texas MD Anderson Cancer Center Houston, TX
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