Important Advances in Cholangiocarcinoma Management

October 2020, Vol 1, No 2
Milind M. Javle, MD
Hubert L. and Olive Stringer Professor
GI Medical Oncology
University of Texas MD Anderson
Cancer Center, Houston
Chair, NCI Task Force,
Hepatobiliary Cancers

While the COVID-19 pandemic rages throughout the world, patients, providers, and healthcare advocates are learning to survive in an alternative (virtual) world that is likely to change the face of medicine forever. Cholangiocarcinoma (CCA) is a complex disease that requires multidisciplinary care. Virtual clinic visits and online tumor boards and symposia represent just a few of the countless innovations introduced over the past year.

In this issue of CCA News, we are highlighting topics presented at 2 important virtual conferences held this year—the Cholangiocarcinoma Foundation (CCF) conference held in July 2020, and the European Society for Medical Oncology (ESMO) conference held in September 2020. Below is a quick review of some of the topics discussed in this issue, with a focus on recent advances and ongoing challenges in CCA.

Timely diagnosis and treatment of CCA continue to present challenges. A survey of 700 patients diagnosed with CCA was discussed at the CCF conference, indicating that, on average, it took 22 months from the onset of CCA symptoms to the time the patient was diagnosed with and received treatment for CCA. Furthermore, on average, patients had to visit 2.5 oncologists before the diagnosis of CCA was established. These sobering statistics reflect the real-life scenario facing patients with this type of cancer.

This year, the CCF annual conference was chaired by Jesús M. Bañales, PhD, Group Leader, Liver Diseases Research Group, Biodonostia Institute, San Sebastián, Spain; Rachna T. Shroff, MD, MS, Chief, Section of GI Medical Oncology, University of Arizona Cancer Center, Tucson; and CCF CEO and Founder, Stacie C. Lindsey. Dr Bañales highlighted the importance of international collaboration in CCA, and Dr Shroff discussed the rapid changes in the evolution of therapies for CCA, with new targets such as HER2/neu and epigenetics.

The keynote address at the CCF conference was presented by William W. Li, MD, Chief Executive Officer, President, and Medical Director of the Angiogenesis Foundation, Cambridge, MA. In a particularly poignant introduction, Bruce Koppock, Vice Chair, CCF Board of Directors, introduced Dr Li to the attendees by highlighting how Dr Li had saved his life after his once-successful treatment for his CCA diagnosis was failing. Dr Li’s speech, titled “Dietary Modification of the Tumor Microenvironment: New Frontiers in Cancer Research and Therapy,” took a deep dive into how molecular nutrition and nutritional oncology will play critical roles in cancer medicine in the future.

Recently, ESMO released the first recommendations from a scientific society about the use of multigene next-generation sequencing (NGS). ESMO’s recommendations represent a key step in guiding clinicians toward a more conscious evidence-based use of multigene NGS in clinical practice. ESMO observed that although NGS has been transformative in cancer management, currently, testing is being ordered too late in the disease course. The availability of therapeutic options should guide the use of NGS, which remains expensive at this time.

The role of immunotherapy in CCA continues to be investigational. In an interesting late-breaking abstract presented at ESMO 2020, researchers discussed the results of a multicenter clinical trial of the combination of lenvatinib (Lenvima) and pembrolizumab (Keytruda) for the treatment of 187 patients with 6 different cancers, including CCA. The response rate was 10%, with a median duration of response of 5 months and a 68% disease control rate in a pretreated population of patients with biliary tract cancers. This study highlights the novel basket designs in clinical trials that may have a particular relevance to CCA.

The results of the FOENIX-CCA2 clinical trial of futibatinib used for the treatment of patients with CCA and FGFR2 fusions were presented at ESMO 2020. In this study, the overall response rate was 37.3%, with a median duration of response of 8.3 months. This study represents an important advance in the use of precision medicine in the treatment of patients with CCA.

Finally, in this issue we also feature the first “Guidelines for Management of Urgent Symptoms in Patients with Cholangiocarcinoma and Biliary Stents or Catheters Using the Modified RAND/UCLA Delphi Process,” which was initially published earlier in the year in Cancers and was developed by a team of international experts in association with the CCF.

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

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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