Cholangiocarcinoma Research Continues to Thrive

December 2021, Vol 2, No 4
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

Dear Readers,

The Third CCA Summit was held on October 21-22, 2021 in New Orleans. For the majority of the cholangiocarcinoma (CCA) community, this was their first “live” meeting since the pandemic. The summit was attended live or virtually by a total of 165 participants, and was co-chaired by Ghassan K. Abou-Alfa, MD, MBA, and Rachna T. Shroff, MD, MS, and provided a valuable opportunity for clinicians and researchers in the field. In this last issue of 2021, we are highlighting key research presented at the summit, as well as in the recent peer-reviewed literature.

Jesús M. Bañales, PhD, discussed the role of epigenetics, posttranslational modifications, and metabolism in CCA and highlighted several targets for therapy, such as the epigenetic axis, the neddylation pathway, and metabolic targeting of fatty acid oxidation with specific inhibitors, which may indeed represent the next generation of inhibitors in CCA.

Nabeel El-Bardeesy, PhD, presented interesting findings from his laboratory regarding immunogenicity of IDH1-altered CCA, rendering these tumors susceptible to checkpoint inhibition.

Lewis Roberts, MBChB, PhD, described the fascinating association between genomic variations and environmental risk factors in a global study led by his team.

Clinical trials have thus far focused on systemic delivery of chemotherapy, whereas liver-directed approaches may be critical for liver-limited CCA, suggested Juan W. Valle, MBChB, MSc, and Skye C. Mayo, MD, MPH. The assessment of radiological response may be more sophisticated today, with enhanced radiomic technologies that may also be invaluable for prognostication.

James Costello, MD, PhD, and Laurent Dercle, MD, PhD, presented novel biomarker imaging data, which may be transformative in the clinic.

Richard Kim, MD, and Do-Youn Oh, MD, PhD, discussed the expanding role of immunotherapy with checkpoint inhibition in CCA, emphasizing that the CCA community eagerly awaits the results of the TOPAZ-1, S1815, and KEYNOTE-966 phase 3 clinical trials, which may change the treatment paradigm of this disease.

It remains to be seen if immunotherapy can benefit the majority of patients with CCA, and efforts to enhance efficacy by combining immunotherapeutic and targeted agents will be critical in this regard, according to Mark Yarchoan, MD.

Multimodality approaches that include surgery, perioperative therapy, and transplantation for liver-limited intrahepatic CCA may be curative in selected patients, suggested Alice C. Wang, MD, MSc, and Shimul A. Shah, MD, MHCM.

Several Keynote presentations and panels were also featured at the summit, including a global Keynote Panel that provided perspectives on CCA from the United States, the European Union, Latin America, and Asia. Dr Bañales highlighted the importance of implementing the new ICD-11 codes (which go into effect on January 1, 2022) to improve our understanding of CCA subtypes across the globe.

Stacie Lindsey, CEO, Cholangiocarcinoma Foundation (CCF), delivered a Keynote Address that highlighted the need for collaboration and the need to focus on the patient in drug development.

And a Keynote Roundtable discussion reviewed the drug pipeline for CCA, highlighting zanidatamab, a bispecific HER2 antibody, which was granted a breakthrough therapy designation by the FDA in November 2020 for patients with HER2-amplified biliary tract cancer.

Also in this issue, Reham Abdelwahab, Director of Research & Chief Scientific Officer of CCF reviews research initiatives sponsored by the foundation, detailing the specific ICD-11 codes, by indication, for immediate reference.

Finally, in this issue, we are highlighting 3 recent articles from the peer-reviewed literature. Together with Mitesh J. Borad, MD, and Nilofer S. Azad, MD, and colleagues, we investigated the use of trastuzumab plus pertuzumab in patients with biliary tract cancer and HER2/neu amplification, which showed a 23% response rate that was often long-lasting in a chemorefractory patient population.

Karthikeyan Murugesan, MS, and colleagues investigated the genomic underpinnings of hepatocellular CCA, pointing out the dimorphic pattern of genomic alterations in this composite disease. And Jianzhen Lin, PhD, and colleagues reviewed the evolution of gallbladder cancer from preneoplastic lesions and the pivotal role of CTNNB1 mutations in the development of this malignancy.

Clearly, CCA research continues to thrive, and we will learn more soon, at the ASCO GI Cancers Symposium in late January 2022!


Milind Javle, MD

Milind M. Javle, MD
Department of Gastrointestinal Medical Oncology
Division of Cancer Medicine
The University of Texas
M.D. Anderson Cancer Center
Houston, TX
NCI Task Force: Hepatobiliary Cancers

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