Highlighting New Research on Next- Generation Agents in the Treatment of Cholangiocarcinoma and Other Biliary Cancers

June/July 2022, Vol 3, 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

Dear readers,

The publication of the TOPAZ-1 trial in the June 1, 2022 issue of NEJM Evidence marks a milestone in the treatment of biliary tract cancers. Although many questions remain, including those pertaining to predictive biomarkers, heterogeneity of cancer types, underlying risk factors and their correlation with clinical outcome, this article clearly heralds the role of immunotherapy in the management of these cancers.

In this issue of CCA News, we are highlighting 2 important conferences: the Cholangiocarcinoma Foundation (CCF) annual meeting and the International Hepato-Pancreato-Biliary Association (IHPBA) World Congress. In the CCF annual meeting, Dr Rachna Shroff from the University of Arizona highlighted the ongoing development of targeted therapeutics. These include novel IDH1 and FGFR inhibitors, bispecific antibodies, and antibody–drug conjugates directed against HER2/neu as well as NRG1, MDM2, and DNA repair-directed therapeutics. These represent the next generation of agents in these cancers, again highlighting the fact that biliary cancers, particularly cholangiocarcinomas (CCAs), are “target-enriched.” Molecular testing is critical in the management of these cancers and, in an insightful review, Drs May Cho and Sepideh Gholami from the University of California, Irvine highlight the problems associated with biomarker testing in CCA and propose practical solutions, including standardized guidelines, molecular tumor boards, and establishment of a leadership structure, to capture molecular data on most/all biliary tract cancer patients.

The IHPBA meeting presented several studies that highlighted multidisciplinary management. Dr Flavio Rocha, from Oregon Health and Sciences University, highlighted early-stage gallbladder cancer and noted that laparoscopic resection may substitute for open procedures for select cases with early-stage disease. Other highlights included the increasing use of neoadjuvant chemotherapy for intrahepatic CCA in the United States with corresponding lower-margin positivity rates and the need for neoadjuvant chemoradiation prior to liver transplantation for perihilar CCA.

Finally, Dr Reham Abdel-Wahab from the CCF reported the results of a research survey conducted at the CCF annual meeting. Among this group of informed participants, 77% received molecular profiling but only 27% enrolled in clinical trials. Although these figures are somewhat disappointing, they compare favorably with some of the more common solid tumors! These and other important investigations are included in this issue.


Milind M. Javle, MD

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