Many informative and interesting posters covering clinical trials were presented at the CCA Summit. The session featured 3 posters that examined the chemotherapy/immunotherapy landscape, including “Gemcitabine plus cisplatin versus non-gemcitabine and cisplatin regimens as neoadjuvant treatment for cholangiocarcinoma patients prior to liver transplantation: an institution experience” (Maen Abdelrahim, MD, PhD, PharmB, Houston Methodist Cancer Center and Institute for Academic Medicine) and “An open-label phase Ib/II trial of induction systemic gemcitabine, cisplatin, and durvalumab (GCD) followed by concurrent hepatic arterial infusion of floxuridine and systemic GCD as first-line for unresectable intrahepatic cholangiocarcinoma (HELIX-2)” (Ranish K. Patel, MD, Oregon Health & Science University), which anticipates opening for enrollment in winter 2023. The poster titled “Characterization of long-term survivors in the TOPAZ-1 study of durvalumab or placebo plus gemcitabine and cisplatin in advanced biliary tract cancer” (Stephen Valerio, MS, AstraZeneca) showed that participants treated with durvalumab plus gemcitabine/cisplatin (GemCis) were more likely to experience long-term survival compared with placebo plus GemCis.
Posters detailing advances in targeted therapy in patients with cholangiocarcinoma (CCA) included “Characterizing outcomes of HER-2 amplified biliary tract cancer” (Sunyoung Lee, MD, PhD, University of Texas MD Anderson Cancer Center) and “FOENIX-CCA4: a phase II study of futibatinib 20 mg and 16 mg in patients with advanced cholangiocarcinoma (CCA) and fibroblast growth factor receptor 2 (FGFR2) fusions/rearrangements” (Teresa Macarulla, MD, PhD, Vall d’Hebron University Hospital); this trial enrollment is currently ongoing in the United States, Europe, and Asia-Pacific. Another poster included “KRAS Mutations in Biliary Tract Cancers” (Gordon Taylor Moffat, MD, Princess Margaret Cancer Centre), which showed that KRAS mutations are common in biliary tract cancer (BTC), and KRAS-mutated tumors trend toward an inflamed tumor-immune microenvironment. The poster titled “CRISPR screening identifies synergy between BET and mTOR inhibitors in cholangiocarcinoma through impaired serine glycine one carbon metabolism” (Yan Zhu [PhD candidate], University of Texas MD Anderson Cancer Center) presented the finding that an unbiased genome-wide CRISPR screen identified a synergistic combination effect between BETp and an impairment of the mTOR pathway, which led to proof of principle that combined BET and mTOR inhibitors can have efficacy against multiple models of CCA. The study mentioned in the poster titled “Brightline-2: a phase 2a/2b, open-label trial of the MDM2-p53 antagonist brigimadlin (BI 907828) in patients with advanced MDM2-amplified, TP53 wild-type (wt) biliary tract cancer (BTC), pancreatic ductal adenocarcinoma (PDAC), or other selected solid tumors” (James J. Harding, MD, Memorial Sloan Kettering Cancer Center) is currently ongoing, and investigators are evaluating the efficacy and safety of the MDM2-p53 antagonist brigimadlin. In addition, 2 posters, “FIRST-308: phase III study of tinengotinib versus physician’s choice in subjects with FGFR-altered, chemotherapy- and FGFR inhibitor-refractory/relapsed cholangiocarcinoma” (Milind Javle, MD, University of Texas MD Anderson Cancer Center) and “Blocking angiogenesis in biliary tract cancer with CTX-009” (Minori Rosales, MD, PhD, Compass Therapeutics), summarized results from phase 1a, 1b, and phase 2 studies, and the subsequent phase 2/3 studies are active and recruiting.
Two posters were presented on the topic of clinical trials in radiology. The poster titled “Hypofractionated radiotherapy-related lymphopenia is associated with worse survival in unresectable intrahepatic cholangiocarcinoma” (Grace Lee, MD, Massachusetts General Hospital) showed that severe lymphopenia is significantly associated with worse overall survival in unresectable intrahepatic CCA. The presenters for the poster titled “Percutaneous thermal ablation for intrahepatic cholangiocarcinoma: efficacy and factors associated with local tumor progression” (Jessica Albuquerque, MD, University of Texas MD Anderson Cancer Center) concluded that percutaneous thermal ablation provides effective and safe intermediate-term local control for intrahepatic CCA patients.
Other posters of interest include “Anticipating cholangiocarcinoma incidence trends: insights from a projection study” (Kanchi Patell, MD, University Hospitals/Case Western Reserve University), in which researchers anticipate increasing incidences of intrahepatic and extrahepatic cancer, highlighting the urgency of identifying at-risk cohorts for targeted screening interventions. Another poster, “Defining ‘high-risk resectable’ intrahepatic cholangiocarcinoma – a survey of the hepatobiliary surgical community” (Tin-Yun Tang, MD, University of Texas MD Anderson Cancer Center), presented the finding that among hepatobiliary surgeons, there was robust support for using single-risk criteria to define high-risk resectable intrahepatic CCA. Another poster titled “Clinical utility of circulating tumor DNA (ctDNA) in patients with early/resected biliary tract cancer (BTC)” (James Yu, MD, H. Lee Moffitt Cancer Center and Research Institute) was a real-world study that found that ctDNA-based minimal residual disease detection after curative-intent therapy can predict disease relapse prior to conventional imaging in BTC. The poster titled “USP1 promotes cholangiocarcinoma progression by deubiquitinating PARP1 to prevent its proteasomal degradation” (Dengyong Zhang, MD, University of Texas MD Anderson Cancer Center) presented the finding that PARP1 is a novel deubiquitination target of USP1 and a potential therapeutic target in CCA. The poster titled “Pan-viral serological profiling in cholangiocarcinoma patients” (Shay Behrens, MD, Oregon Health & Sciences University) showed that VirScan can be used to examine the history of viral exposures in hepatocellular carcinoma and CCA and that viral exposures may differ slightly between disease types.
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