Posted February 1, 2023

Dr. John Hooper, The University of Queensland
Dr. Xinchun Zhou, University of Mississippi Medical Center
Mr. Steven West, RCRP Consumer Peer Reviewer

Cholangiocarcinoma (CCA), also known as bile duct cancer, is a rare aggressive cancer arising in the bile duct. The bile duct serves as a highway for the passage of bile fluid from the liver and gallbladder to the small intestine, where it helps digest food. Tumors can develop in any part of the bile duct and are classified based on their location. Although CCA is a rare cancer, affecting 1 person per 100,000¹ per year in the U.S., CCA incidence is increasing.² CCA often goes undetected until at an advanced stage, at which point it is difficult to treat surgically and is resistant to chemotherapy, contributing to its dismal 5-year survival rate of only 10%.³ The Rare Cancer Research Program (RCRP) is joining in the recognition of World Cholangiocarcinoma Day, February 23, 2023. The RCRP has ongoing support for two CCA-related projects, both funded in fiscal year 2021(FY21), focused on improving our understanding of the biology and etiology of CCA and investigating new therapies.

Dr. John Hooper at The University of Queensland (Australia) received support through a RCRP Idea Discovery Award to investigate the role of a cellular receptor, CDCP1, in CCA pathogenesis. Preliminary data has demonstrated that CDCP1 could be a key contributor to CCA. With the support of this RCRP award, the research team will further define the role of CDCP1 in CCA development and progression in an animal model. They also aim to determine if CDCP1 can be utilized as a target for CCA detection by a commonly used imaging platform, positron emission tomography-computed tomography, better known as PET-CT imaging. Finally, the team will look at the efficacy of a CDCP1-targeted therapy to reduce tumor burden and improve CCA survival in an animal study.

Dr. Xinchun Zhou at the University of Mississippi Medical Center is supported by a RCRP Concept Award to investigate the role of long non-coding RNAs (lncRNA) in CCA. Despite their name, non-coding RNAs have been found to encode small proteins, called micropeptides, that can regulate biological processes and can play a role in diseases such as cancer.⁴ Dr. Zhou hypothesizes that lncRNA can regulate the Notch signaling pathway, which is known to play a role in the pathogenesis and progression of CCA. This effort will lead to improved understanding of CCA biology and opportunities for early-stage diagnosis and treatment.

The RCRP would also like to share the story of Mr. Steven West, a professor in the School of Business at Oklahoma State University, a husband, a father, and a CCA survivor. Mr. West’s doctors first discovered a mass on his liver during a routine health check in 2013 that was diagnosed as a hemangioma, a noncancerous tumor made up of blood vessels. It wasn’t until a year later when receiving treatment for a kidney stone that the urologist told him “forget about the kidney stone… worry about this thing on your liver.” That began an arduous seven-month odyssey of referrals, refusals for biopsies, and lots of “watch-and-wait” scans. Mr. West refused to simply “watch and wait.” Instead, he collected all his medical records, completed detailed reviews of his scans, and insisted upon a biopsy. Biopsy results confirmed that he had CCA. In June 2015, he underwent surgery to remove 75% of his liver and resect a softball size tumor. Since then, Mr. West has undergone additional surgeries to remove cancerous growths and continues to be closely monitored for disease recurrence. Thanks to his refusal to “watch and wait,” Mr. West is a CCA survivor who enjoys spending time with his family and attending car shows.

After being nominated by the Global Liver Institute, which focuses on empowering patient advocates to collaborate with policymakers, clinicians, and industry to drive the liver health field forward, Mr. West has participated as a consumer reviewer for RCRP peer review since FY20. He is also actively involved in the Cholangiocarcinoma Foundation (CCF) as a research advocate and a mentor. In his role as a mentor, Mr. West provides support, empathy, and understanding to fellow CCA fighters, survivors, and caregivers. Mr. West also shares his patient perspective as a research advocate for the CCF to help advance the science and better the lives of CCA patients, similar to his role as a consumer reviewer for the RCRP. The RCRP is committed to promoting partnerships with the patient community through their inclusion in our review processes, as well as funding patient-centric research.

References:
¹Cholangiocarcinoma – Affected Populations

²Javle M, Lee S, Azad NS, et al. 2022. Temporal changes in cholangiocarcinoma incidence and mortality in the United States from 2001 to 2017. Oncologist 27(10):874-883. doi: 10.1093/oncolo/oyac150. PMID: 35972334; PMCID: PMC9526482.

³Bile duct cancer stages

⁴Palazzo AF and Lee ES. 2015. Non-coding RNA: What is functional and what is junk? Frontiers in Genetics 6:2. doi: 10.3389/fgene.2015.00002. PMID: 25674102; PMCID: PMC4306305.

Links:
Public and Technical Abstracts: A Novel Mediator of Cholangiocarcinoma That Has Potential as a Diagnostic and Treatment Target
Public and Technical Abstracts: Role of lncRNA-Encoded Micropeptides in Cholangiocarcinoma

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