A group of 29 expert scientists and clinicians from the world’s leading academic institutions developed potentially transformative project proposals while gathered in Las Vegas for a 2-day meeting called Cure Cholangiocarcinoma Think Tank (C2T2). A white paper summarizing the critical discussion themes and proposed projects from the May 12-14, 2022, meeting outlines next steps for the community. This effort, made possible by generous donations to the Cholangiocarcinoma Foundation, was the first of its kind in the cholangiocarcinoma (CCA) field.
Experts outside of CCA were instrumental in helping identify the topics for the C2T2 meeting. The primary themes—precision prevention and early detection, and precision therapeutics—reflected areas of urgency and opportunity for improving outcomes for people impacted by CCA. Scientific discovery has advanced to the point where addressing knowledge gaps in these areas could have a measurable and profound impact on the trajectory of CCA.
“The inaugural Cure Cholangiocarcinoma Think Tank (C2T2) meeting was a tremendous success and lays the foundation for innovative approaches to collaborating across institutional boundaries with a total focus on helping patients with the most pressing unmet clinical needs,” said Mitesh J. Borad, MD, Chair of the International Cholangiocarcinoma Research Network. “The outputs from this year’s meeting will galvanize the already robust efforts in precision medicine to facilitate earlier diagnosis and more tailored treatments for individual patients. We look forward to releasing a white paper that summarizes the meeting proceedings and sharing it with the CCA research community,” he added.
CCA is a type of cancer that forms in the slender tubes called bile ducts that carry the digestive fluid bile. Bile ducts connect the liver to the gallbladder and the small intestine. An estimated 10,000 Americans are diagnosed yearly with the lethal cancer with a poor prognosis and high mortality rate. A recent study by Rahib and colleagues (JAMA Network Open) forecasted that by 2040, liver and bile duct cancer will be the third deadliest cancer in the United States. This startling statistic invokes immediate action to achieve a cure for CCA, and C2T2 is dedicated to achieving this by addressing all challenges.
Key takeaways from the C2T2 meeting focused on overcoming challenges such as the low incidence of CCA, which results in difficulty in completing early detection studies and developing a screening test that can be sufficiently specific. Most early detection tests are refined through a case-controlled study. To address this, the meeting participants agreed to identify a very high-risk group of patients, such as young patients with primary sclerosing cholangitis or patients with a genetic predisposition to CCA, or seek to include CCA as part of a multi-cancer test. Furthermore, leaders in the CCA field should work to ensure that CCA is included within the studies for a multi-cancer test and that these tests are prespecified to predict CCA. Preliminary data are needed to show a meaningful result in a rare cancer such as CCA. Identifying a genetic predisposition or other risk factor groups, including real-life controls, and conducting prospective studies to define the performance of the test are also needed.
Additional points included the need to understand tumor biology better and the need to more effectively guide where researchers look for treatment targets based on what is found in a tumor at the genomic level. Using a multistep approach to evaluate tissue and relevant proximal body fluids such as bile or stool was also recognized. Challenges with bile include the need for an invasive procedure. It is hard to work with at the protein level, and stents can confound. Evaluating whether it might be possible to use the colonoscopy to gather bile was contemplated.
“The C2T2 meeting was much anticipated. It was enormously beneficial to have invited specialists from outside our research community to share their best practices, innovations, and new methodologies,” said CCF CEO and Founder Stacie Lindsey. “The group labored together for 2 days to define concrete projects to move the science forward in meaningful ways. We are excited to see these projects advancing rapidly.”
C2T2 was not a typical academic gathering designed around a series of didactic talks, data sharing, and discussions around broad themes. The multidisciplinary group of experts who came together for the meeting remains committed to developing research proposals designed to answer concrete questions and advance near-term effects. Because of the leadership of the Foundation and key donors, the projects emerging from the meeting will have seed funding support and be the subject of additional, focused fundraising efforts.
“For the first time in rare cancer like CCA, we put together a summit to gather experts from inside and outside the field to work together productively to solve major challenges in the care of these patients. One major focus is how we can predict which patients with primary sclerosing cholangitis, a high-risk disease of CCA, will develop CCA,” said Nilo Azad, MD, an associate professor of oncology and a member of the Gastrointestinal Oncology Program within the Sidney Kimmel Comprehensive Cancer Center in Baltimore, MD.
She continued, “We have been able to leverage generous philanthropic support along with tools developed by the CCA Foundation and major research institutions around the world to come together to profile biospecimens in the hopes of really understanding which patients most likely develop this cancer. This could result in meaningful improvement in the survival of these patients by catching things early.”
C2T2 is planning subsequent meetings with outcomes anticipated to benefit current and future CCA patients and caregivers and their loved ones. Developments to improve methods of early detection, advance treatment options, and improve the quality of life and overall survival of patients are a C2T2 priority.
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