In 2015, the Cholangiocarcinoma Foundation (CCF) launched the International Cholangiocarcinoma Research Network (ICRN). This network is a global collaborative of clinicians, researchers, and scientists from leading academic medical centers and research institutions working together to improve the prevention, early detection, treatment, and prognosis of cholangiocarcinoma (CCA).
This multidisciplinary global group works in close collaboration with CCF, which allows for its unique patient-centric research focus. The ICRN currently has more than 200 members, including 120 from the United States, representing 97 institutions and 19 countries. This collaborative culture fosters interaction between industry, academia, regulatory, and patient-centered organizations to marry science with patient needs.
ICRN members value collaboration; they have been previously awarded a 4-year grant from the US Department of Defense to study the genomic or immunologic heterogeneity of intrahepatic CCA. They are also conducting 4 multicenter clinical trials and have a large data biorepository of more than 1000 patient cases. All the members are dedicated to working together through CCF for the benefit of patients with CCA.
Over the past 15 years, CCF has convened and led a community of stakeholders, including patients, research networks, clinicians, researchers, scientists, and pharmaceutical and technology companies. CCF’s stakeholder community includes the ICRN, an Industry Council, a Patient Advocacy Team, Invitae (formerly Ciitizen, a patient-centric health information technology company), Komodo Health (a health information technology company), the European Network for the Study of Cholangiocarcinoma (ENS-CCA), and others.
CCF’s ability to engage these stakeholders, leverage and integrate their collective expertise, and align them around joint research and educational priorities has rapidly accelerated advances in the understanding of CCA over the past 5 years.
The gaps in knowledge related to CCA are fueled by its being a rare disease, with a small patient population and high heterogeneity. As in most rare cancers, only limited data are available to support longitudinal natural history studies, which are integral to understanding the course of a disease in the absence of investigational intervention, and are critical for informing drug development, from discovery to clinical trial design.
Currently, only 1 longitudinal database exists, a robust, 4000-patient database to support longitudinal natural history studies of patients with CCA in Europe, which was created and is maintained by the ENS-CCA. No such data set exists for the United States. Existing data sets of patients in the United States are small, disparate, and kept separately by competing, high-volume cancer centers, making them inaccessible to researchers who wish to conduct natural history studies by using aggregated data from US patients. Further complicating the issue of CCA data of patients in the United States is an extremely fragmented health information technology infrastructure, with multiple electronic health record (EHR) systems used by different providers, making integrating data sets very difficult.
To address this unmet need, in 2019, CCF began working with Invitae, a health technology company that enables the secure collection, organization, and sharing of patient EHRs with clinicians to improve patient care and support research, using the patients leveraging their own data. Together, CCF and Invitae designed and piloted an infrastructure for the CCA Natural History Data Set, a centralized database derived from patient data that includes detailed patient demographics, disease risk factors, and detailed clinical information, such as disease staging, laboratory tests, genomics, and outcomes from various treatment modalities.
Through this pilot, CCF and Invitae designed a patient-friendly enrollment interface for patients. They also developed AI (artificial intelligence)-powered workflows for pulling, integrating, de-identifying, structuring, normalizing, validating, and then sharing patient data with researchers in a research-ready format.
CCF recruited and enrolled 800 patients with CCA to the Natural History Study as part of the initial pilot. This is a patient-driven project, and these 800 patients are its cornerstone. Thus, the patient advisory team reaches out to many patients to inform them about the value of the project, and how it will help them access their medical records for research.
However, CCF still needs to make additional efforts to further outreach and increase our global collaboration. We are currently working with clinicians in high-volume cancer centers worldwide to establish an online-based registry database housed at CCF and integrated with the current Natural History Data Set. CCF will make this database accessible for all stakeholders, including clinical investigators, scientists, pharmaceutical companies, and policymakers, to understand disease biology, develop preventive and screening guidelines, design clinical trials, and develop and approve novel therapeutic interventions.
Since August 2020, the ICRN and the ENS-CCA/European Cholangiocarcinoma Network (EURO-CHOLANGIO-NET) European Cooperation in Science and Technology (COST) Action have joined forces to launch a monthly clinical and transitional seminar series. This partnership aims to disseminate emerging basic, translational, and clinical information to interested researchers around the globe to foster new ideas and deeper collaborations among our membership.
We have discussed the genomic and epigenomic landscape, as well as opportunities for repurposing drugs and novel therapeutic approaches from bench to bedside. We have also focused on various topics related to surgical, local, and systemic therapy approaches.
So far, we have successfully organized 15 webinars presented by experts from high-volume cancer institutes in the United States, United Kingdom, Belgium, and Ireland. On average, more than 150 clinicians and researchers have attended and viewed each webinar. Currently, we are focusing on reaching out to more national and international oncology residents, fellows, and postdoctoral fellows to expand our community and engage young researchers.
In September 2021, ICRN and ENS-CCA/EURO-CHOLANGIO-NET COST Action partnered and announced 2 mentorship programs: a 6-month Career Mentoring Program and a 12-month Scientific Mentoring Program. These programs are designed to support and advise junior researchers in their career choices and promote scientific growth, by matching mentees and mentors within the ICRN and the ENS-CCA networks.
Although mentorship is a well-developed practice in most high-volume cancer institutions, tremendous clinical workloads and lack of access to research resources and collaboration opportunities in community hospitals and low- and middle-income countries underscore the need to develop these mentorship programs. Our joint mentorship programs offer junior researchers the opportunity to get support from international, external, and independent highly experienced physician-scientist mentors who share our interest in CCA.
These new programs help to mentor young researchers, expand their communication, gain knowledge from their mentors’ experience, enhance their skills, increase their confidence, learn how to build a strategy to improve their academic career, reach their goals, and discover the best mentor and mentee relationship practice.
Moreover, the Scientific Mentoring Program is designed to allow junior researchers to work closely with experts in various research areas, including cancer research, cell biology, genomics, AI, and other high-yield research areas in the research world. Candidates who will be accepted in either program will also attend a series of panel discussion format lectures, which will be an open discussion between all mentors and mentees to discuss mentees’ needs and their pathway from fellows to faculty positions, how they could get seed funding for their research, publish articles, and many other topics of interest for their careers. These programs support CCF’s goals of expanding its stakeholder community and supporting the new generation of CCA experts.
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