The Lynx Group
Cholangiocarcinoma News

The International Cholangiocarcinoma Research Network: Educational and Research Achievements

December 2021, Vol 2, No 4
Reham Abdel-Wahab, MD, PhD
Director of Research
Chief Scientific Officer
Cholangiocarcinoma Foundation

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.

CCA Natural History

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.

ICRN/ENS-CCA Translational Webinars

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.

Mentorship Programs

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.

Related Items

Think Tank of Multidisciplinary Experts Develop Proposals to Improve Outcomes in Patients with CCA
December 2022, Vol 3, No 4
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).
International Cholangiocarcinoma Patient Registry Accelerates and Supports CCA Research
September 2022, Vol 3, No 3
The Cholangiocarcinoma Foundation hosts a patient registry, the International Cholangiocarcinoma Patient Registry (ICPR), that is a global database comprising patient medical histories and treatments for cholangiocarcinoma (CCA).
Clinical Trial Enrollment Challenges for Patients with Cholangiocarcinoma
By Reham Abdel-Wahab, MD, PhD; Samantha Pear
June/July 2022, Vol 3, No 2
There is a desperate need for patients with CCA to gain access to clinical trials to help improve survival.
The Cholangiocarcinoma Foundation’s Patient Advocacy Initiatives
March 2022, Vol 3, No 1
At the 2022 Cholangiocarcinoma Foundation (CCF) Annual Conference on February 22-25, Melinda Bachini, Director of Advocacy of CCF, provided an update about the foundation’s advocacy and patient support initiatives. The goal of the CCF’s advocacy group is to provide educational and emotional support for the cholangiocarcinoma (CCA) community of patients and caregivers, and to empower them with the knowledge and guidance needed to self-advocate.
New ICD-11 Codes for Cholangiocarcinoma
December 2021, Vol 2, No 4
The Cholangiocarcinoma Foundation (CCF) is encouraging healthcare professionals to support the adoption of the new International Classification of Diseases, 11th Revision (ICD-11), and its role in creating more robust epidemiological data for cholangio­carcinoma (CCA).
Navigating Compassionate Use Options for Your Patients with Cholangiocarcinoma
By Melinda Bachini
September/October 2021, Vol 2, No 3
I believe that the most powerful and positive force in medicine is that of a self-advocating patient. At the Cholangiocarcinoma Foundation, we strive to connect, educate, and empower patients with cholangiocarcinoma (CCA) to partner with their care team in navigating the course of their treatment. However, we understand that the treatment options are extremely limited for many patients with CCA.
A Watershed Moment for the Cholangiocarcinoma Community
By Stacie C. Lindsey
June/July 2021, Vol 2, No 2
In an article published last year in CCA News, I shared the Cholangiocarcinoma Foundation (CCF)’s philosophy on taking risks and leveraging collaboration over competition. The CCF was nearing its 15th anniversary, and the FDA had recently approved pemigatinib (Pemazyre), the first targeted therapy approved for cholangiocarcinoma (CCA).
The Urgent Need to Raise Awareness to Cholangiocarcinoma and Fund Research
By Teresa Delcorso-Ellmann
March 2021, Vol 2, No 1
I first learned of cholangiocarcinoma (CCA) in 2015, when my husband Steve was diagnosed with stage IV inoperable intrahepatic CCA and was given a prognosis of less than a year to live. He defied the prognosis and survived for 40 months, until his death in March 2019. When he was diagnosed, neither one of us had ever heard the word “cholangiocarcinoma” and did not know it was possible to have cancer in the bile duct. We both knew that a diagnosis of other gastrointestinal (GI) cancers, such as pancreatic or colon cancer, could be catastrophic and were stunned to realize that the treatment options and outcomes for patients with CCA were the most dismal of all GI cancers.
Where There Is Hope
By Laura R. Hnat
December 2020, Vol 1, No 3
“You have cancer.” These are the 3 little words that everyone dreads hearing and that doctors wish they never had to utter. Yet according to the American Cancer Society, an estimated 1.8 million people will be diagnosed with cancer in the United States this year, and more than 600,000 people will die of it.
Leading the Way to a Paradigm Shift in Cholangiocarcinoma: The International Cholangiocarcinoma Research Network
By Nilo Azad, MD; Mitesh J. Borad, MD
October 2020, Vol 1, No 2
In the past decade, there has been a sea change in awareness about cholangiocarcinoma (CCA). However, the standard of care has remained unchanged for more than 10 years. This year, the FDA approved a new targeted therapy, pemigatinib, which became a treatment option for approximately 10% to 15% of patients with CCA. The process of drug discovery, as well as changes in the standard of care, are becoming increasingly reliant on collaborative approaches that leverage the expertise and resources of a wide range of partners to strengthen the tools and knowledge that advance the research objectives of all stakeholders.

Subscribe Today!

To sign up for our newsletter or print publications, please enter your contact information below.

I'd like to receive: