A Watershed Moment for the Cholangiocarcinoma Community

June/July 2021, Vol 2, No 2
Stacie C. Lindsey
Founder and CEO
Cholangiocarcinoma Foundation

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).

However, with a standard of care still in place from 2010, I wrote: “We know that the trajectory of CCA must change in significant and meaningful ways. The global statistics do not bode well, which means that we cannot keep doing the same thing and expect a different result. We must be exceptionally forward-thinking and sometimes potentially disruptive.”

A year later, a second targeted treatment has been approved by the FDA for patients with CCA, with the anticipation of more to come. The pipeline is filling with treatment options to consider for our patients.

Research tools and partnerships with biotech companies are poised to significantly increase outreach and education. Collaborations are racing toward data sharing and resources that will lift the global field.

We seem to be approaching a watershed moment in the treatment of patients with CCA.

To understand how we reached this point, and what we must do to move forward, it is helpful to look back at where we started and how far we have come.

Humble Beginnings

In 2006, when the CCF was established, the treatment landscape was very different from the way it is today. Patients with CCA were isolated from one another, with no way to interact or share information. There were scarce educational resources available. Clinical research was siloed within institutions, and research funding was almost nonexistent.

The CCF started as an entirely volunteer organization. There were 5 of us, most with full-time jobs. The online discussion board was created to help us connect with others and gather information. It was primarily personal opinion and conjecture being shared, but we were trying, and we were working with very limited resources. Social media was an idea, not the powerful connector it has become for patients and caregivers.

In 2006, 3 medical conferences featured presentations about CCA. The experts were not convening, collaborating, or connecting in any meaningful way. Not in the United States and not globally.

A Strong Presence

Today, the CCF is proudly celebrating its 15th anniversary.

We have opportunities and the ability to make an impact that was beyond our imagination 15 years ago. The Foundation has more than 30 patient advocates and 320 active volunteers. There are 5200 members registered for our discussion board. Our patient registry has well over 2000 participants, and the website receives more than 750,000 visits each year. We have tens of thousands of followers on social media—and we are just getting started.

Patients have an abundance of resources at their fingertips, including a specialist finder, a clinical trial finder, a patient registry, and scores of patient-friendly resources available on the CCF website.

In 2019, CCA science was presented at more than 100 medical conferences across the world. Global scientific collaborations are expanding significantly, which benefits us all and, most important, the patients.

While research funding still lags behind that of many other cancers, the International Cholangiocarcinoma Research Network (ICRN) is accelerating progress. The Foundation has funded more than $2.5 million in research fellowships to early-career scientists at leading research institutions worldwide.

The effect of this seed funding has been tremendous and continues to increase the reach and impact of the research community.


Collaboration is driving progress through programs such as the NIH-funded Cancer Moonshot being led by Dr Ryan Fields at the Alvin J. Siteman Cancer Center at Washington University in St. Louis, MO, and the upcoming inaugural Cholangiocarcinoma Think Tank (C2T2) this fall.

There, the ICRN will convene invited scientific and clinical experts for a “Call to Action” meeting designed to identify and sharpen priorities and to create an action plan for the field to accelerate progress toward early detection and improve pathways for precision medicine.

A Bright Future

Several new targeted therapies are in the pipeline, with many more following on their heels. In addition, industry has embraced the role of the CCF to provide educational materials, raise awareness of CCA, and engage the community through patient-powered research programs.

While things are moving in the right direction, we cannot lose our sense of urgency. The CCF is redoubling our efforts to be a convener, collaborator, and connector in the CCA field.

Patient advocacy organizations are uniquely positioned for this task. A life sciences group recently surveyed 4000 international patients in different disease states.

The study reported that patients place as much trust in patient organizations for information on therapies and clinical trials as they do in their doctors. In addition, 82% of the patients surveyed reported the desire for their doctors to work more closely with patient organizations.

It has been 15 years, and we are more hopeful now than we have ever been. We see the trajectory of this cancer changing, and that change is coming rapidly. Collaboration has been the key element to moving things forward, and we see endless possibilities on the horizon. Join us.

Related Items

CCF Celebrates Major Milestone in Connecting Patients and Caregivers to Mentorship Program
September 2023, Vol 4, No 3
The Cholangiocarcinoma Foundation (CCF) is celebrating the 1000th mentor–mentee match and 6 years of their mentoring program, CholangioConnect. CCF has developed unique resources to help patients and caregivers navigate their healthcare journeys.
10th Annual Cholangiocarcinoma Foundation Conference: International Collaboration, Patient Connections, Industry Updates
June 2023, Vol 4, No 2
Cancer investigators and clinicians from around the world connected with patients, caregivers, and industry officials at the foundation’s conference in Salt Lake City, UT.
CCF Welcomes a New Standard of Care
By Allison Deragon, Cholangiocarcinoma Foundation
March 2023, Vol 4, No 1
CCF discusses the approval of durvalumab for cholangiocarcinoma, a milestone that, it is hoped, will open doors for other innovative immunotherapy combinations.
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).
The International Cholangiocarcinoma Research Network: Educational and Research Achievements
By Reham Abdel-Wahab, MD, PhD
December 2021, Vol 2, No 4
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).
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.

Subscribe Today!

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

I'd like to receive: