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A Watershed Moment for the Cholangiocarcinoma Community

Web Exclusives - Cholangiocarcinoma
Stacie C. Lindsey
Founder and CEO Cholangiocarcinoma Foundation
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.

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