CCF Corner

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.

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).

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.

“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.

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.

The COVID-19 pandemic has changed the way we do business, the way we interact with one another, and other tangible aspects of our work and personal lives. The pandemic has also provided many of us with much-needed time for personal reflection. Often, when we have the opportunity to take a step back, or in this case when we are forced to step back, the result of our thoughtful reflection can be an opportunity to slingshot forward.