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.
As we grappled with the diagnosis, we focused on hope rather than despair. We felt fortunate that only 5 years earlier, in 2010, there was finally an FDA-approved standard-of-care treatment option for CCA—a chemotherapy combination of cisplatin and gemcitabine. In the words of Steve’s medical oncologist, there was a 30% chance that this treatment would do nothing, a 30% chance that it would provide disease stability, and a 30% chance that it would shrink the tumors.
Steve was one of the lucky ones; his tumors responded and shrunk, buying him precious time with a good quality of life to live life to the fullest and be the beneficiary of experimental treatment options to further extend his life.
When a patient is diagnosed with a rare cancer such as CCA, access to that level of care is imperative, and sadly, is beyond the reach of too many patients. Our approach to manage his cancer was to follow the science to find treatments and clinical trials that offered patients progression-free survival of 1 month to years.
Our ability to follow the science was greatly facilitated by the Cholangiocarcinoma Foundation and its network of patient advocates and mentors. Through the foundation, we found a community of patients and caregivers from around the world, all united to share information, resources, and knowledge to help every patient have the longest and best quality of life possible. A treatment success for one patient represented a possible treatment success for all of us. The story of Steve’s experience with CCA is not different from that of so many other patients.
I share Steve’s story to help build awareness of CCA, and the need for more funding, to improve therapies and conduct research. In Steve’s own words, “This is a terrible cancer, and more needs to be done to fight it.”
Today, except for pemigatinib (Pemazyre), the only new FDA-approved treatment that targets the 9% to 14% of patients with CCA whose disease harbors the FGFR2 fusion, there has not been a significant change in the standard of care since before Steve was diagnosed.
The incidence of CCA is on the rise.1 It accounts for approximately 15% of all primary liver tumors and approximately 3% of all GI cancers, but nearly 20% of GI cancer deaths are from hepatobiliary cancers. The 5-year survival rate is 7% to 20%, and for the fortunate few who are able to have curative surgery or treatment, the recurrence rate is unacceptably high.1-3
There is a desperate need of greater community recognition and research support to help save thousands of lives that are needlessly and painfully lost every year. A rare cancer diagnosis is challenging, because there will be limited treatment options, and a limited number of specialists who understand the disease and are working on research to improve outcomes.4 For more than a decade, researchers have understood that unlocking the scientific mysteries of a rare cancer has the potential to broaden our understanding behind the science of common and rare cancers.4,5
Although the National Institutes of Health (NIH) classifies CCA under primary liver cancer, recent research shows that bile duct cancer and its subgroups are biologically distinct and need different treatment modalities.5 Woefully underfunded, the need for more research to better understand this disease is essential to provide patients with better outcomes and hope for a long life, as well as to support and advance biomedical research that will benefit other cancers and diseases.
The treatment and clinical trial options are limited for patients with CCA, but so many more exist today than at any other moment in modern medical history. Patients with CCA have participated in and have had curative responses to experimental surgical, radiation, and immunotherapy treatments. This gives our patient community great hope and also directly contributes to advancing the science to better understand CCA, and cancer more broadly.
The 2 FDA-approved treatment options for CCA are now followed by many more potential therapies that are currently in the pipeline. This is thanks to the tireless efforts of a small and dedicated cohort of researchers and clinicians from around the world, many in residence at our premiere cancer centers and the NIH. And yet, research funding to support further advances is woefully inadequate.
The National Cancer Institute currently has more than 10,000 funded research projects totaling $7.6 billion. Only 35 of those projects are to support CCA research for a funding total of $24.2 million.6
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