The Cholangiocarcinoma Foundation is an organization dedicated to targeting cures for Cholangiocarcinoma, also known as bile duct cancer because this type of cancer forms in the bile ducts. The Cholangiocarcinoma Foundation (CCF)’s mission is to find a cure and improve the quality of life for those affected by cholangiocarcinoma. Founded in 2006 in Salt Lake City, UT, by a family member who lost a loved one to cholangiocarcinoma, CCF has grown to become a leading U.S. (and increasingly international) resource in research, education, and public awareness.
Initiated in 2012, the International Hepatobiliary Neoplasia Biorepository (IHNB) is a global collaboration between CCF, Mayo Clinic, and multiple academic and medical centers to accelerate research for early diagnosis, novel therapeutics, personalized oncology, and improved clinical outcomes for patients with cholangiocarcinoma. The IHNB has assembled one of the largest bio-specimen repositories for biliary cancers worldwide and serves as a key resource for basic, translational and clinical research in bile duct cancer.
In 2014, CCF launched the International Cholangiocarcinoma Patient Registry (ICPR) to collect bile duct cancer patient information about the incidence and prevalence of cholangiocarcinoma and related disorders.
In 2015, members of CCF’s Medical, Clinical Science, and Basic Science Advisory Boards formed a global consortium of research groups, the International Cholangiocarcinoma Research Network (ICRN), that are working in concert to improve knowledge about cholangiocarcinoma etiology, prevention, early detection, treatment and prognosis with an expectation to accelerate scientific and medical progress on an international level, expedite delivery of innovative care and treatments, and improve health outcomes for patients affected by cholangiocarcinoma.
CCF’s Research Fellowship Program was established in 2015 with the goal to raise awareness about cholangiocarcinoma and inspire innovative, quality research. To date, $400,000 has been awarded to provide critical seed funding for eight physician-scientists at the beginning of their careers. In addition, CCF has funded four Conquer Cancer Young Investigator Awards totaling $240,000, resulting in a publication in Nature Magazine.Cure Today recently reported that at least half of patients with cholangiocarcinoma have mutations in their tumors that may be treatable with existing targeted drugs or immunotherapies through clinical trials.
Yet half of those diagnosed, when surveyed, said their tumor tissue hadn’t undergone molecular profiling to determine if those mutations were present.
Why? Most said they hadn’t known about this kind of testing.
The Cholangiocarcinoma Foundation — which supports and advocates for those with this rare cancer that affects the bile ducts — is determined to make sure everyone with the disease learns about, and has access to, genetic profiling. It’s working toward that goal through its new program called Mutations Matter, funded with a $125,000 grant from Bayer Pharmaceuticals.
Via educational videos and targeted email campaigns, the foundation plans to teach both patients and doctors about why this testing should be done as soon as possible after a cholangiocarcinoma diagnosis.
“Profiling needs to be done immediately, so you can plan what you want to do next when it progresses,” Stacie Lindsey, president of the foundation’s board of directors, said in a presentation at the organization’s annual conference, held Jan. 30 in Utah. She added that “cholangiocarcinoma is the most highly targetable among gastrointestinal cancers,” meaning that it expresses the most mutations that are potentially treatable with drugs already approved to treat other types of cancer.
The gap in knowledge on this topic lies mainly in the fact that 85 percent of patients with cholangiocarcinoma are treated outside major academic cancer centers, where both tumor profiling and access to clinical trials are standard, Lindsey explained. In community treatment settings, she said, oncologists who treat cholangiocarcinoma tend to be generalists rather than gastrointestinal specialists, tumor profiling is limited or discouraged, and patients are offered limited or no access to clinical trials.
The fact that just 5 percent of adults with cancer participate in clinical trials is “tricky” when it comes to a rare cancer like cholangiocarcinoma, she added, because insufficient enrollment can lead to studies being canceled or pharmaceutical companies and academia failing to invest in trials.
The foundation conducted its survey of 132 patients and their profiling histories a year and a half ago, when the immunotherapy Keytruda (pembrolizumab) was approved for all solid tumors that are considered microsatellite instability-high. About 3 percent of patients with cholangiocarcinoma have that status and are eligible for the treatment, but they can’t know that unless their tumor tissue is profiled, Lindsey said.
Of the half of responders who said in the survey that their tumors had been profiled, 62 percent noted that the test results had influenced their treatment decisions.
Lindsey cited 51-year-old survivor Matt Reidy as an example. He had his tumor profiled, and when Keytruda was approved two years later, it turned out he was eligible to take the drug.
“I started Keytruda, and within four months I had a complete response,” Reidy is quoted as saying on the Mutations Matter website. “I continued the therapy for 18 months total, and three years after starting, I remain cancer-free and consider myself cured.”
Lindsey said the foundation is running Mutations Matter with help from a variety of partners.
Komodo Health is responsible for gathering data on where patients are being treated and how, so it will be clear where education is most needed. If hot spots are found at sites within the country, the foundation can wage targeted email campaigns to community oncologists there.
Digital communications company DMD is in charge of those targeted email efforts, and EIN Communications is a group of public relations experts who have created a communications plan and are reaching out to media.
It was D2 Creative that made the simple, digestible videos — called “Illuminations” — that are available in six languages and aimed at patients and doctors. The Cholangiocarcinoma Foundation has established a blueprint for outreach to other nonprofit organizations that might benefit from sharing the videos with their members, Lindsey noted.
Finally, Perthera, Inc., a health care artificial intelligence company, is acting as a patient concierge, helping to facilitate genetic profiling and then generating a free report for each individual that includes a list of potentially relevant clinical trials. Perthera is offering its services at no cost for the program’s first year, which began in June 2018.