Recently, as a result of FGFR clinical trials for bile duct cancer (also known as cholangiocarcinoma), the FDA approved pemigatinib, a targeted therapy used to treat bile duct cancer. Pemigatinib was approved for adult patients with unresectable, locally advanced, or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement. Until this approval, a combination of chemotherapy drugs were used as standard-of-care treatment.
What is Bile Duct Cancer?
Bile duct cancer is a rare disease in which cancer cells form in the bile ducts. It occurs in the bile ducts inside (intrahepatic cholangiocarcinoma) or outside (extrahepatic cholangiocarcinoma) the liver. Risk factors include primary sclerosing cholangitis, chronic ulcerative colitis, and cysts in the bile duct.
Bile duct cancer is a cancer that forms in the narrow tubes (bile ducts) that carry bile, which is a fluid made by the liver to break down fats during digestion. Bile ducts connect the liver to the gallbladder and the small intestine. It passes through the cystic duct, which connects the gallbladder to the common hepatic duct, and flows into the small intestine.
The most common signs and symptoms of bile duct cancer include:
- Yellowing of the skin and the whites of the eyes (jaundice)
- Extremely itchy skin
- Oily and light-colored stools
- Darkening of the urine
- Abdominal pain
- Unexplained weight loss
Biliary Tract Cancer Epidemiology
Bile duct cancer makes up for about 2% of all cancers, while its incidence within the community is about 1-2 per 100,000. It is more common in men than in women and most patients are over 65 years of age. In the United States alone, bile duct cancer affects less than 5,000 people per year, making it the 5th most common gastrointestinal cancer.
Treatment of Bile Duct Cancer
Different types of treatments are available for patients with bile duct cancer. Some treatments are standard and are currently being used, and some are being tested in clinical trials. Treatment of biliary tract cancer is based on whether the cancer can be completely removed by surgery, stage, and symptoms caused by the cancer. Bile duct cancer is usually found after it has spread and can rarely be removed by surgery.
For patients with advanced stage disease, external or internal radiation therapy or chemotherapy can be considered. In addition, clinical trials of external radiation therapy combined with hyperthermia therapy, radiosensitizer drugs, or chemotherapy are being tested.
Drugs such as pemigatinib, which is a targeted therapy for FGFR2 fusions and other rearrangements, were first tested in clinical trials. Massive Bio can help you understand if you are eligible for clinical trials. Many patients can take part in a clinical trial, regardless of their geographical location, that is specific to their cancer type and biomarkers tested.