Immunotherapy for Bile Duct Cancer
Immunotherapy uses medicines to help a person¡¯s immune system better recognize and destroy cancer cells. Many types of immunotherapy are being tested in clinical trials, and some are used to treat bile duct cancer.
How tumors are tested before immunotherapy
Your care team might use biomarker testing to find which immunotherapy drugs might work to treat your cancer. Learn more about Biomarker and Tumor Marker Tests.
Genetic changes you see in your biomarker results might include:
- A high level of microsatellite instability (MSI-H) or a defect in a mismatch repair gene (dMMR). Normally, cells have a built-in system to fix mistakes when DNA is copied. This system is called mismatch repair. If it isn¡¯t working properly (called deficient MMR, or dMMR), or if a cell makes a lot of mistakes when it copies DNA (called MSI-H, or microsatellite instability-high), these errors build up and can lead to cancer.
- A high tumor mutational burden (TMB-H), meaning the cells have a lot of gene mutations (changes in their DNA).
Immune checkpoint inhibitors
The immune system keeps itself from attacking the body's healthy cells. One way it does this is by using ¡°checkpoint¡± proteins on immune cells. These proteins act like switches that need to be turned on or off to start an immune response. Cancer cells sometimes use these checkpoints to avoid being attacked by the immune system.
Medicines that target these checkpoints are called immune checkpoint inhibitors. These can be used to treat some bile duct cancers.
Pembrolizumab (Keytruda), nivolumab (Opdivo), and dostarlimab (Jemperli) are drugs that target PD-1.
PD-1 is a checkpoint protein on immune system cells called T cells. PD-1 normally helps keep T cells from attacking other cells in the body, including some cancer cells. By blocking PD-1, this drug boosts the immune response against cancer cells. This can shrink some tumors or slow their growth.
These PD-1 inhibitors can be used in different ways to treat bile duct cancers:
- Pembrolizumab (Keytruda) with chemotherapy (gemcitabine and cisplatin) might be used to treat bile duct cancer that can¡¯t be removed by surgery or that has spread to other parts of the body.
- Pembrolizumab (Keytruda) alone can be used if biomarker testing shows a high level of microsatellite instability (MSI-H), high tumor mutational burden (TMB-H) or a defect in a mismatch repair gene (dMMR).
- Nivolumab (Opdivo) with ipilimumab (Yervoy) might be used if the tumor cells have many gene mutations (TMB-H).
- Dostarlimab (Jemperli) alone might be used if your tumor cells have trouble fixing mistakes in the DNA (dMMR).
These drugs are given as an intravenous (IV) infusion, typically every 2 to 6 weeks.
Durvalumab (Imfinzi) is a drug that targets the PD-L1 protein. This protein is related to PD-1 and is found on some tumor cells and immune cells. Blocking this protein can help boost the immune response against cancer cells.
This drug can be used along with the chemotherapy drugs gemcitabine and cisplatin. This combination helps to treat bile duct cancer that can¡¯t be removed by surgery or that has spread to other parts of the body.
This drug is given as an intravenous (IV) infusion, typically every 2 to 4 weeks.
Ipilimumab (Yervoy) is another checkpoint inhibitor, but it has a different target. It blocks CTLA-4, a protein on T cells that normally helps keep them from becoming too active. This drug helps these T cells become more active to attack cancer cells.
It can be given with Nivolumab (Opdivo) to people with bile duct tumors that have a high mutational burden (TMB-H).
This drug is given as an intravenous (IV) infusion, usually once every 3 weeks.
Possible side effects of immune checkpoint inhibitors
Some of the more common side effects of these drugs can include fatigue, cough, nausea, skin rash, poor appetite, constipation, joint pain, and diarrhea.
Other, more serious side effects occur less often.
Infusion reactions
Some people might have an infusion reaction while getting these drugs. This is like an allergic reaction. It can include fever, chills, flushing of the face, rash, itchy skin, feeling dizzy, wheezing, and trouble breathing. It¡¯s important to tell your cancer care team right away if you have any of these symptoms while getting these drugs.
Autoimmune reactions
These drugs remove one of the safeguards on the body¡¯s immune system. Sometimes the immune system responds by attacking other parts of the body, which can cause serious or even life-threatening problems in the lungs, intestines, liver, hormone-making glands, kidneys, or other organs.
It¡¯s very important to report any new side effects to someone on your cancer care team as soon as possible. If serious side effects do occur, treatment may need to be stopped and you might be given high doses of corticosteroids to suppress your immune system.
More information about immunotherapy
To learn more about how drugs that work on the immune system are used to treat cancer, see Cancer Immunotherapy.
To learn about some of the side effects listed here and how to manage them, see Managing Cancer-related Side Effects.
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- References
Developed by the ÌÇÐÄTV¹ÙÍø Cancer Society medical and editorial content team with medical review and contribution by the ÌÇÐÄTV¹ÙÍø Society of Clinical Oncology (ASCO).
Andr¨¦ T, Berton D, Curigliano G, et al. Antitumor activity and safety of dostarlimab monotherapy in patients with mismatch repair deficient solid tumors: A nonrandomized controlled trial. JAMA Oncol. 2023;9(10):1435-1444.
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Moris D, Palta M, Kim C, Allen PJ, Morse MA, Lidsky ME. Advances in the treatment of intrahepatic cholangiocarcinoma: An overview of the current and future therapeutic landscape for clinicians. CA Cancer J Clin. 2023;73(2):198-222.
National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Biliary Tract Cancers. Version 1.2026. Accessed at https://www.nccn.org/professionals/physician_gls/pdf/btc.pdf on September 9, 2026.
Last Revised: October 27, 2026
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