Targeted and immunotherapy drugs for bile duct cancer

You might have targeted or immunotherapy drugs for advanced bile duct cancer. This is cancer that has grown into the lymph nodes Open a glossary item or organs near the bile duct such as the liver or small bowel. Or that has spread to another part of the body such as the lungs.

Some targeted and immunotherapy drugs are only used to treat bile duct cancer that has certain gene Open a glossary item changes (mutations).

What are targeted and immunotherapy drugs?

Targeted cancer drugs work by targeting the differences in cancer cells that help them to grow and survive. Other drugs help the immune system to attack cancer. These are called immunotherapies.

Unfortunately, targeted and immunotherapy drugs cannot cure bile duct cancer. But they can control it and improve symptoms.

When you might have targeted or immunotherapy drugs for bile duct cancer

You might have immunotherapy to control the cancer if it can't be removed, or if it has come back after surgery. You normally have this with chemotherapy. This called your first line treatment. It is the first treatment you have when you are diagnosed with cancer. Or it is the first treatment you have if the cancer comes back.

If this treatment stops working, you might have more treatment. This is called second line treatment. It might be either:

  • further immunotherapy
  • a targeted cancer drug

Which drug you have depends on whether the cancer has certain gene changes or not. Your doctor will test the cancer for these changes. If the cancer doesn't have any of these, you usually have further chemotherapy. 

Tests on the cancer cells

Doctors look for certain gene changes in the bile duct cancer. These help it to grow and divide. Some cancer drugs only work if there is a particular change in one of the cancer’s genes.

You doctor tests a sample of the bile duct cancer from when you were first diagnosed. This is to see if it has any of these changes. The result of the test shows which cancer drugs are suitable for you. 

Bile duct cancer can have a number of different gene changes. These include:

  • changes in the genes involved in DNA Open a glossary itemrepair
  • fibroblast growth factor receptor 2 (FGFR2)
  • isocitrate dehydrogenase-1 (IDH-1)

Targeted cancer drugs

The following targeted cancer drugs are used to treat bile duct cancer:

  • pemigatinib
  • futibatinib
  • ivosidenib

Pemigatinib and futibatinib

These are both a type of targeted cancer drug called a tyrosine kinase inhibitor (TKI). They block chemical messengers called tyrosine kinases. Tyrosine kinase helps to send growth signals in cells. Blocking it stops the cell from growing and dividing. 

You might have pemigatinib if your first line treatment hasn’t worked and the cancer has a change in the FGFR2 gene.

If you are in England, Wales or Northern Ireland, you might have futibatinib instead of pemigatinib. The Scottish Medicines Consortium (SMC) approves drugs for use in Scotland. It is currently looking at futibatinib as a treatment for bile duct cancer. 

You take pemigatinib or futibatinib for as long as the treatment is helping you and the side effects aren’t too bad.

Ivosidenib

Ivosidenib is a type of cancer growth blocker. It works by finding and blocking proteins made by the IDH-1 gene. These proteins help the cancer grow. Blocking them stops or slows down the growth of the cancer.

You might have ivosidenib if your first line treatment hasn’t worked and the cancer has a change in the IDH-1 gene.

You take ivosidenib for as long as the treatment is helping you and the side effects aren’t too bad.

Immunotherapy

The following immunotherapy drugs are used to treat bile duct cancer:

  • durvalumab
  • pembrolizumab

Durvalumab

Durvalumab is a type of immunotherapy called a checkpoint inhibitor. These block the proteins in cells that stop our immune system from attacking cancer.

You normally have durvalumab with the chemotherapy drugs gemcitabine and cisplatin as your first line treatment. You have this for for 6 months. You then carry on having durvalumab on it's own. You have it for as long as it works, and the side effects aren't too bad.

Pembrolizumab (Keytruda)

Pembrolizumab is a checkpoint inhibitor. It blocks a protein called a programmed cell death protein 1 (PD-1). PD-1 stops the immune system from attacking the cancer cells.

You may have pembrolizumab if your first line treatment hasn’t worked and the cancer has DNA changes called microsatellite instability Open a glossary item (MSI-high) or mismatch repair deficiency Open a glossary item (dMMR). 

You can have pembrolizumab for up to 2 years as long as it is controlling the cancer.

Having targeted cancer drugs and immunotherapy

You normally have immunotherapy drugs through a tube into your bloodstream.

You usually take targeted cancer drugs as tablets or capsules.

Into the bloodstream

You have the treatment through a drip into your arm. A nurse puts a small tube (a cannula) into one of your veins and connects the drip to it.

You may need a central line. This is a long plastic tube that gives the drugs into a large vein, either in your chest or through a vein in your arm. It stays in while you’re having treatment. This may be for a number of months.

Tablets or capsules

You must take tablets and capsules according to the instructions your doctor or pharmacist gives you.

You should take the right dose, not more or less.

Talk to your healthcare team before you stop taking a cancer drug, or if you have missed a dose.

Side effects

Everyone is different and the side effects vary from person to person. The side effects you get can depend on:

  • which drug you have
  • whether you have it alone or with other drugs
  • the amount of drug you have (the dose)
  • your general health

A side effect may get better or worse during the course of your treatment. Or more side effects may develop as the treatment goes on. For more information about the side effects of your treatment go to the individual drugs page.

Research into targeted drugs and immunotherapy for bile duct cancer

Researchers continue to look at different targeted cancer drugs and immunotherapy for bile duct cancer. This includes:

  • new types of immunotherapy
  • targeted cancer drugs already used for other cancer types
  • combining targeted cancer drugs with chemotherapy

Your doctor might ask you to have treatment as part of a clinical trial.

  • National Institute for Health and Care Excellence (NICE)
    Various guidance
    Last accessed October 2024

  • Scottish Medicines Compendium
    Various guidance
    Last accessed October 2024

  • Biliary tract cancer: ESMO Clinical Practice Guideline for diagnosis, treatment and follow up
    A Vogel and others
    Annals of Oncology, 2023. Volume 34, Issue 2, Pages 127–140

  • British Society of Gastroenterology guidelines for the diagnosis and management of cholangiocarcinoma
    SM Rushbrook and others
    Gut, 2024. Volume 73, Pages 16-46

  • Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study
    GK Abou-Alfa and others
    Lancet Oncology, 2020. Volume 21, Issue 6, Pages 796-807

  • The information on this page is based on literature searches and specialist checking. We used many references and there are too many to list here. Please contact patientinformation@cancer.org.uk with details of the particular issue you are interested in if you need additional references for this information.

Last reviewed: 
11 Oct 2024
Next review due: 
11 Oct 2027

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