Targeted cancer drugs

Targeted cancer drugs work by ‘targeting’ those differences that help a cancer cell to survive and grow. Find out what they are and when you might have this treatment for acute myeloid leukaemia (AML).


Before you have some types of targeted drugs you might need to have a test using some of your leukaemia cells to find out whether the treatment is likely to work. Your doctor looks at a sample from your bone marrow test or less commonly a blood test. These tests look for changes in certain proteins or genes 

Your consultant can tell you if this applies to your treatment. This is not the case for all targeted drugs and you don’t always need this test.

Types of targeted drugs for AML

There are many different types of targeted drugs and these are grouped according to the way they work.

Cancer growth blockers

For example, cancer growth blockers stop the growth factors that trigger the cancer cell to divide and grow. Midostaurin (Rydapt) is a type of cancer growth blocker called a tyrosine kinase inhibitor. Tyrosine kinases help to send growth signals in cells, so blocking them stops the cell growing and dividing. 

You might have midostaurin if your AML has a gene change (mutation) called FLT-3. You may have it:

  • alongside chemotherapy to get rid of your leukaemia (induction) and reduce the risk of it coming back (consolidation) 
  • on its own as a maintenance treatment following consolidation

You have as this drug through a drip into your vein.

Monoclonal antibodies

Another group of targeted drug is a monoclonal antibody (MAB). These target specific proteins on cancer cells and work in different ways to kill the cancer cell and stop it from growing.

Gemtuzumab ozogamicin (Mylotarg) is a MAB. You might have this if there is a protein receptor called CD33 on your leukaemia cells. 

You have gemtuzumab ozogamicin tablets with chemotherapy at different points during your induction and consolidation treatment.

Ask your specialist about targeted drugs. They can explain:

  • whether this treatment is suitable for you
  • what the aim of treatment would be
  • what it would involve and the side effects

Side effects

Side effects depend on the drug you are having. 

Clinical trials

Researchers continue to look at new and different combinations of targeted cancer drugs and to treat AML. Your doctor might ask you to have treatment as part of a clinical trial.

When you go home

This treatment can be difficult to cope with. Your nurse will give you an advice line number to call if you have any problems at home.

Tell your doctor or nurse about any problems or side effects you have.

Last reviewed: 
07 Dec 2020
Next review due: 
07 Dec 2023
  • Acute myeloblastic leukaemias in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up
    M Fey and C Buske
    Annals of Oncology, 2013. Volume 24, Supplement 6, vi138 to vi143

  • National Institute for Health and Care Excellence (NICE), various guidance
    Accessed May, 2020

  • Scottish Medicines Consortium (SMC), various guidance
    Accessed May 2020

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