Targeted cancer drugs for acute myeloid leukaemia (AML)
Targeted cancer drugs work by ‘targeting’ the differences that help a cancer cell to survive and grow.
There are different types of targeted cancer drugs for AML. They are grouped together depending on how they work. The main targeted cancer drugs in AML include:
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midostaurin
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gemtuzumab ozogamicin (Mylotarg)
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venetoclax
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gilteritinib
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ivosidenib
You might have other targeted cancer drugs if you are having treatment as part of a 
.
When you have targeted cancer drugs for AML
You might have a targeted cancer drug with chemotherapy. Or you might have it on its own.
Treatment for AML is generally divided into intensive and non intensive treatment.
Intensive treatment
Intensive treatment aims to cure your AML. You might have this if you doctor thinks you are fit and well enough to have this treatment.
The targeted cancer drug you might have in the intensive treatment includes:
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midostaurin OR
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gemtuzumab ozogamicin (Mylotarg)
Non intensive treatment
Non intensive treatment aims to control your cancer for as long as possible.
Non intensive treatment generally causes fewer and less severe side effects. You might have this treatment if you are not that fit and well, or you have other health conditions. For example, heart or lung problems.
You might have a drug called venetoclax or ivosidenib as part of your non intensive treatment.
AML that comes back or resists treatment
AML that isn’t responding to treatment is called resistant or refractory leukaemia. AML that comes back after treatment has finished is called relapse. In these situations, you might have a targeted drug called gilteritinib.
Tests on your leukaemia cells
When you are diagnosed with AML you have several different tests that look for changes:
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on the surface of your leukaemia cells
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in the genes and chromosomes of your leukaemia cells
Your doctor looks at a sample of your cancer from a bone marrow test to do this. Your doctor may use a blood test to do this, but this is less common.
The results of the tests show whether a targeted cancer drug is suitable for you.
Names of targeted cancer drugs for AML
Midostaurin (Rydapt)
Midostaurin is a type of targeted drug called a cancer growth blocker. Cancer growth blockers stop the growth factors that trigger the cancer cell to divide and grow.
You might have midostaurin if your AML has a called FLT-3. You may have it:
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alongside chemotherapy to get rid of your leukaemia (induction) and reduce the risk of it coming back (consolidation)
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on its own as a maintenance treatment following consolidation
You have this drug through a drip into your vein.
Venetoclax (Venclyxto)
This is another type of cancer growth blocker. You have venetoclax with a chemotherapy drug. This is either azacitidine or low dose cytarabine.
You may have this combination of drugs as a first treatment for AML if you are unable to have intensive chemotherapy.
Venetoclax comes as tablets that you take once a day.
Gilteritinib (Xospata)
This is also a cancer growth blocker. You might have gilteritinib if your AML has a gene change (mutation) called FLT-3. It’s for people with AML that has come back or relapsed.
You have gilteritinib on its own. It comes as tablets that you take once a day.
Gemtuzumab ozogamicin (Mylotarg)
Gemtuzumab ozogamicin belongs to a group of targeted drugs called monoclonal antibodies (MABs). MABS target specific proteins on cancer cells and work in different ways to kill the cancer cell and stop it from growing.
You might have gemtuzumab ozogamicin if there is a protein receptor called CD33 on your leukaemia cells. The gemtuzumab sticks to all the CD33 proteins it finds. Then ozogamicin enters the cells and kills them.
You have gemtuzumab ozogamicin with the chemotherapy drugs daunorubicin and cytarabine. We have more information about this combination of drugs in our cancer drugs section.
Ivosidenib
Ivosidenib is also a type of cancer growth blocker. You might have ivosidenib if your AML has a gene change (mutation) called IDH1. You have this with another drug called azacitidine.
You may have this combination of drugs as a first treatment for AML if you are unable to have intensive chemotherapy.
Ivosidenib comes as a tablet that you take once a day.
Side effects
Targeted cancer drugs can cause different side effects. Some of these can be serious. Your doctor or nurse will talk to you about this. Always tell them about any side effects you have and follow the advice they give you.
Choose a drug on our A to Z list to read about side effects and find out more about the drug.
Research into targeted cancer drugs for AML
Researchers are looking at:
- new targeted cancer drugs
- different combinations of these drugs with other treatment
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.
