Acute myelogenous leukemia

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Targeted therapy for acute myelogenous leukemia (AML)

Targeted therapy is sometimes used to treat acute myelogenous leukemia (AML). It uses drugs to target specific molecules (such as proteins) on cancer cells or inside them. These molecules help send signals that tell cells to grow or divide. By targeting these molecules, the drugs stop the growth and spread of cancer cells and limit harm to normal cells. Targeted therapy may also be called molecular targeted therapy.

The healthcare team will consider your personal needs to plan the drugs, doses and schedules of targeted therapy. You may also receive other treatments.

Targeted therapy drugs used for acute myelogenous leukemia

There are a few types of targeted therapy drugs used to treat AML.

FLT3 inhibitors

Some people with AML have a mutated FLT3 gene in the leukemia cells that helps them grow. Drugs that inhibit or block FLT3 can help treat AML that has the FLT3 gene mutation.

Midostaurin (Rydapt) is an FLT3 inhibitor used along with chemotherapy to treat newly diagnosed AML with the FLT3 mutation.

Gilteritinib (Xospata) is an FLT3 inhibitor used to treat AML with the FLT3 mutation that does not respond to other treatments or comes back after treatment.

IDH inhibitors

Some people with AML have a mutated IDH1 or IDH2 gene in the leukemia cells that stops them from maturing the way normal cells do. Drugs that inhibit or block IDH1 or IDH2 can help treat AML that has the IDH1 or IDH2 mutation.

Enasidenib mesylate (Idhifa) is an IDH inhibitor used to treat AML with the IDH1 or IDH2 mutation that does not respond to other treatments or comes back after treatment.

Monoclonal antibodies

Monoclonal antibodies are both a type of immunotherapy and a targeted therapy. They bind to specific antigens on cancer cells to help destroy them.

Gemtuzumab ozogamicin (Mylotarg) is a monoclonal antibody linked to a chemotherapy drug. It targets the CD33 antigen on leukemia cells and delivers a chemotherapy drug called calicheamicin. It may be used along with chemotherapy for treatment of CD33-positive AML.

Side effects

Side effects can happen with any type of treatment for AML, but every person’s experience is different. Some people have many side effects. Other people have few or none at all.

Targeted therapy attacks cancer cells but doesn’t usually damage healthy cells, so there are usually fewer and less severe side effects than with chemotherapy or radiation therapy. Chemotherapy and radiation therapy can damage healthy cells along with cancer cells.

Side effects can happen any time during, immediately after or a few days or weeks after targeted therapy. Sometimes late side effects develop months or years after targeted therapy. Most side effects go away on their own or can be treated, but some side effects may last a long time or become permanent.

Side effects of targeted therapy will depend mainly on the type of drug or combination of drugs, the dose, how it’s given and your overall health. Some common side effects of targeted therapy for AML are:

Tell your healthcare team if you have these side effects or others you think might be from targeted therapy. The sooner you tell them of any problems, the sooner they can suggest ways to help you deal with them.

Information about specific cancer drugs

Details on specific drugs change regularly. Find out more about sources of drug information and where to get details on specific drugs.

Questions to ask about targeted therapy

Find out more about targeted therapy. To make the decisions that are right for you, ask the healthcare team questions about targeted therapy.


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