Targeted therapy for acute myeloid leukemia

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Targeted therapy 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.

Targeted therapy is sometimes used to treat acute myeloid leukemia (AML) with certain genetic mutations or AML that expresses certain proteins. If you have targeted therapy, your healthcare team will use what they know about the cancer and about your health to plan the drugs, doses and schedules.

You may have targeted therapy:

  • to treat AML with certain genetic mutations
  • to treat AML that expresses certain proteins
  • along with chemotherapy to help it work better
  • to treat AML that doesn't respond to chemotherapy
  • to treat older people or people with health conditions that prevent them from having high-dose chemotherapy

Types of targeted therapy used for AML

Different types of targeted therapy are used for 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 used along with chemotherapy to treat newly diagnosed AML with the FLT3 mutation.

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

Sorafenib (Nexavar) is used along with chemotherapy to treat AML with the FLT3 mutation.

These drugs are taken by mouth.

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 (Idhifa) is an IDH inhibitor used to treat AML with the IDH2 mutation. This drug is taken by mouth.

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. It is given by IV.

BCL2 inhibitors

BCL2 is a protein in cancer cells that helps them live longer. BCL2 inhibitors work by inhibiting or blocking BCL2. Venetoclax (Venclexta) is a BCL2 inhibitor that may be used along with chemotherapy to treat people who are older or not healthy enough to have strong chemotherapy. It's taken by mouth. It is usually given in combination with a chemotherapy drug such as azacitidine (Vidaza), decitabine (Dacogen, Demylocan, Inqovi) or low-dose cytarabine (Cytosar).

Hedgehog pathway inhibitors

Some people with AML have leukemia cells with mutations in genes that are part of a cell signalling pathway called hedgehog. Hedgehog pathway inhibitors work by inhibiting or blocking the hedgehog pathway. Glasdegib (Daurismo) is a hedgehog pathway inhibitor that may be used along with chemotherapy to treat people who are older or not healthy enough to have strong chemotherapy. It is taken by mouth.

Side effects

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. Tell your healthcare team if you have side effects that you think are from targeted therapy. The sooner you tell them of any problems, the sooner they can suggest ways to help you deal with them. Talk to your doctor or pharmacist about what side effects to expect.

Find out more about targeted therapy

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

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

Expert review and references

  • Robert Turner, MD, FRCPC
  • John Storring, MD, CM