Targeted Therapy for Acute Myeloid Leukemia (AML)

In recent years, drugs that target specific parts of cancer cells have been developed. Targeted drugs work differently from standard chemotherapy (chemo) drugs and tend to have different side effects. They can sometimes be helpful even when chemo isn’t, or they can be used along with chemo to help it work better.

Some of these drugs can be useful in treating certain people with acute myeloid leukemia (AML).

FLT3 inhibitors

In some people with AML, the leukemia cells have a mutation in the FLT3 gene. This gene helps the cells make a protein (also called FLT3) that helps the cells grow. Drugs that target the FLT3 protein can help treat some of these leukemias.

Midostaurin (Rydapt) is a drug that works by blocking FLT3 and several other proteins on cancer cells that can help the cells grow. This drug can be used along with certain chemotherapy drugs to treat newly diagnosed adults whose leukemia cells have an FLT3 gene mutation. Your doctor can test your blood to see if you have this mutation.

Midostaurin is taken by mouth twice a day.

Common side effects can include low levels of white blood cells (with increased risk of infection), fever, nausea, vomiting, redness or sores in the mouth, headache, muscle or bone pain, bruising, nosebleeds, high blood sugar levels, and upper respiratory infections.

Less often, this drug can cause serious lung problems, which might show up as a cough, chest pain, or shortness of breath. Tell someone on your cancer care team right away if you have any of these symptoms.

IDH inhibitors

In some people with AML, the leukemia cells have a mutation in the IDH1 or IDH2 gene. These genes help the cells make certain proteins, which are also called IDH1 and IDH2. Mutations in one of these genes can stop blood cells from maturing the way they normally would.

Targeted drugs called IDH inhibitors can block these IDH proteins. These drugs seem to work by helping the leukemia cells mature (differentiate) into more normal cells. Because if this, they are sometimes referred to as a differentiation agents.

These drugs can be used to treat AML with an IDH1 or IDH2 mutation. Your doctor can test your blood or bone marrow to see if your leukemia cells have one of these mutations.

  • Ivosidenib (Tibsovo) is an IDH1 inhibitor. It can be used to treat AML with an IDH1 mutation that comes back after treatment or is no longer responding to other treatments.
  • Enasidenib (Idhifa) is an IDH2 inhibitor. It can be used to treat AML with an IDH2 mutation that comes back after treatment or is no longer responding to other treatments. 

These drugs are taken by mouth, once a day.

Common side effects can include nausea, vomiting, diarrhea, fatigue, joint pain, shortness of breath, increased levels of bilirubin (a substance found in bile), and loss of appetite. 

An important possible side effect of these drugs is known as differentiation syndrome. This occurs when the leukemia cells release certain chemicals into the blood. It most oftenoccurs during the first treatment cycle. Symptoms can include fever, breathing problems from fluid buildup in the lungs and around the heart, low blood pressure, liver or kidney damage, and severe fluid buildup elsewhere in the body. It can often be treated by stopping the drugs for a while and giving a steroid such as dexamethasone.

Gemtuzumab ozogamicin (Mylotarg)

This targeted therapy consists of a monoclonal antibody (a man-made immune protein) linked to a chemotherapy drug. The antibody attaches to a protein called CD33, which is found on most AML cells. The antibody acts like a homing signal, bringing the chemo drug to the leukemia cells, where it enters the cells and kills them when they try to divide into new cells.

This drug can be used along with chemotherapy as part of the initial treatment of AML that has the CD33 protein. It can also be used by itself, either as the first treatment (especially in people who might not be healthy enough for intense chemo), or if other treatments are no longer working. It is given as an infusion into a vein (IV).

The most common side effects are fever, nausea and vomiting, low levels of blood cells (with increased risks of infection, bleeding, and fatigue), swelling and sores in the mouth, constipation, rash, and headaches.

Less common but more serious side effects can include:

  • Severe liver damage, including veno-occlusive disease (blockage of veins in the liver)
  • Reactions during the infusion (similar to an allergic reaction). You will likely be given medicines before each infusion to help prevent this.
  • Serious or life-threatening infections, especially in people who have already had a stem cell transplant
  • Changes in the rhythm of the heart

To learn more about targeted therapy drugs as a treatment for cancer, see Targeted Cancer Therapy.

The American Cancer Society medical and editorial content team
Our team is made up of doctors and master's-prepared nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Larson RA. Induction therapy for acute myeloid leukemia in younger adults. UpToDate. 2018. Accessed at on June 20, 2018.

National Comprehensive Cancer Network. NCCN Practice Guidelines in Oncology: Acute Myeloid Leukemia. V.1.2018. Accessed at on June 20, 2018.

Last Medical Review: August 21, 2018 Last Revised: August 21, 2018

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