Targeted Therapy for Acute Myeloid Leukemia

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

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

Midostaurin (Rydapt)

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. Researchers are now developing drugs that target the FLT3 protein.

Midostaurin 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.

This drug is taken as pills, 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 your doctor or nurse right away if you have any of these symptoms.

Enasidenib (Idhifa)

In some people with AML, the leukemia cells have a mutation in the IDH2 gene. This gene helps the cells make a protein (also called IDH2) that helps the cells grow. Mutations in the IDH2 gene can stop blood cells from maturing the way they normally would. 

Enasidenib is a drug that works by blocking the IDH2 protein on leukemia cells. It seems to work by helping the leukemia cells mature (differentiate) into more normal cells. Because if this, it is sometimes referred to as a differentiation agent

This drug can be used to treat AML that comes back after treatment or is no longer responding to other treatments, and in which the leukemia cells have an IDH2 gene mutation. Your doctor can test your blood to see if you have this mutation.

This drug is taken as pills, once a day.

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

An important possible side effect of this drug is known as differentiation syndrome. This occurs when the leukemia cells release certain chemicals into the blood. It is most often seen during the first cycle of treatment. 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 is a targeted therapy that consists of a monoclonal antibody (a manmade immune protein) linked to a chemotherapy drug. The antibody attaches to 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. 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.

Last Medical Review: May 8, 2017 Last Revised: November 21, 2017

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