Medications for AML include chemotherapy and newer targeted therapies. With several options available, the right medication for you will depend on the specific characteristics of your cancer.

Acute myeloid leukemia (AML) is a blood cancer that causes your bone marrow to produce abnormal platelets and blood cells. It’s the most common form of acute (quickly developing) leukemia in adults and requires prompt treatment.

Several treatment options for AML are available. Your treatment team will carefully consider the type of AML you have, whether the cancer has spread, and other factors when deciding your best treatment options. Medications are typically the first-line treatment.

Keep reading to learn more about the medications doctors use to treat AML, including chemotherapy drugs and targeted therapies.

Chemotherapy (“chemo”) is a class of drugs that works by either killing cancer cells directly or damaging them to a point where they stop dividing and creating new cancerous cells. While effective, chemo is also known to cause side effects, such as hair loss, nausea, and infections.

Experts estimate that 2 out of 3 people with newly diagnosed AML who receive chemo as an induction therapy enter remission after treatment.

Options often used in the systemic treatment of AML include:

  • azacitidine (Onureg, Vidaza)
  • cytosine arabinoside (cytarabine)
  • daunorubicin (Cerubidine)
  • decitabine (Dacogen)
  • idarubicin (Idamycin)
  • mitoxantrone (Novantrone)
  • methotrexate (Trexall, Xatmep) for combination therapy only

In some cases, doctors may also consider combination drugs, such as:

  • daunorubicin and cytarabine liposome (Vyxeos)
  • cytarabine and methotrexate

The way healthcare professionals administer chemotherapy for AML depends on whether the cancer has spread to your central nervous system (CNS). If it has, they inject chemo directly into the cerebrospinal fluid, the fluid around your brain and spinal cord. This process is also called intrathecal chemotherapy.

If the cancer has not spread to the CNS, a doctor might recommend systemic chemo instead. This is administered by mouth or as an injection.

Sometimes, doctors may combine chemo with a stem cell transplant to help ensure the creation of new, healthy blood cells that these cancer drugs might otherwise destroy.

Targeted therapies are among the most widely researched treatment options for AML because they can target specific genes and their proteins that contribute to cancer growth.

These drugs can also cause side effects, though they range in severity and incidence between individuals. Skin changes are the most common, with other possibilities including infections, swelling, and more.

Before administering these drugs, your oncologist will test to see if you have a genetic mutation.

Doctors may use the following targeted therapy at the same time as, or in place of, chemo for AML:

FLT3 inhibitors

FLT3 inhibitors target mutations in the FLT3 gene, which can sometimes contribute to cancer. Examples include:

You would usually take these 1 to 2 times daily.

IDH inhibitors

IDH inhibitors block excess proteins related to genetic mutations affecting the IDH1 or IDH2 genes. Unlike other targeted therapies, IDH inhibitors can directly help immature leukemia blood cells transition into typical, healthy ones.

IDH inhibitors currently approved to treat AML include:

You take these by mouth 1 to 2 times per day.

BCL-2 inhibitors

BCL-2 inhibitors target cellular proteins of the same name. Doctors most often prescribe these drugs for adults over age 75 who receive an AML diagnosis for the first time.

Currently, the only approved BCL-2 inhibitor for AML treatment is venetoclax (Venclexta).

You take this drug by mouth once daily.

Hedgehog pathway inhibitors

Like BCL-2 inhibitors, doctors most commonly prescribe hedgehog pathway inhibitors for people ages 75 or older.

The hedgehog signaling pathway is a key part of cell differentiation, the process in which a stem cell becomes a specific type of cell. Overactivation of this pathway can lead to some cancers, like leukemia.

Currently, the only approved treatment for AML in this class is glasdegib (Daurismo). It’s not recommended for people who are or could become pregnant.

You also take these pills daily.

Monoclonal antibodies

Monoclonal antibodies target cells with the help of lab-created proteins. While numerous monoclonal antibodies address a variety of illnesses and chronic conditions, only one is currently approved for AML: gemtuzumab ozogamicin (Mylotarg).

Mylotarg works by attaching to CD33 proteins inside AML cells. It can work alongside chemotherapy by making it more effective.

Unlike other targeted therapies for AML, healthcare professionals administer Mylotarg intravenously (by IV).

APL is an aggressive subtype of AML. To help quickly stop this type of leukemia from progressing further, a healthcare professional may recommend the following cancer medications:

All trans-retinoid acid (ATRA)

ATRA is a substance derived from vitamin A. ATRA may treat APL by potentially killing cancer cells and preventing them from dividing and growing. You take this drug by mouth.

Arsenic trioxide (ATO)

ATO treatment (Trisenox) is an injectable medication doctors may recommend in cases of APL where the PML-RARA gene is involved. It works by destroying proteins on the gene that can subsequently help destroy leukemia cancer cells.

Doctors may recommend Trisenox for APL that hasn’t improved or if the cancer has returned after using other AML treatment methods.

What’s the drug of choice in acute myeloid leukemia?

The best AML drug of choice depends on several factors, such as your age and overall health, whether the cancer has progressed, and whether you’ve previously undergone treatment and are experiencing a relapse.

What is the new drug treatment for acute myeloid leukemia?

Quizartinib (Vanflyta) is the most recent drug approved for AML treatment, having received approval from the Food and Drug Administration (FDA) in July 2023. It’s an FLT3 inhibitor, a type of targeted therapy.

Researchers continue to look into new targeted therapies and immunotherapies as potential AML treatments. Menin inhibitors are among the newest classes showing promise.

What is the survival rate for acute myeloid leukemia?

The 5-year relative survival rate for AML was 31.9% between 2014 and 2020. This means 31.9% of people were alive 5 years after diagnosis.

Factors that affect survival rate include AML that has spread to the CNS, older age, and a white blood cell count higher than 100,000 per microliter of blood when diagnosed.

AML is an aggressive type of leukemia, especially if it’s not treated quickly. Many treatments are available for this cancer, with new therapies continually under research.

Talk with a healthcare professional about your medication options to determine how they might fit into your overall AML treatment plan.