Treatment for Acute Myeloid Leukemia (AML)

The standard treatment approaches for acute myeloid leukemia include chemotherapy and hematopoietic stem cell (or bone marrow) transplantation. Radiation therapy — treatment with high-energy rays that destroy cancer cells — is sometimes used for AML in the central nervous system or elsewhere and can be included as part of treatment in preparation for stem cell transplant.

Our doctors plan a course of treatment for each AML patient that takes a number of factors into consideration, primarily any chromosomal alterations that have been found and the presence of gene mutations, among other factors.

Treatment for AML is typically divided into two phases: remission induction and post-remission therapy.

Remission Induction

The goal of the remission induction phase is to induce a remission, a state in which there is no visible evidence of disease and blood counts are normal. Most patients receive a two-drug regimen during this phase including cytarabine and daunorubicin or idarubicin.

Post-Remission Therapy

In the post-remission therapy phase, patients may receive additional treatment to eliminate any remaining leukemia cells. Treatment may consist of cytarabine given alone in higher doses.

Some AML patients may undergo either an autologous transplant (using the patient’s own stem cells) or an allogeneic transplant (using stem or bone marrow cells from someone other than the patient) during this phase of treatment. We are world leaders in this field. Learn more about blood and marrow stem cell transplantation.

Treatment for Acute Promyelocytic Leukemia (APL)

Patients with the form of AML called acute promyelocytic leukemia (APL) may receive initial treatment with all-trans-retinoic acid (ATRA) in combination with arsenic trioxide, both of which were pioneered at Memorial Sloan Kettering. This treatment approach induces a complete response in the vast majority of patients and allows for some lower risk patients to be spared entirely from receiving conventional chemotherapy.

Our doctors helped to develop the drugs ATRA and arsenic trioxide for the treatment of APL.

Following remission induction with ATRA and arsenic trioxide, APL patients are then given several courses of consolidation therapy, which includes additional cycles of ATRA and arsenic trioxide. Some higher risk patients may require intra thecal chemotherapy, as well as a couple years of intermittent maintenance therapy with ATRA and arsenic trioxide.

Treatment for CNS Involvement

In rare cases, AML can spread to the central nervous system, which is the part of the nervous system that consists of the brain and the spinal cord. Our doctors are experienced in preventing or controlling central nervous system involvement and may administer chemotherapy directly to the fluid that bathes the spinal cord and brain in a process known as intrathecal chemotherapy. Patients may receive high-dose systemic chemotherapy or cranial irradiation (radiation therapy to the head) to prevent the spread of disease to the central nervous system.

Investigational Approaches

Our researchers are constantly pursuing new approaches to treat ALL — approaches that can kill tumor cells directly, inhibit the body’s production of substances that promote their growth, or enhance the immune response against leukemic cells. See an up-to-date listing of our clinical trials.

Targeted Therapies

Our researchers are conducting clinical trials targeting specific genetic mutations or chromosomal abnormalities commonly found in AML. These genes include FLT3, IDH1, IDH2, and abnormalities of MLL. Such therapies have much fewer side effects as compared with conventional chemotherapy, and they appear to have the potential to induce remissions in patients with relapsed/refractory AML. In the future, some of these promising targeted therapies may be combined with chemotherapy to improve upfront therapy.

New Chemotherapy Drugs

Our doctors are continually studying new types of chemotherapy drugs that may be more effective at killing leukemia cells or have enhanced delivery to leukemia cells. 

Monoclonal Antibodies

Monoclonal antibodies are genetically engineered proteins designed to target specific sites called antigens located on the surface of tumor cells. They can kill a targeted tumor cell by stimulating the normal immune system to destroy it or by blocking key mechanisms needed for the survival of cancer cells. Our researchers pioneered this approach for the treatment of AML.

Additionally, monoclonal antibodies may serve as vehicles to deliver a radioactive substance, chemotherapy, or toxin to a cancer cell. Our researchers have used antibodies to deliver radioactive metals that emit short-range high-energy alpha particles to tumor cells and are currently studying this approach in patients with AML.


Researchers at Memorial Sloan Kettering are evaluating the effectiveness of vaccines made from protein pieces (peptides) found on AML cells that the immune system recognizes as abnormal. The studies are designed to see if these vaccines can trigger an immune response in patients with AML and other types of cancer that contain the abnormal protein.