Research in acute myeloid leukemia (AML)

We are always learning more about cancer. Researchers and healthcare professionals use what they learn from research studies to develop better practices that will help find and treat acute myeloid leukemia (AML). They are also looking for ways to improve the quality of life of people with AML.

The following is a selection of research showing promise for AML. We’ve included information from PubMed, which is the research publication database of the National Library of Medicine. Each research article in PubMed has an identity number (called a PMID) that links to a brief overview (called an abstract). We have also included links to abstracts of the research presented at meetings of the American Society of Clinical Oncology (ASCO), which are held throughout the year. You can find information about ongoing clinical trials in Canada from and Clinical trials are given an identifier called a national clinical trial (NCT) number. The NCT number links to information about the clinical trial.


Researchers are trying to find better ways to help doctors predict a prognosis (how likely it is that the cancer can be successfully treated or that it will come back after treatment) for AML. They are also trying to determine the best treatment options based on certain characteristics of the disease, such as specific biomarkers. Biomarkers are substances, such as proteins, genes or pieces of genetic material like DNA and RNA, that are found naturally in the body. They can be measured in body fluids, including blood, lymph fluid and bone marrow, or on certain types of cells, such as cancer cells. Doctors can look for and measure these biomarkers to check if cancer is present or is responding to treatment.

Prognostic and predictive biomarkers for AML can be used to help plan treatment. Prognostic biomarkers can be used to identify people who have a greater risk that the disease will progress or come back after treatment (recur or relapse). Predictive biomarkers are used to identify people who are more likely to have a favourable or unfavourable effect from treatment compared to people without the biomarker. Researchers are looking at the following biomarkers to see if they can help doctors diagnose, predict a prognosis for and find out which treatments will benefit a person with AML:


Researchers are looking for new ways to improve treatment for AML. Advances in cancer treatment and new ways to manage the side effects from treatment have improved the outlook and quality of life for many people with cancer. The following is noteworthy research into treatment for AML.


Researchers are studying new chemotherapy drugs to treat AML. They are testing these drugs alone and with other chemotherapy drugs typically used to treat AML. Chemotherapy drugs that are showing promise include:

Targeted therapy

Targeted therapy drugs target specific molecules (usually proteins) that cause cancer cells to grow. Researchers are studying the following types of targeted therapy drugs in treating AML.

FLT3 inhibitors

About 30% of people with AML have mutations in the FLT3 gene, which is a type of tyrosine kinase receptor. People with a mutation in this gene have a poorer prognosis because there is a greater chance that the AML will relapse. Compounds that inhibit FLT3 are called FLT3 inhibitors. Midostaurin (Rydapt) is an FLT3 inhibitor currently used to treat people with AML and the FLT3 mutation. Researchers are looking at the following FLT3 inhibitors as possible treatments for people with AML who have the FLT3 mutation:

Histone deacetylase (HDAC) inhibitors

HDAC is an enzyme that can make cancer cells grow. HDAC inhibitors are targeted therapy drugs that block the actions of HDAC to slow or stop the growth of cancer cells. HDAC inhibitors may be a treatment option for AML. They may improve the effectiveness of chemotherapy by making AML cells more sensitive to certain drugs, such as cytarabine (Cytosar), daunorubicin (Cerubidine, daunomycin) or azacitidine (Vidaza). Some HDAC inhibitors showing promise include:

Mouse double minute 2 (MDM2) homolog inhibitors

MDM2 is a protein that regulates TP53 (also known as p53). TP53 is a gene that helps control cell growth and may help limit the growth of cancer cells. Cancer cells can grow out of control when they make too much (overexpress) MDM2. MDM2 inhibitors are targeted therapy drugs that block the actions of MDM2 to slow or stop the growth of cancer cells. Some clinical trials are looking at the following MDM2 inhibitors as possible treatments for AML:

Aurora kinase inhibitors

Aurora kinases are overexpressed in several different types of cancer. Aurora kinase inhibitors work by blocking aurora kinases and causing cancer cells to die. Researchers are studying aurora kinase inhibitors to treat AML, including the following:

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. Researchers are looking at using the following monoclonal antibodies alone and with other drugs to treat AML.

Gemtuzumab ozogamicin is an antibody that targets the CD33 antigen on leukemia cells and delivers a powerful chemotherapy drug called calicheamicin. Researchers are studying gemtuzumab ozogamicin in clinical trials to treat CD33-positive AML (Haematologica, PMID 26921360; Annals of Hematology, PMID 25307457; Journal of Clinical Oncology, PMID 26811524; Leukemia, PMID 26365212).

Other monoclonal antibodies that researchers are studying to treat AML include:

Mutant IDH1 and IDH2 enzyme inhibitors

Isocitrate dehydrogenase 1 and 2 (IDH1 and IDH2) are important metabolic enzymes. Some people with AML have IDH1 or IDH2 gene mutations. Research shows that inhibitors of mutant IDH1 and IDH2 may be useful in treating AML with that specific gene mutation. Enasidenib is an inhibitor of IDH2 that is effective in treating relapsed or refractory AML with certain genetic mutations. It was recently approved by Health Canada and the FDA in the United States to treat relapsed or refractory AML in certain people with the IDH2 gene mutation (Blood, PMID 28588019, PMID 28588020). Ivosidenib is an inhibitor of mutant IDH1 that researchers are studying in clinical trials to treat AML with the IDH1 mutation (ASCO, Abstract TPS7074;, NCT03173248).

Other targeted therapy drugs

Researchers are also studying the following targeted therapy drugs as possible treatments for AML:


Immunotherapy boosts or helps the immune system find and destroy cancer cells. Researchers are studying the following types of immunotherapy for AML.


Immune checkpoint inhibitors work by stopping cancer cells from affecting immune system cells in our bodies. The immune system normally stops itself from attacking healthy cells in the body by having some cells make specific proteins called checkpoints. Cancer cells sometimes use these checkpoints to avoid being attacked by the immune system. Immune checkpoint inhibitors are monoclonal antibodies that work by blocking checkpoint proteins so T cells (a type of white blood cell) can attack and kill cancers cells. Researchers are studying the immune checkpoint inhibitor nivolumab (Opdivo) in clinical trials as maintenance therapy for high-risk AML (ASCO, Abstract 7014;, NCT02532231).

Dendritic cell vaccines are made from a person’s white blood cells. In the lab, the white blood cells are exposed to cancer cells, antigens found on cancer cells or chemicals, which turn them into a specialized type of white blood cell called a dendritic cell. The dendritic cells are then injected back into the person and help other immune cells in the body find and attack cancer cells. Dendritic cell vaccines are showing promise in helping to prevent or delay recurrence or relapse of AML (Cancer, PMID 28411378; Blood, PMID 28830889).

Stem cell transplant

Stem cell transplant replaces a person’s blood-forming (hematopoietic) stem cells. It is used when stem cells or the bone marrow has been damaged by chemotherapy drugs, radiation therapy or disease (such as cancer). The new stem cells make healthy blood cells.

Stem cell transplants are commonly used to treat AML. Researchers are studying the following types of stem cell transplant to see if they could be safer, easier and more effective for people with AML.

Reduced-intensity allogeneic transplant uses lower doses of chemotherapy or radiation therapy before the transplant. The lower doses don’t completely destroy the recipient’s bone marrow, so blood cell counts don’t drop as low as they do in standard stem cell transplants. For this reason, there is a lower risk for complications. This may be of great importance for older adults who can’t tolerate the higher doses of chemotherapy and radiation normally used with stem cell transplants (Journal of Clinical Oncology, PMID 28380315, PMID 28463633, PMID 26527780, PMID 25534391; Bone Marrow Transplantation, PMID 28112749; Biology of Blood and Marrow Transplantation, PMID 25460357, PMID 25960307; Journal of Cancer Research and Clinical Oncology, PMID 26424692; Haematologica, PMID 27561720).

Half-matched stem cell transplant (also called haploidentical transplant) is a new type of stem cell transplant. To do a stem cell transplant, the donor and recipient are matched through a process called human leukocyte antigen (HLA) typing. Usually the antigens on the donor’s and recipient’s stem cells need to be a perfect or nearly perfect match. But a half-match stem cell transplant can be done if only half of the donor’s and recipient’s stem cells match. All parents and children, and about 50% of siblings, are half-matched. Half-matched stem cell transplant may be an option for adults with AML who do not have a matched donor (Blood, PMID 26130705, PMID 25940714; Leukemia, PMID 25882700).

Cord blood stem cell transplant uses stem cells collected from blood in the placenta and umbilical cord of newborn babies. Researchers have found that cord blood seems to be a good alternate source for stem cells for people who don’t have a suitable related or unrelated donor. Cord blood stem cell transplants are also linked with a lower risk of a life-threatening condition called graft-versus-host disease (GVHD). But this type of transplant may have a higher risk of graft failure, which is when the donated stem cells don’t start to make new blood cells or they start to work but then suddenly stop (Current Opinion in Hematology, PMID 23314845). More research is needed to determine the role that cord blood transplants may play in treating AML (British Journal of Haematology, PMID 25272241; Biology of Blood and Marrow Transplantation, PMID 25498906, PMID 25460357, PMID 26238810; Haematologica, PMID 27125981).

Lowering the risk for relapse or recurrence and GVHD is a significant area of research regarding treatment of AML. Some studies include using the following:

Learn more about cancer research

Researchers continue to try to find out more about AML. Clinical trials are research studies that test new ways to prevent, detect, treat or manage AML. Clinical trials provide information about the safety and effectiveness of new approaches to see if they should become widely available. Most of the standard treatments for AML were first shown to be effective through clinical trials.

Find out more about clinical trials.