Taking part in a clinical trial may be the best treatment choice for some acute myeloid leukemia (AML) patients. Clinical trials are under way for patients at every treatment stage and for patients in remission. Today's standard treatments for cancer are based on earlier clinical trials. The Leukemia & Lymphoma Society continues to invest funds in AML research.
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Current AML Research and Clinical Trials
- Genetics of Leukemia. The many chromosomal and genetic abnormalities in AML make treating this particular disease challenging. There is a need to identify these genetic variations and customize treatment options based on the genetic characteristics of the leukemia cells. Newer techniques in gene sequencing have revealed previously unknown mutations that may be involved in the development of AML. This information will help researchers develop new targeted therapies, tailored to specific disease characteristics in each patient. There are several ongoing multi-center studies around the world in which patients are asked to donate samples of blood and bone marrow so these can be analyzed and stored for future study.
- New Drugs and Treatment Regimens. Researchers are trying to find more effective and safer treatments for AML. They are studying new drugs, as well as existing drugs given in different doses and with different methods of delivery (such as liposomal encapsulation). In the last 10 years, improvements in overall survival of AML patients has been driven by advances in the understanding of the genetics of the disease, as well as finding new ways to use existing medications. Researchers are continuing to modify and reformulate traditional chemotherapy drugs to improve overall survival. They are also evaluating combinations of chemotherapy drugs with newer targeted therapies. Treatment approaches under investigation include:
- Targeted therapy. A treatment that uses drugs or other substances to block the action of certain enzymes, proteins or other molecules involved in the growth and survival of cancer cells but cause less harm to healthy cells.
- FLT3 inhibitor. Approximately one-third of AML patients have a mutation in the FLT3 gene that can increase the growth and division of AML cells. Patients with FLT3 mutations have a poor prognosis. Sorafenib (Nexavar®), gilteritinib (Xospata®), midostaurin (Rydapt®), quizartinib (AC-220) and crenolanib are FLT3 inhibitors that target this gene mutation.
- BCL-2 inhibitor. Overexpression of the BCL2 protein allows cancer cells to evade “programmed cell death.” One promising drug under research is venetoclax (Venclexta®), a BCL-2 inhibitor that binds to the leukemia cell and triggers apoptosis, a process that causes the cell to die.
- IDH1 and IDH2 inhibitors. Mutations in the IDH1 and IDH2 genes cause cells to remain immature and grow too quickly. Several IDH inhibitors are being studied in patients with such genetic markers in their leukemia cells, including enasidenib (Idhifa®), which is already FDA approved.
- PLK inhibitors. Volasertib is a potent PLK inhibitor that is being studied. Volasertib is designed to inhibit the activity of PLK1, an enzyme that regulates cell division. This inhibition ultimately results in cell death.
- HDAC inhibitor. Histone deacetylase (HDAC) inhibitor is a substance that causes a chemical change that stops cancer cells from dividing. HDAC inhibitors under study in clinical trials include vorinostat (Zolinza®), pracinostat (SB939) and panobinostat (Farydak®).
- Immunotherapy. A type of biological therapy that is designed to either boost or suppress the immune system to help the body fight cancer. It uses materials made either by the body or in a laboratory to improve, target, or restore immune system function.
- Monoclonal antibody treatment. This treatment is a type of targeted therapy being studied to treat AML. Antibodies are part of the immune system. Normally, the body creates antibodies in response to an antigen such as bacteria, viruses and even cancer cells. The antibodies attach to the antigen in order to help destroy the antigen. Researchers are analyzing specific antigens, including CD33, a marker that is found on most AML cells.
- Gemtuzumab ozogamicin (Mylotarg®) is a monoclonal antibody with the toxin, calicheamicin, attached to it. When gemtuzumab ozogamicin binds to the CD33 antigen, it releases the toxin resulting in the death of the myeloid cell. Gemtuzumab ozogamicin is FDA approved for AML patients.
- Researchers are also studying SGN-33A, another anti-CD33 monoclonal antibody designed to deliver the cytotoxic agent, pyrrolobenzodiazepine (PBD) dimer, to myeloid leukemia cells. SGN33A is undergoing clinical investigation as a single agent and in combination with hypomethylating agents. Another approach is using the treatment AMG330 to harness T cells, which are part of the body’s immune system, to target cells with the CD33 antigen.
- Vaccine therapy. Researchers are developing vaccines that can be personalized to individual patients to stimulate a strong immune response against their cancer. For instance, a recent study evaluated the efficacy of giving a peptide vaccine together with GM-CSF therapy to stimulate the immune system in different ways in order to build an effective response against AML, myelodysplastic syndromes (MDS) and other types of cancer.
- CAR T-cell therapy. This is a promising new way to get the immune system to fight leukemia. For this technique, immune cells called “T cells” are removed from the patient’s blood and altered in the lab so they have specific substances (called “chimeric antigen receptors” [CARs]) that will help them attach to leukemia cells. The T cells are then grown in the lab and infused back into the patient’s blood, where they can now seek out the leukemia cells and attack them.
- Monoclonal antibody treatment. This treatment is a type of targeted therapy being studied to treat AML. Antibodies are part of the immune system. Normally, the body creates antibodies in response to an antigen such as bacteria, viruses and even cancer cells. The antibodies attach to the antigen in order to help destroy the antigen. Researchers are analyzing specific antigens, including CD33, a marker that is found on most AML cells.
- Targeted therapy. A treatment that uses drugs or other substances to block the action of certain enzymes, proteins or other molecules involved in the growth and survival of cancer cells but cause less harm to healthy cells.
For information about the drugs listed on this page, visit Drug Listings.
Related Links
- Download or order The Leukemia & Lymphoma Society's free booklet, Acute Myeloid Leukemia.