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Clinical Trials

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 disease particularly challenging. There is a need to identify these genetic variations and customize treatment options based on the specific genetic characteristics of the leukemia cells. New gene sequencing techniques 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.

New Drugs and Treatment Regimens. Researchers are working to develop safer and more effective treatments for AML. They are studying new drugs, as well as the use of different doses and delivery methods for existing drugs. During the last few decades, advances in the understanding of disease genetics have led to improvements in the overall survival of AML patients. Researchers are also continuing to modify and reformulate traditional chemotherapy drugs and are evaluating combinations of chemotherapy drugs with newer targeted therapies
to improve overall survival. Treatment approaches being studied for use in AML patients include:

  • Novel Targeted Therapies. A targeted therapy is a type of 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, while causing less harm to healthy cells.
    • FLT3 Inhibitors. Newer generation FLT3 inhibitors, including quizartinib (formerly AC-220) and crenolanib, are being investigated in combination with chemotherapy to treat patients with newly diagnosed and relapsed AML with FLT3 mutations.
    • p53 Inhibitors. Mutations in the p53 gene deactivate processes (tumor suppressor genes) which prevent normal healthy cells from becoming cancerous. APR-246 (eprenetapopt), a novel small molecular inhibitor, is being studied for the treatment of AML with p53 mutations. APR-246 targets and restores the function of the mutated gene.
    • Menin Inhibitors. Research has shown that the menin protein plays a role in the development and growth of some leukemias with mutations in the KMT2A gene (formerly known as the mixed-lineage leukemia or MLL gene), as well as the NPM-1 (nucleophosmin-1) gene. In the laboratory, menin inhibitors have been shown to produce anti-leukemic effects in AML with KMT2A and NPM-1 mutations. Two menin inhibitors (KO-539 and SNDX-5613) are being studied in clinical trials and may represent promising approaches to treat these specific genetic types of leukemia.
    • Targeting Cancer Metabolism. A new drug called devimistat (CPI-613®) targets enzymes involved in cancer cell energy metabolism and increases the sensitivity of cancer cells to a range of chemotherapies. Combining existing treatments with devimistat will potentially make them more effective and allow for the possibility of using lower doses of drugs that are generally toxic.
  • Immunotherapy. This is a type of biological therapy designed to either boost or suppress the immune system, as needed, to help the body fight cancer. It uses substances made naturally by the body or synthetically in a laboratory to improve, target or restore immune system function.
    • Monoclonal Antibody Therapy. This 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 antigens, such as bacteria, viruses and even cancer cells. The antibodies attach to the antigens in order to help destroy them. Researchers are analyzing specific antigens as potential targets, including CD123, which is found on most AML cells. A promising example is magrolimab (an anti-CD47 antibody), which works by making leukemia cells recognizable to macrophage cells. These are the immune system cells whose job is to “eat” and remove infected and diseased cells from the body. Treatment with magrolimab combined with azacitidine appears to be effective in older patients with newly diagnosed AML, even in those with mutations like p53. Another antibody called cusatuzumab targets CD70, which is expressed on leukemia cells, particularly leukemic stems cells—the specific population of cells responsible for disease relapse. Treatment with cusatuzumab combined with azacitidine is being explored in older patients with AML.
    • Bispecific T-Cell Engager (BiTE) Antibody Therapy. Specialized antibodies have been designed to simultaneously target AML cells and activate the patient’s immune system to attack these leukemia cells. One such agent, called AMG-330, is designed to harness T cells to target AML cells with the CD33 antigen. Another, called flotetuzumab, recruits T cells to target CD123, which is expressed on chemotherapy-resistant AML cells.
    • Vaccine Therapy. Researchers are developing vaccines that can be personalized to individual patients to stimulate a strong immune response against their cancer. These vaccines are designed for patients who are in remission. They work by generating an immune response against the leukemia cells to hopefully prevent the disease from coming back in the future. Some of the targets of this vaccine approach include the WT1 (Wilm’s tumor 1) antigen expressed on many AML cells.
    • Chimeric Antigen Receptor (CAR) T-Cell Therapy. In this type of immunotherapy, the patient’s own immune cells are genetically engineered to recognize and attack cancer cells. Scientists are conducting research to see whether these treatments are effective in patients with AML. They are also exploring whether it is possible to use someone else’s engineered immune cells in an “off the shelf” approach, instead of the patient’s own cells, to treat patients more quickly. Click here to learn more.

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