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Clinical Trials

When it comes to finding the right treatment for your child's cancer, a clinical trial may be an option. Your child will have access to new or improved therapies under study and not yet on the market. Discuss with your child's doctor the possibility of participating in a clinical trial, where treatment is administered in a safe, closely monitored environment.

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Research Approaches

A number of approaches are under study in clinical trials for the treatment of patients with AML.

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 for specific subgroups of patients with AML.

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 has 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:

  • Targeted therapy. This is a type of treatment 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. Approximately 25 percent of children with AML have a mutation in the FLT3 gene that allows cancer cells to grow. Patients with FLT3 mutations have a poor prognosis. FLT3 inhibitors have been approved for use in adults, but they are still being studied in children. Some of these drugs include crenolanib, gilteritinib (Xospata®), midostaurin (Rydapt®), quizartinib (AC220) and sorafenib (Nexavar®).
    • BCL2 inhibitor. Overexpression of the BCL2 protein allows cancer cells to evade “programmed cell death.” One promising drug being researched for use in AML treatment is venetoclax (Venclexta®). This BCL2 inhibitor binds to the leukemia cells and triggers apoptosis, a process that causes them to die.
  • 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 immunotherapy 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 that play a role in AML, including CD33, a marker found on most AML cells.
    • Gemtuzumab ozogamicin (Mylotarg™) is a monoclonal antibody that has 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 CD33+ AML patients.
    • Bispecific T-cell engager (BiTE) antibody technology is an immunotherapy approach that helps the body’s T cells target cancer cells. BiTEs are antibodies with two arms. One arm of the drug attaches to a specific protein on the cancer cell. The other arm of the drug activates T cells in the patient to kill the cancer cells. AMG 330 is a BiTE antibody being studied to harness T cells against cancer cells with the CD33 antigen.
    • Researchers are studying the dual affinity re-targeting (DART) protein, called flotetuzumab, which targets AML cells that express the CD123 protein.
    • CAR T-cell therapy is a promising new way to harness the immune system to fight leukemia. CAR T cells can be engineered to attack specific proteins (antigens) on the surface of AML cells. In this approach, T cells are collected from the patient’s blood. The T cells are altered in the laboratory to attack specific proteins (antigens) on the surface of leukemia cells. The T cells are then multiplied in the lab and later infused back into the patient’s blood, where they attack and kill the leukemia cells. Researchers are focusing on identifying targets, such as CD33, for this type of therapy. This research may help to develop new CAR T-cell immunotherapies for children with AML. See the free LLS booklet Chimeric Antigen Receptor (CAR) T-Cell Therapy Facts for more information on how this treatment works.


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