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What’s New in AML Clinical Trials?
Posted: Aug 19, 2021
What’s New in AML Clinical Trials? image

With over 300 open clinical trials, there’s a lot going on right now in AML research. Here’s a review of some of the major topics researchers are currently exploring:

AML Genetics

There are many chromosomal and genetic abnormalities in AML which makes treatment challenging. Clinical trials are currently identifying genetic changes and customizing treatments options based on the unique genetic characteristics of an individual patient’s leukemia cells. Currently, there are several ongoing multi-center studies around the world in which patients are asked to donate a sample of blood and bone marrow to be analyzed and stored for genetic analysis in future studies. 

New Drugs and Treatment Regimens

Over the last 10 years, improvements in overall survival of AML patients have been driven by advances in understanding the genetics of the disease and using this information to create new treatments. Researchers also have been working on modifying and reformulating traditional chemotherapy drugs to improve overall survival and tolerability. Additionally, there are multiple studies evaluating the combination of various older chemotherapy drugs with newer targeted therapies. Current treatment approaches under investigation include:

Targeted Therapy:

The use of drugs or substances in order to block the action of specific enzymes, proteins, or other molecules involved in the growth and survival of the leukemia cells and at the same time cause less harm to healthy cells. 

  • FLT3 inhibitors: approximately one-third of patients with AML have a mutation in the FLT3 gene which increases the growth and division of AML cells. 
    • Current drugs targeting FLT3 mutation: Sorafenib (Nexavar®), gilteritinib (Xospata®), midostaurin (Rydapt®), quizartinib (AC-220) and crenolanib. 
  • BCL-2 inhibitor: overexpression of the BCL-2 protein allows cancer cells to escape programmed cell death, a pathway that the body has designed to kill abnormal cells. 
    • Venetoclax (Venclexta®) is a BCL-2 inhibitor that has been FDA approved.
  • IDH1 and IDH2 inhibitors: IDH1 and IDH2 gene mutations cause cells to remain immature and grow quickly. 
    • Enasidenib (Idhifa®) is an FDA-approved drug that targets the IDH enzyme. 
  • PLK inhibitors: PLK are enzymes that regulate cell division. Currently, volasertib is a potent PLK-1 inhibitor being studied. 
  • Histone deacetylase (HDAC) inhibitor: HDAC is a substance that causes a chemical change that stops cancer cells from dividing.
    • Some drugs currently under study include vorinostat (Zolinza®), pracinostat (SB939) and panobinostat (Farydak®).


A biological therapy that either boosts or suppresses the immune system to help fight cancer. 

  • Monoclonal antibody treatment: researchers are analyzing specific antigens, including CD33, a marker found on most AML cells in order to create targeted therapies using antibodies. 
    • Gemtuzumab ozogamicin (Mylotarg®), an FDA-approved drug for AML patients, is a monoclonal antibody with the toxin, calicheamicin, attached to it. When the drug binds to the CD33 antigen, it releases the toxin into the leukemia cell resulting in cell death.
    • Researchers are also studying SGN-33A, another anti-CD33 monoclonal antibody designed to deliver the cytotoxic agent, pyrrolobenzodiazepine (PBD) dimer, to leukemia cells. SGN33A is undergoing clinical investigation as a single agent and in combination with hypomethylating agents. 
  • Vaccine therapy: Researchers are developing vaccines that can be personalized for the patient to stimulate a strong immune response against cancer cells. 
    • A recent study evaluated the efficacy of giving a peptide vaccine together with GM-CSF therapy to stimulate the immune system in different ways to build an effective response against AML, myelodysplastic syndromes, and other types of cancers. 
  • CAR T-cell therapy: immune cells called “T cells” are removed from the patient’s blood and altered in the lab so they have specific substances that will help them attach to leukemia cells. Then, T cells are grown in the lab and infused back into the patient’s blood, where the modified cells can seek out leukemia cells and attack them. 

If you want to learn more about open AML clinical trials, check out our:

 AML Clinical Trial Finder

The author Kerith Amen

about the author
Kerith Amen

Kerith joined HealthTree Foundation as a Community Co-Director for AML in 2022. She is a mother to a spirited eight-year-old daughter, Adair, and their beloved rescue, Violet. She lost her best friend and husband, Rob, to AML in March 2018. Kerith wishes she had a resource such as HealthTree for AML during Rob’s illness. She is a strong supporter of HealthTree's mission. She hopes that by sharing her experience, she may help other patients and caregivers navigate an AML diagnosis.

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