New Treatments & Clinical Trials for Acute Myeloid Leukemia

Acute myeloid leukemia is a fast-moving blood cancer where bone marrow makes abnormal white blood cells. Treatment depends on genetic markers in the leukemia cells, age, and overall health. Standard care includes chemotherapy and sometimes stem cell transplant, but outcomes vary widely based on disease subtype.

What's actually going on in research

Trials are testing targeted drugs against specific genetic mutations like FLT3, IDH1, and IDH2. Researchers are studying venetoclax combinations that let older patients avoid intensive chemotherapy, new antibody-drug conjugates that deliver toxins directly to leukemia cells, and checkpoint inhibitors to help the immune system fight the disease. CAR T-cell therapy and other cell therapies are being adapted from their success in other blood cancers.

What to know before you search

Eligibility typically depends on genetic mutations in leukemia cells, disease status (newly diagnosed or relapsed), prior treatments, age, kidney and liver function, and overall fitness for intensive therapy.

What types of trials are currently open

• First-line treatment trials — Testing new drugs or combinations as initial treatment, often comparing them to standard intensive chemotherapy. Some focus on older patients who can't tolerate high-dose chemo.
• Relapse trials — Studying treatments for AML that returns after initial treatment. These often test drugs with different mechanisms than the first treatment used.
• Targeted therapy trials — Testing drugs designed for specific mutations in your leukemia, like FLT3, IDH1, IDH2, or TP53. Entry requires genetic testing of cancer cells.
• Maintenance trials — Testing treatments given after remission to prevent relapse, especially for people not getting transplants.
• Stem cell transplant studies — Studying ways to improve transplant success, including conditioning regimens, donor types, and prevention of graft-versus-host disease.