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Condition Guide

New Treatments & Clinical Trials for Acute Myeloid Leukemia

Last updated September 2026Data from ClinicalTrials.gov1,020 active trials
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Acute myeloid leukemia is a blood cancer that starts in the bone marrow and moves quickly. Treatment typically begins with intensive chemotherapy, sometimes followed by stem cell transplant. Survival depends heavily on age, genetic changes in the cancer cells, and how well the leukemia responds to initial treatment.

What's actually going on in research

Trials are testing targeted therapies that match specific mutations (like FLT3 and IDH inhibitors), venetoclax combinations that have changed treatment for older adults, and newer approaches to stem cell transplant. Researchers are also studying menin inhibitors for leukemias with certain genetic rearrangements, and ways to prevent relapse after remission.

Menin inhibitors

This new drug class targets leukemias driven by KMT2A rearrangements and NPM1 mutations. Early trial results show some people achieving remission when other treatments had failed.

Maintenance therapy

Studies are testing whether giving targeted drugs after transplant or chemotherapy can prevent the cancer from returning. This approach aims to turn AML into a manageable condition for more people.

Combination strategies

Trials are pairing venetoclax with different chemotherapy backbones and targeted drugs. The goal is finding gentler regimens that work for people who can't tolerate intensive chemotherapy.

What to know before you search

Eligibility typically depends on genetic mutations in the leukemia, prior treatments, transplant status, age, and overall fitness for intensive therapy.

What types of trials are currently open

  • Induction trialsTesting new combinations to get people into remission, often comparing different chemotherapy backbones or adding targeted drugs like FLT3 or IDH inhibitors.
  • Maintenance trialsTesting whether continuing treatment after remission prevents relapse, particularly after transplant.
  • Relapsed/refractory trialsTesting new drugs or combinations for people whose leukemia has returned or didn't respond to initial treatment.
  • Transplant trialsStudies of different transplant approaches, graft-versus-host disease prevention, and decisions about who benefits most from transplant.
  • Older adult trialsTesting gentler treatment approaches, often venetoclax-based, for people who can't tolerate intensive chemotherapy.

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