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 trials — Testing new combinations to get people into remission, often comparing different chemotherapy backbones or adding targeted drugs like FLT3 or IDH inhibitors.
- Maintenance trials — Testing whether continuing treatment after remission prevents relapse, particularly after transplant.
- Relapsed/refractory trials — Testing new drugs or combinations for people whose leukemia has returned or didn't respond to initial treatment.
- Transplant trials — Studies of different transplant approaches, graft-versus-host disease prevention, and decisions about who benefits most from transplant.
- Older adult trials — Testing gentler treatment approaches, often venetoclax-based, for people who can't tolerate intensive chemotherapy.
Recently added Acute Myeloid Leukemia trials
Interferon-α Maintenance in Favorable-Risk AML
Acute myeloid leukemia (AML) is a blood cancer with a substantial risk of relapse after initial treatment. Although patients with favorable-risk AML generally have better outcomes, relapse remains a major concern. Interferon-alpha (IFN-α) may help prevent relapse, but its effectiveness as maintenance therapy has not been confirmed in prospective studies. This prospective, single-arm study aims to evaluate the efficacy and safety of IFN-α as maintenance therapy in patients with favorable-risk AML. Participants will receive IFN-α maintenance treatment and will be followed for disease relapse, survival, and treatment-related adverse events.
A Study Comparing Mesutoclax + Azacitidine Versus Venetoclax + Azacitidine in Untreated Acute Myeloid Leukemia (AML) Patients
A Phase III study comparing Mesutoclax + azacitidine versus venetoclax + azacitidine in newly diagnosed acute myeloid leukemia (AML) patients
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