Where acute myeloid leukemia treatment is heading

About 22,000 people in the US are diagnosed with acute myeloid leukemia each year, mostly older adults. AML is now treated by its mutations: FLT3, IDH1/2, NPM1 and others each have targeted pills. Lower-intensity venetoclax combinations have become standard for older patients, and menin-blocking pills are the newest class, moving from relapse into first treatment.

• 855studies open or running
• 75phase 3 trials
• 3,470papers published this year
• 40NIH-funded projects

1. Now

   Nov 2026

   Gentler treatment beat intensive chemoHappened 14.5 mo event-free survival

   Azacitidine-venetoclax gave 14.5 months of event-free survival vs 6.2 with intensive chemo in fit, mostly higher-risk adults.

   14.5 mo: event-free survival with azacitidine-venetoclax, vs 6.2 months.

   • PARADIGM trial

   An all-pill regimen for older patientsHappened 63% remission

   Oral decitabine-cedazuridine plus venetoclax: 63% reached complete or near-complete remission.

   63%: complete response or complete response with incomplete recovery.

   • All-oral AML trial

   Menin inhibitors approved after relapseHappened 46.9% responded

   Revumenib produced responses in 46.9% of relapsed NPM1-mutated AML; ziftomenib was also approved.

   46.9%: of tumors responded.

   • AUGMENT-101 trial (NPM1)

2. 6 months

   by May 2027

   Standard regimen for older patients holdsLikely

   Venetoclax-azacitidine remains the base that new drugs are added to.

   • VIALE-A long-term follow-up

3. 12 months

   by Nov 2027

   A drug to protect stem cell transplantsPossible

   MO-TRANS (366 people) tests mocravimod to reduce relapse after transplant; due May 2027.

   • MO-TRANS

4. 3 years

   by late 2029

   Menin inhibitors as first treatmentPossible 46.9% responded

   Phase 3 trials of revumenib, ziftomenib and bleximenib added to chemo or ven-aza report from 2027 to 2029.

   46.9%: of tumors responded.

   • AUGMENT-101 trial (NPM1)
   • REVEAL-ND NPM1
   • cAMeLot-2

   Triplets for IDH1-mutated AMLPossible

   EVOLVE 1 tests adding venetoclax to ivosidenib and azacitidine; due December 2028.

   • EVOLVE 1

5. 5 years

   by late 2031

   Lower-intensity treatment for fit adultsLong shot

   If confirmed in larger trials, venetoclax-based regimens may replace intensive chemo for more people.

   • PARADIGM trial

The research paths

Borrowed from other cancers

Open to join now

• Newly diagnosed NPM1-mutated

  Revumenib with intensive chemo

  Phase 3 · 173 sites

  Locations and eligibility →

• Newly diagnosed NPM1 or KMT2A

  Ziftomenib combinations

  Phase 3 · 141 sites

  Locations and eligibility →

• Newly diagnosed, unfit

  Bleximenib with ven-aza

  Phase 3 · 270 sites

  Locations and eligibility →

• Undergoing stem cell transplant

  Mocravimod

  Phase 3 · 111 sites

  Locations and eligibility →

See every recruiting AML trial →

When the trials finish

Completion dates the trials registered. Results usually follow 3 to 12 months later.

What just happened

Standard treatment today

• Fit, favorable or intermediate riskIntensive "7+3" chemotherapy, plus midostaurin or quizartinib if FLT3-mutated; transplant for some.
• Older or unfitVenetoclax plus azacitidine or decitabine; ivosidenib combinations for IDH1.
• RelapsedTargeted pills by mutation: gilteritinib (FLT3), ivosidenib/olutasidenib (IDH1), enasidenib (IDH2), revumenib or ziftomenib (NPM1, KMT2A).
• After transplantMaintenance with targeted pills for some mutations.

See AML trials recruiting now →