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🗺️ Disease Atlas · Hematologic disorder

acute myeloid leukemia

Acute myeloid leukemia (AML) is a group of neoplasms arising from precursor cells committed to the myeloid cell-line differentiation. All of them are characterized by clonal expansion of myeloid blasts. AML manifests by fever, pallor, anemia, hemorrhages and recurrent infections.

🔔 Follow weekly
🧬12979genes / proteins with evidence
💊587drugs and clinical candidates
🧪12+recruiting trials worldwide
✅19approved
📈99phase 3
🧬

Understand acute myeloid leukemia

Cancer · Cells that grow out of control
🎓 Learn more

What it is

Leukemia is a term for cancers of the blood cells. Leukemia starts in blood-forming tissues such as the bone marrow. Your bone marrow makes the cells which will develop into white blood cells, red blood cells, and platelets. Each type of cell has a different job:

White blood cells help your body fight infection

Red blood cells deliver oxygen from your lungs to your tissues and organs

From MedlinePlus: Acute Myeloid Leukemia (US National Library of Medicine) ↗

🧬 Cancer: Cancer is a group of diseases in which some of the body's cells grow and divide without control, and can spread to other parts of the body.

❓ Why it happens

  • Changes (mutations) build up in the genes that tell a cell when to grow, divide and die.
  • Most of these changes happen during life: tobacco, too much sun, alcohol, some infections (such as HPV and hepatitis B and C), radiation, obesity and ageing all raise the risk.
  • Some people inherit a faulty gene that raises their risk, such as BRCA1 or BRCA2.

🫀 What it does to the body

  • Cancer cells form a lump (tumour) that can press on or invade nearby organs.
  • They can travel through the blood or lymph to start new tumours elsewhere (metastasis).
  • Blood cancers crowd out healthy blood cells in the bone marrow.

🧱 Why it is hard to cure

  • Every cancer is different, even between two people with the same type - and inside one tumour.
  • Cancer cells keep changing, so they can learn to resist a medicine that worked at first.
  • Cancer cells are the body's own cells, so killing them without harming healthy cells is hard.
  • Many cancers cause no symptoms until they have spread, and a new medicine often takes more than ten years to go from the lab to patients.

🚀 What research is trying now

  • Targeted medicines against the exact gene change in a tumour (for example EGFR, ALK or KRAS).
  • Immunotherapy that helps the immune system recognise cancer, including CAR-T cell therapy.
  • Personalised cancer vaccines built from each tumour's mutations, now in late trials.
  • Blood tests that look for tumour DNA to find cancer earlier, and AI to read scans and slides.

General information about cancer, reviewed by people; it does not describe any one person. Below: the evidence for acute myeloid leukemia itself.

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AI briefing

Where the science on acute myeloid leukemia stands, from the evidence on this page

AI reads the targets, drugs, trials and papers below and writes where the science stands — every claim cited. It is shared with everyone who opens acute myeloid leukemia.

🎯

Top targets

Genes and proteins ranked by Open Targets evidence (0-1)
DNMT3A DNA methyltransferase 3 alphaPROTACsmall molecule🧊 AlphaFold structure ↗ 0.87
CEBPA CCAAT enhancer binding protein alphaPROTAC🧊 AlphaFold structure ↗ 0.85
FLT3 fms related receptor tyrosine kinase 3PROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.85
IDH1 isocitrate dehydrogenase (NADP(+)) 1PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.80
TET2 tet methylcytosine dioxygenase 2PROTACsmall molecule🧊 AlphaFold structure ↗ 0.79
SRSF2 serine and arginine rich splicing factor 2PROTAC🧊 AlphaFold structure ↗ 0.79
RUNX1 RUNX family transcription factor 1PROTACsmall molecule🧊 AlphaFold structure ↗ 0.78
KIT KIT proto-oncogene, receptor tyrosine kinasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.77
ASXL1 ASXL transcriptional regulator 1PROTAC🧊 AlphaFold structure ↗ 0.77
GATA2 GATA binding protein 2PROTAC🧊 AlphaFold structure ↗ 0.76
KRAS KRas proto-oncogene, GTPasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.75
NRAS NRAS proto-oncogene, GTPasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.75
IDH2 isocitrate dehydrogenase (NADP(+)) 2PROTACsmall molecule🧊 AlphaFold structure ↗ 0.75
TP53 tumor protein p53PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.74
JAK2 Janus kinase 2PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.73

Green labels: what the target is known to be druggable by. The score combines genetics, somatic mutations, drugs, expression and literature.

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Drugs and candidates

Every medicine linked to acute myeloid leukemia, by how far it has got
Approved · 19
AzacitidineDNA (cytosine-5)-methyltransferase 3A inhibitor; DNA (cytosine-5)-methyltransferase 1 inh…Approved
CytarabineDNA polymerase (alpha/delta/epsilon) inhibitorApproved
DaunorubicinDNA topoisomerase II alpha inhibitorApproved
Daunorubicin HydrochlorideDNA topoisomerase II alpha inhibitorApproved
Enasidenib MesylateIsocitrate dehydrogenase [NADP], mitochondrial inhibitorApproved
Gemtuzumab OzogamicinMyeloid cell surface antigen CD33 binding agentApproved
Show all 19
GilteritinibTyrosine-protein kinase receptor FLT3 inhibitor; Tyrosine-protein kinase receptor UFO inh…Approved
Gilteritinib FumarateTyrosine-protein kinase receptor FLT3 inhibitor; Tyrosine-protein kinase receptor UFO inh…Approved
GlasdegibSmoothened homolog antagonistApproved
Glasdegib MaleateSmoothened homolog antagonistApproved
Histamine DihydrochlorideApproved
Idarubicin HydrochlorideDNA topoisomerase II alpha inhibitorApproved
IvosidenibIsocitrate dehydrogenase [NADP] cytoplasmic inhibitorApproved
MelphalanApproved
MidostaurinTyrosine-protein kinase receptor FLT3 inhibitor; Stem cell growth factor receptor inhibit…Approved
OlutasidenibIsocitrate dehydrogenase [NADP] cytoplasmic inhibitorApproved
Quizartinib DihydrochlorideTyrosine-protein kinase receptor FLT3 inhibitorApproved
SargramostimGranulocyte-macrophage colony-stimulating factor receptor agonistApproved
TagraxofuspInterleukin-3 receptor subunit alpha binding agent; Elongation factor 2 inhibitorApproved
In phase 2 / 3 · 20
AclarubicinPhase 3
AldesleukinInterleukin-2 receptor agonistPhase 3
AlemtuzumabCAMPATH-1 antigen inhibitorPhase 3
AmonafidePhase 3
AmsacrineDNA topoisomerase II inhibitorPhase 3
Antilymphocyte Immunoglobulin (Horse)Phase 3
Show all 20
Apg-2575Apoptosis regulator Bcl-2 inhibitorPhase 3
Arsenic TrioxideThioredoxin reductase 1 inhibitorPhase 3
AsparaginasePhase 3
Asparaginase Erwinia ChrysanthemiPhase 3
BleximenibPhase 3
Bortezomib26S proteasome inhibitorPhase 3
BusulfanPhase 3
CarmustineGlutathione reductase inhibitorPhase 3
Caspofungin1,3-beta-glucan synthase inhibitorPhase 3
CedazuridineCytidine deaminase inhibitorPhase 3
CladribinePhase 3
ClofarabineRibonucleoside-diphosphate reductase RR1 inhibitor; DNA polymerase (alpha/delta/epsilon) …Phase 3
Cpi 613Phase 3
CrenolanibPlatelet-derived growth factor receptor inhibitor; Tyrosine-protein kinase receptor FLT3 …Phase 3
Early trials · 20
8-ChloroadenosinePhase 1/2
Ab-8939Phase 1/2
AcadesineAMP-activated protein kinase, AMPK activatorPhase 1/2
Akn-028Tyrosine-protein kinase receptor FLT3 inhibitorPhase 1/2
AlrizomadlinTumour suppressor p53/oncoprotein Mdm2 inhibitorPhase 1/2
AsciminibTyrosine-protein kinase ABL inhibitor; Bcr/Abl fusion protein inhibitorPhase 1/2
Show all 20
At-9283Tyrosine-protein kinase ABL inhibitor; Serine/threonine-protein kinase Aurora-A inhibitorPhase 1/2
AtezolizumabProgrammed cell death 1 ligand 1 inhibitorPhase 1/2
AtorvastatinHMG-CoA reductase inhibitorPhase 1/2
AvelumabProgrammed cell death 1 ligand 1 otherPhase 1/2
Azd-5153Phase 1/2
Azd-5991Phase 1/2
AzenosertibSerine/threonine-protein kinase WEE1 inhibitorPhase 1/2
Bendamustine HydrochloridePhase 1/2
BeroterkibPhase 1/2
BexmarilimabPhase 1/2
BinimetinibDual specificity mitogen-activated protein kinase kinase 1 inhibitor; Dual specificity mi…Phase 1/2
BirabresibBromodomain and extra-terminal motif (BET) inhibitorPhase 1/2
BirinapantcIAP1/cIAP2 inhibitorPhase 1/2
Bn-104Phase 1/2
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Recruiting trials

Worldwide, open to new patients now
Clinical Study Protocol of Maintenance Therapy With Venetoclax in Elderly Patients With AML in First Complete…NCT05909293 · NA · 📍 NanjingRegistry of Myelodysplastic Syndromes and Therapy-related Acute Myeloid LeukemiaNCT06956755 · n/a · 📍 RouenEvaluation of Combined Sensitising and Hypomethylating Therapy Outcomes in AML PDXNCT06782971 · n/a · 📍 CanberraTreatment With ABT-199 (Venetoclax) and Purine Analogues in Relapsed/Refractory Acute Myeloid LeukemiaNCT05506332 · Phase 1 · 📍 EdegemVyxeos Plus Gilteritinib in Relapsed or Refractory, FLT3-Mutated AMLNCT05024552 · Phase 1 · 📍 TampaCD180 CART for Relapsed or Refractory CD180 Positive Hematologic MalignanciesNCT07106749 · Phase 1 · 📍 TianjinChemotherapy (Decitabine in Combination With FLAG-Ida) and Total-Body Irradiation Followed by Donor Stem Cell…NCT06928662 · Phase 1, Phase 2 · 📍 SeattleDonor Derived CD117 CAR-T Cells in the Treatment of R/R Acute Myeloid LeukemiaNCT07144020 · EARLY Phase 1 · 📍 HangzhouA Phase 1b Study of Lonitoclax + Azacitidine in Acute Myeloid Leukemia PatientsNCT07303660 · Phase 1 · 📍 MoscowFludarabine Phosphate, Cytarabine, Filgrastim-sndz, Gemtuzumab Ozogamicin, and Idarubicin Hydrochloride in Tr…NCT00801489 · Phase 2 · 📍 HoustonHVA vs IA/DA or VA in the Treatment of ND HR-AMLNCT06810791 · Phase 3 · 📍 GuangzhouIntestinal Microbiota Transplant Prior to Allogeneic Stem Cell Transplant (MAST) TrialNCT06355583 · Phase 2 · 📍 Birmingham Search more trials →
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