OncoSignal

Acute myeloid leukemia

Menin inhibitors, FLT3 and IDH combinations, venetoclax backbones and what comes after.

Live from Open Targets Platform, fetched 2026-11-11 15:17 UTC. Every row links to its source record.
Targets linked to acute myeloid leukemia
12,979
the top 50 by overall evidence are shown
With inherited (germline) evidence
20
of the top 50, score above 0.1
Strong genetics, few drugs
20
genetic evidence 0.5 or more, drug evidence under 0.5
20 of the top 50 acute myeloid leukemia targets have strong human genetic evidence but little drug evidence so far

Each dot is one target. Higher means stronger genetic evidence in people (inherited variants or tumour mutations, whichever is stronger). Further right means more evidence from drugs already in trials or approved.

Has inherited (germline) evidence above 0.1Tumour (somatic) or other evidence only
000.250.250.50.50.750.7511Drug evidence (trials and approvals) →Human genetic evidence →Genetics strong, few drugs: open betsGenetics strong, drugs in the clinicDrugs in the clinic, weaker geneticsDNMT3A: genetic evidence 0.85 (germline 0.85, somatic 0.85), drug evidence 0.98CEBPA: genetic evidence 0.89 (germline 0.87, somatic 0.89), drug evidence 0.00FLT3: genetic evidence 0.87 (germline 0.55, somatic 0.87), drug evidence 0.98IDH1: genetic evidence 0.80 (germline 0.56, somatic 0.80), drug evidence 0.95TET2: genetic evidence 0.87 (germline 0.78, somatic 0.87), drug evidence 0.00SRSF2: genetic evidence 0.95 (germline 0.95, somatic 0.83), drug evidence 0.00RUNX1: genetic evidence 0.87 (germline 0.87, somatic 0.85), drug evidence 0.00KIT: genetic evidence 0.66 (germline 0.32, somatic 0.66), drug evidence 0.95ASXL1: genetic evidence 0.87 (germline 0.75, somatic 0.87), drug evidence 0.00GATA2: genetic evidence 0.83 (germline 0.83, somatic 0.82), drug evidence 0.00KRAS: genetic evidence 0.81 (germline 0.80, somatic 0.81), drug evidence 0.00NRAS: genetic evidence 0.87 (germline 0.00, somatic 0.87), drug evidence 0.00IDH2: genetic evidence 0.87 (germline 0.32, somatic 0.87), drug evidence 0.86TP53: genetic evidence 0.83 (germline 0.68, somatic 0.83), drug evidence 0.52JAK2: genetic evidence 0.79 (germline 0.74, somatic 0.79), drug evidence 0.68DDX41: genetic evidence 0.93 (germline 0.93, somatic 0.00), drug evidence 0.00TERT: genetic evidence 0.79 (germline 0.79, somatic 0.46), drug evidence 0.14NPM1: genetic evidence 0.86 (germline 0.02, somatic 0.86), drug evidence 0.12GATA1: genetic evidence 0.81 (germline 0.00, somatic 0.81), drug evidence 0.00PTPN11: genetic evidence 0.82 (germline 0.00, somatic 0.82), drug evidence 0.00RARA: genetic evidence 0.61 (germline 0.00, somatic 0.61), drug evidence 0.96PDGFRB: genetic evidence 0.57 (germline 0.00, somatic 0.57), drug evidence 0.95SETBP1: genetic evidence 0.69 (germline 0.69, somatic 0.65), drug evidence 0.00PDGFRA: genetic evidence 0.53 (germline 0.00, somatic 0.53), drug evidence 0.94NSD1: genetic evidence 0.83 (germline 0.83, somatic 0.36), drug evidence 0.00POLD1: genetic evidence 0.46 (germline 0.00, somatic 0.46), drug evidence 0.97POLE: genetic evidence 0.46 (germline 0.00, somatic 0.46), drug evidence 0.97WT1: genetic evidence 0.79 (germline 0.24, somatic 0.79), drug evidence 0.56FANCD2: genetic evidence 0.53 (germline 0.00, somatic 0.53), drug evidence 0.00KDR: genetic evidence 0.46 (germline 0.00, somatic 0.46), drug evidence 0.93U2AF1: genetic evidence 0.83 (germline 0.00, somatic 0.83), drug evidence 0.00ETV6: genetic evidence 0.68 (germline 0.19, somatic 0.68), drug evidence 0.00RET: genetic evidence 0.46 (germline 0.00, somatic 0.46), drug evidence 0.90DNMT1: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.98SMO: genetic evidence 0.46 (germline 0.00, somatic 0.46), drug evidence 0.89CD33: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.96TOP2A: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.98RARG: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.96PRKCB: genetic evidence 0.30 (germline 0.00, somatic 0.30), drug evidence 0.91PRKD1: genetic evidence 0.30 (germline 0.00, somatic 0.30), drug evidence 0.91RAD21: genetic evidence 0.72 (germline 0.00, somatic 0.72), drug evidence 0.00AXL: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.94EZH2: genetic evidence 0.77 (germline 0.00, somatic 0.77), drug evidence 0.06RARB: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.96TXNRD1: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.97SBDS: genetic evidence 0.46 (germline 0.00, somatic 0.46), drug evidence 0.00PRIM2: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.97POLA2: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.97POLD2: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.97POLD3: genetic evidence 0.00 (germline 0.00, somatic 0.00), drug evidence 0.97DNMT3ACEBPAIDH1SRSF2KITGATA2IDH2TP53JAK2TERTNPM1RARASETBP1PDGFRAPOLEWT1FANCD2KDRSMOPRKCBPRKD1EZH2SBDS

Scores run from 0 to 1 and are Open Targets' summary of how much evidence exists, not the chance a drug will work. The dividing lines sit at 0.5. Hover a dot for its numbers, click it for the target's page. Source: Open Targets Platform, fetched live.

Where the genetics points and the pipeline has not gone yet

The 20 targets in the top-left of the chart, strongest genetic evidence first. Click one for its drugs, tractability and other cancers.

All 50 targets, by type of evidence

Ranked by overall score for acute myeloid leukemia on the Open Targets Platform. Darker cells mean stronger evidence of that type.

Peds RMTLmarks the 17 of these targets on the FDA's Pediatric Relevant Molecular Target List: a drug against one of them can be required to be studied in children under the RACE for Children Act.

TargetOverallGermline geneticsSomaticgenetic literatureClinicalPathwaysLiteratureAnimal models
DNMT3A
DNA methyltransferase 3 alpha
0.870.850.85
CEBPA
CCAAT enhancer binding protein alpha
0.850.870.89
FLT3Peds RMTL
fms related receptor tyrosine kinase 3
0.850.550.87
IDH1Peds RMTL
isocitrate dehydrogenase (NADP(+)) 1
0.800.560.80
TET2Peds RMTL
tet methylcytosine dioxygenase 2
0.790.780.87
SRSF2
serine and arginine rich splicing factor 2
0.790.950.83
RUNX1
RUNX family transcription factor 1
0.780.870.85
KITPeds RMTL
KIT proto-oncogene, receptor tyrosine kinase
0.770.320.66
ASXL1
ASXL transcriptional regulator 1
0.770.750.87
GATA2
GATA binding protein 2
0.760.830.82
KRAS
KRas proto-oncogene, GTPase
0.750.800.81
NRAS
NRAS proto-oncogene, GTPase
0.75–0.87
IDH2Peds RMTL
isocitrate dehydrogenase (NADP(+)) 2
0.750.320.87
TP53Peds RMTL
tumor protein p53
0.740.680.83
JAK2Peds RMTL
Janus kinase 2
0.730.740.79
DDX41
DEAD-box helicase 41
0.720.93–
TERTPeds RMTL
telomerase reverse transcriptase
0.710.790.46
NPM1
nucleophosmin 1
0.700.020.86
GATA1
GATA binding protein 1
0.69–0.81
PTPN11Peds RMTL
protein tyrosine phosphatase non-receptor type 11
0.69–0.82
RARA
retinoic acid receptor alpha
0.69–0.61
PDGFRBPeds RMTL
platelet derived growth factor receptor beta
0.69–0.57
SETBP1
SET binding protein 1
0.670.690.65
PDGFRAPeds RMTL
platelet derived growth factor receptor alpha
0.66–0.53
NSD1
nuclear receptor binding SET domain protein 1
0.660.830.36
POLD1
DNA polymerase delta 1, catalytic subunit
0.66–0.46
POLE
DNA polymerase epsilon, catalytic subunit
0.66–0.46
WT1Peds RMTL
WT1 transcription factor
0.650.240.79
FANCD2
FA complementation group D2
0.64–0.53
KDR
kinase insert domain receptor
0.64–0.46
U2AF1
U2 small nuclear RNA auxiliary factor 1
0.64–0.83
ETV6
ETS variant transcription factor 6
0.640.190.68
RETPeds RMTL
ret proto-oncogene
0.63–0.46
DNMT1
DNA methyltransferase 1
0.62––
SMOPeds RMTL
smoothened, frizzled class receptor
0.62–0.46
CD33Peds RMTL
CD33 molecule
0.61––
TOP2A
DNA topoisomerase II alpha
0.61––
RARG
retinoic acid receptor gamma
0.61––
PRKCB
protein kinase C beta
0.60–0.30
PRKD1
protein kinase D1
0.60–0.30
RAD21
RAD21 cohesin complex component
0.60–0.72
AXLPeds RMTL
AXL receptor tyrosine kinase
0.60––
EZH2Peds RMTL
enhancer of zeste 2 polycomb repressive complex 2 subunit
0.60–0.77
RARB
retinoic acid receptor beta
0.60––
TXNRD1
thioredoxin reductase 1
0.60––
SBDS
SBDS ribosome maturation factor
0.60–0.46
PRIM2
DNA primase subunit 2
0.59––
POLA2
DNA polymerase alpha 2, accessory subunit
0.59––
POLD2
DNA polymerase delta 2, accessory subunit
0.59––
POLD3
DNA polymerase delta 3, accessory subunit
0.59––

33 biomarkers in Acute myeloid leukemia have validated or clinical evidence in CIViC

Accepted evidence from CIViC, the open (CC0) expert-curated knowledgebase, searched by the disease name “Acute Myeloid Leukemia”. Level A is validated (for example in guidelines or an approval), level B comes from clinical trials or studies. 272 accepted items exist; the first 100 were read. This is the published evidence, not treatment advice.