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Atlas / Disease / Leukemia

Leukemia

disease
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Also known as blood (leukemia)leukaemia (disease)leukaemia NOSleukemia (disease)leukemia, diseaseleukemia, malignantleukemiasleukemias, general

Summary

Leukemia (MONDO:0005059) is a cancer (an umbrella term covering 12 Mondo subtypes) with 3 cohort genes (15 GWAS associations across 9 studies; 3 CIViC-evidence somatic drivers) and 1,712 clinical trials. Molecularly, TP53 Wildtype confers sensitivity to RG7112 in Leukemia (CIViC Level B); 8 further subtype–drug associations are mapped below. Top therapeutic interventions include cyclophosphamide anhydrous, mercaptopurine anhydrous, and cytarabine.

At a glance

  • Classification: Cancer
  • Umbrella term: 12 Mondo subtypes
  • Cohort genes: 3
  • GWAS associations: 15
  • Clinical trials: 1,712
  • Precision-medicine evidence (CIViC): 9 subtype–drug associations

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical nameleukemia
Mondo IDMONDO:0005059
EFOEFO:0000565
MeSHD007938
DOIDDOID:1240
NCITC3161
SNOMED CT93143009
UMLSC0023418
MedGen9725
Is cancer (heuristic)yes

Also known as: blood (leukemia) · leukaemia (disease) · leukaemia NOS · leukemia · leukemia (disease) · leukemia, disease · leukemia, malignant · leukemias · leukemias, general

Data availability: 15 GWAS associations (9 studies) · 1 HPO phenotype · 27 cell lines.

Disease family

An umbrella term covering 12 Mondo subtypes.

Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › hematopoietic and lymphoid system neoplasm › hematopoietic and lymphoid cell neoplasm › leukemia

Related subtypes (7): central nervous system hematopoietic neoplasm, refractory hematologic cancer, lymphoid neoplasm, myeloid neoplasm, histiocytic and dendritic cell neoplasm, myeloid/lymphoid neoplasms associated with eosinophilia and abnormality of PDGFRA, PDGFRB, FGFR1 or JAK2, myelodysplastic syndrome with excess blasts

Subtypes (12): chronic leukemia, chronic erythremia, testicular leukemia, central nervous system leukemia, aleukemic leukemia, splenic manifestation of leukemia, childhood leukemia, monocytic leukemia, myeloid leukemia, lymphoid leukemia, acute leukemia, mast cell leukemia

Genetics & variants

GWAS landscape

15 GWAS associations across 9 studies. Top hits map to 8 distinct genes (as reported by GWAS).

Top associations by p-value

rsIDp-valueGeneRisk alleleOdds ratio
rs348555782e-22SP140C0.2
rs773754931e-20JAK2G1.51
rs7356651e-20GRAMD1BG0.19
chr2:2310912234e-20T0.21
rs8720712e-13IRF4A0.13
rs98112165e-13ACTRT3 - MYNNT0.14
rs1405519866e-12CHEK2G1.29
rs9190547e-12DRAICG0.12
chr2:1116430653e-11A0.12
rs1470016333e-11DNMT3AC1.64
rs5305807624e-08MYH16?

Top studies (by case count)

StudyLead authorYearCasesControlsTitle
GCST90475611Verma A20246,503443,163Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90435647Zhou W20181,661404,466Efficiently controlling for case-control imbalance and sample relatedness in large-scale genetic association studies.
GCST90477248Verma A20241,465119,986Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90479834Verma A20241,465119,986Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90651647Liu TY2025865235,192Diversity and longitudinal records: Genetic architecture of disease associations and polygenic risk in the Taiwanese Han population.
GCST90481542Verma A202446059,306Diversity and scale: Genetic architecture of 2068 traits in the VA Million Veteran Program.
GCST90041908Jiang L2021207456,069A generalized linear mixed model association tool for biobank-scale data.
GCST90042803Jiang L2021147456,201A generalized linear mixed model association tool for biobank-scale data.
GCST90246038Walters RG202311875,771Genotyping and population characteristics of the China Kadoorie Biobank.

Variant details and genetic-evidence tiers

Tier distribution (top 50 variants)

TierVariants
Tier 1: coding2
Tier 2: splice/UTR1
Tier 3: regulatory0
Tier 4: intronic/intergenic8

MAF distribution

BucketVariants
common (>=0.05)7
low_freq (0.01-0.05)0
rare (<0.01)3
unknown1

Functional consequences

ConsequenceCount
intron_variant4
missense_variant2
unknown2
3_prime_UTR_variant1
non_coding_transcript_exon_variant1
intergenic_variant1

Top variants

rsIDChrPosAllelesMAFConsequenceGenep-valueTier
rs348555782230250275C>G0.169intron_variantSP1402e-22Tier 4: intronic/intergenic
rs7737549395073770G>A,C,T0missense_variantJAK21e-20Tier 1: coding
rs73566511123490689G>A0.169intron_variantGRAMD1B1e-20Tier 4: intronic/intergenic
chr2:2310912230.1864e-20Tier 4: intronic/intergenic
rs8720716411064A>G,T0.493_prime_UTR_variantIRF42e-13Tier 2: splice/UTR
rs98112163169769713T>C,G0.281non_coding_transcript_exon_variantACTRT3 - MYNN5e-13Tier 4: intronic/intergenic
rs1405519862228711870G>A,C,T0.001intron_variantCHEK26e-12Tier 4: intronic/intergenic
rs9190541569698198G>A,C,T0.392intergenic_variantDRAIC7e-12Tier 4: intronic/intergenic
chr2:1116430650.3683e-11Tier 4: intronic/intergenic
rs147001633225234373C>A,G,T0missense_variantDNMT3A3e-11Tier 1: coding
rs530580762799279832C>A,G,Tintron_variantMYH164e-08Tier 4: intronic/intergenic

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 31 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

GeneintOGen roleCancer typesCIViC
SF3B1ActAML,BLCA,BRCA,CHOL,CLLSLL,HCC,LUNG,MBL,MEL,PAAD,PCM,PRAD,SKCM,UMCIViC #44
TP53LoFACC,ALL,AML,ANGS,ANSC,BCC,BL,BLADDER,BLCA,BRCA,CCRCC,CEAD,CESC,CHOL,CHRCC,CLLSLL,COAD,COADREAD,CSCC,DLBCLNOS,EGC,ES,ESCA,ESCC,GB,GBC,GBM,GIST,HCC,HGGNOS,HNSC,LGGNOS,LIPO,LMS,LNM,LUAD,LUSC,MBL,MEL,MLYM,MT,NBL,NETNOS,NHL,NPC,NSCLC,OS,OVT,PAAD,PANCREAS,PAST,PCM,PLMESO,PRAD,PRCC,PROSTATE,RCC,READ,SACA,SARCNOS,SCLC,SIC,SKCM,SKIN,SOFT_TISSUE,STAD,STOMACH,THYM,UCEC,UCS,UTUC,VULVA,WDTC,WTCIViC #45
FLT3ActALL,AMLCIViC #24

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
SF3B1Orphanet:39044Uveal melanoma
SF3B1Orphanet:75564Acquired idiopathic sideroblastic anemia
TP53Orphanet:1333Familial pancreatic carcinoma
TP53Orphanet:145Hereditary breast and/or ovarian cancer syndrome
TP53Orphanet:1501Adrenocortical carcinoma
TP53Orphanet:210159Adult hepatocellular carcinoma
TP53Orphanet:251576Gliosarcoma
TP53Orphanet:251579Giant cell glioblastoma
TP53Orphanet:251899Choroid plexus carcinoma
TP53Orphanet:2807Papilloma of choroid plexus
TP53Orphanet:293199Pleomorphic rhabdomyosarcoma
TP53Orphanet:3318Essential thrombocythemia
TP53Orphanet:524Li-Fraumeni syndrome
TP53Orphanet:52688Myelodysplastic syndrome
TP53Orphanet:585909B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)
TP53Orphanet:667662Breast implant-associated anaplastic large cell lymphoma
TP53Orphanet:668Osteosarcoma
TP53Orphanet:67038B-cell chronic lymphocytic leukemia
TP53Orphanet:70573Small cell lung cancer
TP53Orphanet:96253Cushing disease
TP53Orphanet:99756Alveolar rhabdomyosarcoma
TP53Orphanet:99757Embryonal rhabdomyosarcoma
FLT3Orphanet:102724Acute myeloid leukemia with t(8;21)(q22;q22) translocation
FLT3Orphanet:585909B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)
FLT3Orphanet:589534Mixed phenotype acute leukemia with t(9;22)(q34.1;q11.2)
FLT3Orphanet:589595Mixed phenotype acute leukemia with t(v;11q23.3)
FLT3Orphanet:98829Acute myeloid leukemia with abnormal bone marrow eosinophils inv(16)(p13q22) or t(16;16)(p13;q22)
FLT3Orphanet:98832Acute myeloid leukemia with minimal differentiation
FLT3Orphanet:98833Acute myeloblastic leukemia without maturation
FLT3Orphanet:98834Acute myeloblastic leukemia with maturation
FLT3Orphanet:99861Precursor T-cell acute lymphoblastic leukemia

Cohort genes → proteins

3 cohort genes, 3 distinct canonical proteins.

Evidence partition

SubsetGenes
civic_only3

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
SF3B1HGNC:10768ENSG00000115524O75533Splicing factor 3B subunit 1civic_evidence
TP53HGNC:11998ENSG00000141510P04637Cellular tumor antigen p53civic_evidence
FLT3HGNC:3765ENSG00000122025P36888Receptor-type tyrosine-protein kinase FLT3civic_evidence

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
SF3B1Splicing factor 3B subunit 1Component of the 17S U2 SnRNP complex of the spliceosome, a large ribonucleoprotein complex that removes introns from transcribed pre-mRNAs.
TP53Cellular tumor antigen p53Multifunctional transcription factor that induces cell cycle arrest, DNA repair or apoptosis upon binding to its target DNA sequence.
FLT3Receptor-type tyrosine-protein kinase FLT3Tyrosine-protein kinase that acts as a cell-surface receptor for the cytokine FLT3LG and regulates differentiation, proliferation and survival of hematopoietic progenitor cells and of dendritic cells.

Protein-family classification

Druggable: 1 · Difficult: 1 · Unknown: 1 · Druggable fraction: 0.33

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

FamilyGenesFoldFDR
Kinase19.2×0.313
Transcription factor12.8×0.482
Other/Unknown10.6×0.914

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
SF3B1Other/UnknownnoARM-like, SF3b_su1, ARM-type_fold
TP53Transcription factornop53_tumour_suppressor, p53-like_TF_DNA-bd_sf, p53_tetrameristn
FLT3Kinaseyes2.7.10.1Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Tyr_kinase_rcpt_3_CS

Expression context

Cohort genes with no expression data: 0.

3 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)3
unknown0

Top tissues across cohort

TissueCohort genes
ventricular zone2
epithelium of nasopharynx1
tibia1
ganglionic eminence1
tendon of biceps brachii1
cerebellar cortex1
cerebellar hemisphere1
male germ line stem cell (sensu Vertebrata) in testis1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
SF3B1295ubiquitousmarkertibia, ventricular zone, epithelium of nasopharynx
TP53223ubiquitousmarkerventricular zone, ganglionic eminence, tendon of biceps brachii
FLT3166broadmarkermale germ line stem cell (sensu Vertebrata) in testis, cerebellar hemisphere, cerebellar cortex

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
TP5322,736
SF3B14,582
FLT33,570

Structural data

PDB: 3 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
TP53P04637313
SF3B1O7553374
FLT3P3688811

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 85. Enrichment computed across 3 evidence-associated genes (3 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

PathwayCohort genesFoldFDRSample cohort genes
FLT3 mutants bind TKIs13806.7×0.001FLT3
KW2449-resistant FLT3 mutants13806.7×0.001FLT3
semaxanib-resistant FLT3 mutants13806.7×0.001FLT3
crenolanib-resistant FLT3 mutants13806.7×0.001FLT3
gilteritinib-resistant FLT3 mutants13806.7×0.001FLT3
lestaurtinib-resistant FLT3 mutants13806.7×0.001FLT3
midostaurin-resistant FLT3 mutants13806.7×0.001FLT3
pexidartinib-resistant FLT3 mutants13806.7×0.001FLT3
ponatinib-resistant FLT3 mutants13806.7×0.001FLT3
quizartinib-resistant FLT3 mutants13806.7×0.001FLT3
sorafenib-resistant FLT3 mutants13806.7×0.001FLT3
sunitinib-resistant FLT3 mutants13806.7×0.001FLT3
tandutinib-resistant FLT3 mutants13806.7×0.001FLT3
linifanib-resistant FLT3 mutants13806.7×0.001FLT3
tamatinib-resistant FLT3 mutants13806.7×0.001FLT3
Loss of function of TP53 in cancer due to loss of tetramerization ability13806.7×0.001TP53
Regulation of TP53 Expression11903.3×0.003TP53
Transcriptional activation of cell cycle inhibitor p211951.7×0.005TP53
Activation of NOXA and translocation to mitochondria1634.4×0.007TP53
RUNX3 regulates CDKN1A transcription1543.8×0.007TP53
STAT5 Activation1543.8×0.007FLT3
FLT3 signaling through SRC family kinases1543.8×0.007FLT3
FLT3 signaling by CBL mutants1543.8×0.007FLT3
PI5P Regulates TP53 Acetylation1423.0×0.008TP53
Activation of PUMA and translocation to mitochondria1380.7×0.009TP53
STAT5 activation downstream of FLT3 ITD mutants1380.7×0.009FLT3
TP53 Regulates Transcription of Caspase Activators and Caspases1317.2×0.010TP53
TP53 Regulates Transcription of Death Receptors and Ligands1317.2×0.010TP53
Urea cycle1292.8×0.010TP53
Regulation of TP53 Activity through Association with Co-factors1271.9×0.010TP53

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
negative regulation of helicase activity15617.3×0.005TP53
cellular response to actinomycin D15617.3×0.005TP53
regulation of intrinsic apoptotic signaling pathway by p53 class mediator15617.3×0.005TP53
negative regulation of G1 to G0 transition15617.3×0.005TP53
positive regulation of mitochondrial membrane permeability12808.7×0.005TP53
oligodendrocyte apoptotic process12808.7×0.005TP53
negative regulation of glucose catabolic process to lactate via pyruvate12808.7×0.005TP53
negative regulation of pentose-phosphate shunt12808.7×0.005TP53
leukocyte homeostasis11872.4×0.005FLT3
obsolete homolactic fermentation11872.4×0.005TP53
signal transduction by p53 class mediator11872.4×0.005TP53
negative regulation of miRNA processing11872.4×0.005TP53
intrinsic apoptotic signaling pathway in response to hypoxia11872.4×0.005TP53
regulation of fibroblast apoptotic process11872.4×0.005TP53
T cell proliferation involved in immune response11404.3×0.005TP53
pro-B cell differentiation11404.3×0.005FLT3
positive regulation of programmed necrotic cell death11404.3×0.005TP53
oxidative stress-induced premature senescence11404.3×0.005TP53
B cell lineage commitment11123.5×0.005TP53
T cell lineage commitment11123.5×0.005TP53
mRNA transcription11123.5×0.005TP53
positive regulation of RNA polymerase II transcription preinitiation complex assembly11123.5×0.005TP53
positive regulation of thymocyte apoptotic process11123.5×0.005TP53
cellular response to UV-C11123.5×0.005TP53
regulation of apoptotic process255.6×0.005TP53, FLT3
regulation of mitochondrial membrane permeability involved in apoptotic process1936.2×0.006TP53
myeloid progenitor cell differentiation1802.5×0.006FLT3
viral process1802.5×0.006TP53
mitochondrial DNA repair1802.5×0.006TP53
lymphocyte proliferation1802.5×0.006FLT3

Therapeutics

Drugs indicated for this disease

9 approved, 69 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

DrugDevelopment status
AllopurinolApproved (phase 4)
BusulfanApproved (phase 4)
Cortisone AcetateApproved (phase 4)
DexamethasoneApproved (phase 4)
HydrocortisoneApproved (phase 4)
Methylprednisolone AcetateApproved (phase 4)
PrednisoloneApproved (phase 4)
PrednisoneApproved (phase 4)
RasburicaseApproved (phase 4)
AldesleukinPhase 3 (in late-stage trials)
AlemtuzumabPhase 3 (in late-stage trials)
Amphotericin BPhase 3 (in late-stage trials)
AmsacrinePhase 3 (in late-stage trials)
Arsenic TrioxidePhase 3 (in late-stage trials)
AsparaginasePhase 3 (in late-stage trials)
Asparaginase Erwinia ChrysanthemiPhase 3 (in late-stage trials)
AzacitidinePhase 3 (in late-stage trials)
BlinatumomabPhase 3 (in late-stage trials)
BortezomibPhase 3 (in late-stage trials)
CalciumPhase 3 (in late-stage trials)
CarmustinePhase 3 (in late-stage trials)
ChlorambucilPhase 3 (in late-stage trials)
CladribinePhase 3 (in late-stage trials)
ClofarabinePhase 3 (in late-stage trials)
CyclosporinePhase 3 (in late-stage trials)
CytarabinePhase 3 (in late-stage trials)
DactinomycinPhase 3 (in late-stage trials)
Dasatinib AnhydrousPhase 3 (in late-stage trials)
DaunorubicinPhase 3 (in late-stage trials)
DecitabinePhase 3 (in late-stage trials)
Dexamethasone AcetatePhase 3 (in late-stage trials)
DexrazoxanePhase 3 (in late-stage trials)
DoxorubicinPhase 3 (in late-stage trials)
EculizumabPhase 3 (in late-stage trials)
EnasidenibPhase 3 (in late-stage trials)
Epoetin AlfaPhase 3 (in late-stage trials)
ErgocalciferolPhase 3 (in late-stage trials)
EtoposidePhase 3 (in late-stage trials)
FilgrastimPhase 3 (in late-stage trials)
FludarabinePhase 3 (in late-stage trials)
Fludarabine PhosphatePhase 3 (in late-stage trials)
Gemtuzumab OzogamicinPhase 3 (in late-stage trials)
HydroxyureaPhase 3 (in late-stage trials)
IdarubicinPhase 3 (in late-stage trials)
IfosfamidePhase 3 (in late-stage trials)
ImatinibPhase 3 (in late-stage trials)
Insulin AspartPhase 3 (in late-stage trials)
Insulin GlarginePhase 3 (in late-stage trials)
Interferon AlfaPhase 3 (in late-stage trials)
LaromustinePhase 3 (in late-stage trials)
LintuzumabPhase 3 (in late-stage trials)
MelphalanPhase 3 (in late-stage trials)
MethotrexatePhase 3 (in late-stage trials)
MethylprednisolonePhase 3 (in late-stage trials)
MidostaurinPhase 3 (in late-stage trials)
MitoxantronePhase 3 (in late-stage trials)
MorphinePhase 3 (in late-stage trials)
Mycophenolate MofetilPhase 3 (in late-stage trials)
Nalotimagene CarmaleucelPhase 3 (in late-stage trials)
NelarabinePhase 3 (in late-stage trials)
NilotinibPhase 3 (in late-stage trials)
NystatinPhase 3 (in late-stage trials)
Oblimersen SodiumPhase 3 (in late-stage trials)
OfatumumabPhase 3 (in late-stage trials)
Omacetaxine MepesuccinatePhase 3 (in late-stage trials)
PR1 LEUKEMIA PEPTIDE VACCINEPhase 3 (in late-stage trials)
PaliferminPhase 3 (in late-stage trials)
PegaspargasePhase 3 (in late-stage trials)
RituximabPhase 3 (in late-stage trials)
SargramostimPhase 3 (in late-stage trials)
ThioguaninePhase 3 (in late-stage trials)
ThiotepaPhase 3 (in late-stage trials)
TretinoinPhase 3 (in late-stage trials)
ValspodarPhase 3 (in late-stage trials)
VinblastinePhase 3 (in late-stage trials)
VincristinePhase 3 (in late-stage trials)
VindesinePhase 3 (in late-stage trials)
VoriconazolePhase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Acetaminophen, Aclarubicin, Aflibercept, Amifostine, Anidulafungin, Atorvastatin, Auranofin, Bendamustine, Bevacizumab, Bexarotene, Bosutinib, CPI 613, Carboplatin, Cisplatin, Daclizumab, Daratumumab, Darbepoetin Alfa, Denileukin Diftitox, Duvelisib, Eltrombopag, Epratuzumab, Erlotinib, Etanercept, Etoposide Phosphate, Forodesine, Fospropofol, Ginseng, American, Human Immunoglobulin G, Hydroxychloroquine, INDIUM IN 111 IBRITUMOMAB TIUXETAN, Ibrutinib, Idoxuridine, Imiquimod, Inotuzumab Ozogamicin, Irinotecan, Isotretinoin, Ixabepilone, Ixazomib, Lenalidomide, Lestaurtinib, Micafungin, Mycophenolic Acid, Nivolumab, Obinutuzumab, Oxiglutatione, PEGINTERFERON ALFA-2B, Paclitaxel, Pacritinib, Panobinostat, Pegfilgrastim, Pentostatin, Perifosine, Pirtobrutinib, Ponatinib, Propofol, QS-21, Revumenib, Ridaforolimus, Ruxolitinib, Sapacitabine, Sirolimus, Sonidegib, Sorafenib, Tacrolimus Anhydrous, Tanespimycin, Temozolomide, Temsirolimus, Teniposide, Thalidomide, Tisagenlecleucel, Tocilizumab, Topotecan, Trastuzumab, Valacyclovir, Valganciclovir, Valproic Acid, Venetoclax, Vorinostat, YTTRIUM Y 90 IBRITUMOMAB TIUXETAN.

Drug target analysis

Approved (phase 4): 2 · Phase ≥3: 2 · Phased (≥1): 3 · Undrugged: 0

Druggability breadth: 3 of 3 evidence-associated genes (100%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Genes with an approved drug

The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.

SymbolExample approved molecule
TP53NITROFURANTOIN
FLT3PONATINIB

Top cohort targets by molecule count

SymbolMoleculesMax phase
TP531964
FLT31434
SF3B112

Drugs targeting cohort genes (top 30)

MoleculeMax phaseTargets in cohort
NITROFURANTOIN4TP53
DIOSMIN4TP53
VERTEPORFIN4TP53
CANDESARTAN CILEXETIL4TP53
DIENESTROL4TP53
CLOTRIMAZOLE4TP53
COLCHICINE4TP53
NABUMETONE4TP53
SALMETEROL XINAFOATE4TP53
AMIODARONE HYDROCHLORIDE4TP53
FURAZOLIDONE4TP53
AMOXAPINE4TP53
RALOXIFENE HYDROCHLORIDE4TP53
NICARDIPINE HYDROCHLORIDE4TP53
SULCONAZOLE NITRATE4TP53
PYRITHIONE ZINC4TP53
LACTIC ACID4TP53
OXYMETHOLONE4TP53
CHLOROXINE4TP53
PROPIOLACTONE4TP53
CLOMIPRAMINE HYDROCHLORIDE4TP53
PHENYL AMINOSALICYLATE4TP53
THIORIDAZINE HYDROCHLORIDE4TP53
AMITRIPTYLINE HYDROCHLORIDE4TP53
ETHOPROPAZINE HYDROCHLORIDE4TP53
MECHLORETHAMINE HYDROCHLORIDE4TP53
ECONAZOLE NITRATE4TP53
TRIFLUPROMAZINE HYDROCHLORIDE4TP53
PROCHLORPERAZINE EDISYLATE4TP53
DEQUALINIUM CHLORIDE4TP53

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

SymbolAssaysType breakdown
FLT33,132Binding:3096, Functional:24, ADMET:8, Toxicity:4
TP53869Binding:775, ADMET:83, Functional:10, Toxicity:1
SF3B122Binding:22

Cohort enzymes (BRENDA EC)

SymbolEC numbersNames
FLT32.7.10.1receptor protein-tyrosine kinase

Cohort genes with high screening signal

≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.

SymbolChEMBL assays
TP53869
FLT33,132

Pharmacogenomics

Cohort genes with a PharmGKB record: 3; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Drug repurposing candidates

30 approved/phased drugs hit cohort targets but don’t yet appear in disease-level clinical trials. Target-inhibition rationale is strongest for cancer driver genes; a bioactivity hit is a screening signal, not a treatment claim.

CompoundMax phaseCohort target (bioactivity)
NITROFURANTOIN4TP53
DIOSMIN4TP53
VERTEPORFIN4TP53
CANDESARTAN CILEXETIL4TP53
DIENESTROL4TP53
CLOTRIMAZOLE4TP53
COLCHICINE4TP53
NABUMETONE4TP53
SALMETEROL XINAFOATE4TP53
AMIODARONE HYDROCHLORIDE4TP53
FURAZOLIDONE4TP53
AMOXAPINE4TP53
RALOXIFENE HYDROCHLORIDE4TP53
NICARDIPINE HYDROCHLORIDE4TP53
SULCONAZOLE NITRATE4TP53
PYRITHIONE ZINC4TP53
LACTIC ACID4TP53
OXYMETHOLONE4TP53
CHLOROXINE4TP53
PROPIOLACTONE4TP53
CLOMIPRAMINE HYDROCHLORIDE4TP53
PHENYL AMINOSALICYLATE4TP53
THIORIDAZINE HYDROCHLORIDE4TP53
AMITRIPTYLINE HYDROCHLORIDE4TP53
ETHOPROPAZINE HYDROCHLORIDE4TP53
MECHLORETHAMINE HYDROCHLORIDE4TP53
ECONAZOLE NITRATE4TP53
TRIFLUPROMAZINE HYDROCHLORIDE4TP53
PROCHLORPERAZINE EDISYLATE4TP53
DEQUALINIUM CHLORIDE4TP53

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

TierDefinitionGenesSymbols
AApproved (phase 4 drug)2TP53, FLT3
BPhased (≥1) drug, not yet approved1SF3B1
CDruggable family + PDB, no drug0
DDruggable family + AlphaFold only, no drug0
EDifficult family or no structure, no drug0

Undrugged target profiles

0 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Clinical trials & evidence

Clinical trials

Clinical trials: 1,712.

Phase distribution (across all retrieved trials)

PhaseTrials
PHASE2553
PHASE1/PHASE2184
PHASE1182
PHASE3142
PHASE2/PHASE320
PHASE415
EARLY_PHASE14

Top trials by phase / activity

NCTPhaseStatusTitle
NCT00003398PHASE4COMPLETEDBone Marrow Transplantation in Treating Patients With Hematologic Cancer
NCT00362544PHASE4COMPLETEDPROPHYSOME: Pilot Study on Safety of a Weekly Administration of 10mg/kg of AmBisome® in Antifungal Prophylaxis Treatment of Allogeneic Stem-cell Transplantation and Acute Leukaemia
NCT00365508PHASE4COMPLETEDCounseling and Nicotine Replacement Therapy in Helping Adult Smokers Quit Smoking
NCT00415103PHASE4COMPLETEDAMENO-2: Aprepitant Plus Palonosetron Versus Granisetron in the Prevention of Nausea and the Emesis Induced by Chemotherapy in Patients Treated With Haematopoietic Progenitors
NCT00666978PHASE4COMPLETEDHealth Education Counseling With or Without Bupropion in Helping African Americans Stop Smoking
NCT00808171PHASE4COMPLETEDEvaluation of the Analgesy With Emla and/or Nitrous Oxide in Pediatric Patients for Lumbar Puncture
NCT01191541PHASE4COMPLETEDCytarabine (Ara-C) in Children With Acute Promyelocytic Leukemia (APL)
NCT01222819PHASE4TERMINATEDSubCutaneous (SC) Versus Intravenous (IV) Granulocyte Colony Stimulating Factors (G-CSF) for the Treatment of Neutropenia in Hospitalized Haemato-oncological Patients
NCT01339988PHASE4UNKNOWNBusulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide for Hematological Malignancies Hematocrit (HCT)
NCT02095951PHASE4COMPLETEDPreemptive Ethanol Lock Therapy in Pediatric Bloodstream Infection
NCT02347878PHASE4UNKNOWNSelf-control Trial to Evaluate the Role of Aprepitant in the Prophylaxis of Post-lumbar-punture-headache (PLPH)
NCT02784561PHASE4UNKNOWNStudy of Busulfan and FLAG Conditioning Regimen for Allogeneic Peripheral Blood Stem Cell Transplantation
NCT03677596PHASE4COMPLETEDA Study Of Two Inotuzumab Ozogamicin Doses in Relapsed/ Refractory Acute Lymphoblastic Leukemia Transplant Eligible Patients
NCT04187755PHASE4COMPLETEDCefepime vs Ceftazidime as Empirical Therapy for Neutropenic Fever
NCT04636918PHASE4UNKNOWNIkervis for DED Due to GVHD Post Allo-HSCT
NCT03057054PHASE3ACTIVE_NOT_RECRUITINGLactobacillus Plantarum in Preventing Acute Graft Versus Host Disease in Children Undergoing Donor Stem Cell Transplant
NCT04547049PHASE3ACTIVE_NOT_RECRUITINGA Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
NCT06578247PHASE3RECRUITINGQuizartinib or Placebo Plus Chemotherapy in Newly Diagnosed Patients With FLT3-ITD Negative AML
NCT07082452PHASE3NOT_YET_RECRUITINGA Multicenter Trial Evaluating Efficacy and Safety of A Reduced Venetoclax Exposure To Seven Days Versus Standard Continuous Venetoclax Exposure Combined With Azacitidine in Treatment Naïve Subjects With Acute Myeloid Leukemia Who Are Ineligible for Intensive Induction
NCT07202052PHASE2/PHASE3RECRUITINGRole of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy Platform Trial (RATIONAL-PT)
NCT07202065PHASE2/PHASE3RECRUITINGRole of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy Dosing Immunoglobulin (Dose Ig)
NCT07202078PHASE2/PHASE3RECRUITINGRole of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy: Starting Immunoglobulin (Start Ig)
NCT07202091PHASE2/PHASE3RECRUITINGRole of Antibiotic Therapy or Immunoglobulin On iNfections in hAematoLogy Immunoglobulin Stopping or Extension (Stop Ig)
NCT00000591PHASE3COMPLETEDT-Cell Depletion in Unrelated Donor Marrow Transplantation
NCT00002456PHASE3COMPLETEDGraft-Versus-Host Disease Prevention in Treating Patients Who Are Undergoing Bone Marrow Transplantation
NCT00002499PHASE2/PHASE3UNKNOWNCombination Chemotherapy in Treating Children With Relapsed Acute Lymphocytic Leukemia
NCT00002514PHASE3COMPLETEDStem Cell Transplantation Compared With Standard Chemotherapy in Treating Patients With Acute Lymphoblastic Leukemia in First Remission
NCT00002517PHASE3COMPLETEDCombination Chemotherapy in Treating Children With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome
NCT00002534PHASE3COMPLETEDBone Marrow Transplantation in Treating Patients With Acute Leukemia in First or Second Remission
NCT00002549PHASE3UNKNOWNCombination Chemotherapy Followed by Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Acute Myelogenous Leukemia
NCT00002658PHASE3UNKNOWNCombination Chemotherapy, Biological Therapy, and Bone Marrow Transplantation in Treating Patients With Acute Myeloid Leukemia
NCT00002700PHASE3COMPLETEDChemotherapy With or Without Bone Marrow Transplantation in Treating Patients With Acute Lymphoblastic Leukemia
NCT00002701PHASE3UNKNOWNCombination Chemotherapy With or Without Bone Marrow Transplantation in Treating Patients With Acute Promyelocytic Leukemia
NCT00002719PHASE3COMPLETEDCombination Chemotherapy With or Without G-CSF in Treating Older Patients With Acute Myeloid Leukemia
NCT00002742PHASE3COMPLETEDAntifungal Therapy for Fever and Neutropenia in Patients Receiving Treatment for Hematologic Cancer
NCT00002744PHASE3COMPLETEDCombination Chemotherapy in Treating Children With Newly Diagnosed Acute Lymphoblastic Leukemia
NCT00002757PHASE3COMPLETEDTITLE:Less Intensive Therapy for Children With Non-Hodgkin’s Lymphoma
NCT00002766PHASE3COMPLETEDComparison of Two Combination Chemotherapy Regimens in Treating Adults With Previously Untreated Leukemia or Lymphoma
NCT00002771PHASE3UNKNOWNChemotherapy, Interferon, and Bone Marrow Transplantation in Treating Patients With Chronic Myelogenous Leukemia
NCT00002789PHASE3COMPLETEDBone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myeloid Leukemia

Drugs tested across these trials (top 30)

MoleculeMax phaseTrials referencing
CYCLOPHOSPHAMIDE ANHYDROUS4272
MERCAPTOPURINE ANHYDROUS4110
CYTARABINE444
IDARUBICIN440
MITOXANTRONE HYDROCHLORIDE438
IMATINIB437
BUSULFAN436
ASPARAGINASE435
DAUNORUBICIN HYDROCHLORIDE433
2-MERCAPTOETHANESULFONIC ACID430
THIOGUANINE429
ETOPOSIDE423
LEUCOVORIN423
SARGRAMOSTIM420
THIOTEPA419
VINCRISTINE SULFATE418
FILGRASTIM416
FLUDARABINE PHOSPHATE416
TRETINOIN411
AMSACRINE46
AMPHOTERICIN B42
APREPITANT42
BUPROPION HYDROCHLORIDE41
CEFEPIME41
CEFTAZIDIME41
CYCLOSPORINE41
GRANISETRON41
LIDOCAINE HYDROCHLORIDE41
NICOTINE41
PALONOSETRON41

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 9 predictive associations from 9 curated evidence items; also 1 oncogenic.

Molecular subtypeTherapyEffectLevelCIViC
TP53 WildtypeRG7112Sensitivity/ResponseCIViC BEID2965
TP53 Deleterious MutationRG7112ResistanceCIViC BEID2966
FLT3 D835YLestaurtinibSensitivity/ResponseCIViC DEID11095
FLT3 D835YSorafenibSensitivity/ResponseCIViC DEID11096
FLT3 ITDSunitinibSensitivity/ResponseCIViC DEID7374
FLT3 ITDSunitinib + SorafenibSensitivity/ResponseCIViC DEID7382
SF3B1 K700EEtoposide + OlaparibSensitivity/ResponseCIViC DEID10139
TOP2A TOP2A/90EtoposideResistanceCIViC DEID9634
TOP2A TOP2A/90Etoposide + Pixantrone + DVP Regimen + 4’-(9-acridinylamino)methanesulfon-m-anisidide + MVT RegimenResistanceCIViC DEID9635

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 73

This page pulls from 73 datasets indexed by BioBTree:

alphafoldantibodybgeebgee_evidencebindingdbbiogrid_interactionbrendabrenda_kineticscellosauruschembl_activitychembl_assaychembl_documentchembl_moleculechembl_targetciviccivic_evidenceclingen_dosageclinical_trialsclinvarctd_gene_interactiondbsnpdepmapdiamond_similaritydoidefoensemblentrezesm2_similarityfantom5_genefantom5_promotergenccgenerifgogtopdbgtopdb_interactiongwasgwas_studyhgncintactinterprointogenmedgenmeshmimmondomondochildmondoparentncitorphanetpatentpatent_compoundpdbpfampharmgkb_clinicalpharmgkb_genepharmgkb_guidelinepharmgkb_variantpubchempubchem_activitypubchem_assayreactomerefseqrhearnacentralsctidscxa_expressionscxa_gene_experimentsignorstring_interactiontranscriptufeatureumlsuniprot