Pediatric lymphoma

Summary

Pediatric lymphoma (MONDO:0003659) is a cancer with 7 cohort genes (6 CIViC-evidence somatic drivers) and 2,187 clinical trials. Molecularly, ZMYM2::FGFR1 Fusion OR BCR::FGFR1 Fusion OR CEP43::FGFR1 Fusion OR TRIM24::FGFR1 Fusion OR FGFR1 Translocation confers sensitivity to Pemigatinib in Lymphoma (CIViC Level A); 6 further subtype–drug associations are mapped below. Top therapeutic interventions include cyclophosphamide anhydrous, procarbazine, and vincristine sulfate.

At a glance

• Classification: Cancer
• Cohort genes: 7
• Clinical trials: 2,187
• Precision-medicine evidence (CIViC): 7 subtype–drug associations

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	pediatric lymphoma
Mondo ID	MONDO:0003659
DOID	DOID:5823
NCIT	C5165
UMLS	C1332979
MedGen	232062
GARD	0023606
Is cancer (heuristic)	yes

Also known as: childhood lymphoma · lymphoma · pediatric lymphoma

Data availability: 1 GenCC gene-disease record · 50 cell lines.

Disease family

An umbrella term covering 3 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 › lymphoid neoplasm › lymphoma › pediatric lymphoma

Related subtypes (26): prostate lymphoma, nasal cavity lymphoma, bladder lymphoma, tracheal lymphoma, retroperitoneal lymphoma, ureteral lymphoma, ovarian lymphoma, B-cell lymphoma, unclassifiable, with features intermediate between diffuse large b-cell lymphoma and classical Hodgkin lymphoma, adult lymphoma, breast lymphoma, heart lymphoma, chest wall lymphoma, lung lymphoma, mediastinal malignant lymphoma, eye lymphoma, B-cell neoplasm, gastrointestinal lymphoma, Hodgkins lymphoma, peripheral T-cell lymphoma, not otherwise specified, composite lymphoma, AIDS-related primary central nervous system lymphoma, primary organ-specific lymphoma, non-Hodgkin lymphoma, extranodal nasal NK/T cell lymphoma, methotrexate-associated lymphoproliferative disorders, progressive transformation of germinal centers

Subtypes (3): childhood acute lymphoblastic leukemia, T-cell childhood lymphoblastic lymphoma, systemic Epstein-Barr virus-positive T-cell lymphoproliferative disease of childhood

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 1 · Orphanet: 59 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
TP53	LoF	ACC,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,WT	CIViC #45
CCND3	Act	BL,DLBCLNOS,MLYM,NHL	CIViC #10
FGFR1	Act	BLCA,GBM,OVT,PANCREAS,PAST,PGNG,WDTC	CIViC #1885
KRAS	Act	ALL,AML,ANSC,BLADDER,BLCA,BRCA,CEAD,CESC,CHOL,CLLSLL,COAD,COADREAD,DLBCLNOS,EGC,ESCA,ESCC,HCC,LUAD,LUSC,MEL,MGCT,MT,NSCLC,OVT,PAAD,PANCREAS,PAST,PCM,PRAD,PRCC,READ,STAD,STOMACH,UCEC,UCS,WDTC	CIViC #30
ATM	LoF	BLCA,BRCA,CCRCC,CHOL,CLLSLL,COAD,COADREAD,ESCA,HCC,LUAD,LUSC,MEL,NSCLC,PAAD,PANCREAS,PANET,PCM,PLMESO,PRAD,PROSTATE,STAD,UCEC,UTUC,WDTC	CIViC #69
BCL2	Act	DLBCLNOS,MLYM,NHL	CIViC #59

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

Gene	Classification	Inheritance	Disease	Records
NR1I2	Limited	Autosomal recessive	pediatric lymphoma

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
TP53	Orphanet:1333	Familial pancreatic carcinoma
TP53	Orphanet:145	Hereditary breast and/or ovarian cancer syndrome
TP53	Orphanet:1501	Adrenocortical carcinoma
TP53	Orphanet:210159	Adult hepatocellular carcinoma
TP53	Orphanet:251576	Gliosarcoma
TP53	Orphanet:251579	Giant cell glioblastoma
TP53	Orphanet:251899	Choroid plexus carcinoma
TP53	Orphanet:2807	Papilloma of choroid plexus
TP53	Orphanet:293199	Pleomorphic rhabdomyosarcoma
TP53	Orphanet:3318	Essential thrombocythemia
TP53	Orphanet:524	Li-Fraumeni syndrome
TP53	Orphanet:52688	Myelodysplastic syndrome
TP53	Orphanet:585909	B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)
TP53	Orphanet:667662	Breast implant-associated anaplastic large cell lymphoma
TP53	Orphanet:668	Osteosarcoma
TP53	Orphanet:67038	B-cell chronic lymphocytic leukemia
TP53	Orphanet:70573	Small cell lung cancer
TP53	Orphanet:96253	Cushing disease
TP53	Orphanet:99756	Alveolar rhabdomyosarcoma
TP53	Orphanet:99757	Embryonal rhabdomyosarcoma
FGFR1	Orphanet:168953	Myeloid/lymphoid neoplasm associated with FGFR1 rearrangement
FGFR1	Orphanet:2117	Hartsfield syndrome
FGFR1	Orphanet:220386	Semilobar holoprosencephaly
FGFR1	Orphanet:2396	Encephalocraniocutaneous lipomatosis
FGFR1	Orphanet:251576	Gliosarcoma
FGFR1	Orphanet:251579	Giant cell glioblastoma
FGFR1	Orphanet:251615	Pilomyxoid astrocytoma
FGFR1	Orphanet:2645	Osteoglosphonic dysplasia
FGFR1	Orphanet:280200	Microform holoprosencephaly
FGFR1	Orphanet:314950	Primary hypereosinophilic syndrome
FGFR1	Orphanet:3157	Septo-optic dysplasia spectrum
FGFR1	Orphanet:3366	Non-syndromic metopic craniosynostosis
FGFR1	Orphanet:432	Normosmic congenital hypogonadotropic hypogonadism
FGFR1	Orphanet:478	Kallmann syndrome
FGFR1	Orphanet:93258	Pfeiffer syndrome type 1
FGFR1	Orphanet:93924	Lobar holoprosencephaly
FGFR1	Orphanet:99798	Oligodontia
KRAS	Orphanet:1333	Familial pancreatic carcinoma
KRAS	Orphanet:1340	Cardiofaciocutaneous syndrome
KRAS	Orphanet:144	Lynch syndrome
KRAS	Orphanet:146	Differentiated thyroid carcinoma
KRAS	Orphanet:2396	Encephalocraniocutaneous lipomatosis
KRAS	Orphanet:251615	Pilomyxoid astrocytoma
KRAS	Orphanet:2612	Linear nevus sebaceus syndrome
KRAS	Orphanet:268114	RAS-associated autoimmune leukoproliferative disease
KRAS	Orphanet:3339	Oculoectodermal syndrome
KRAS	Orphanet:648	Noonan syndrome
KRAS	Orphanet:86834	Juvenile myelomonocytic leukemia
ATM	Orphanet:100	Ataxia-telangiectasia
ATM	Orphanet:1331	Familial prostate cancer

Cohort genes → proteins

7 cohort genes, 7 distinct canonical proteins.

Evidence partition

Subset	Genes
civic_only	6
multi_evidence	1

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
TP53	HGNC:11998	ENSG00000141510	P04637	Cellular tumor antigen p53	civic_evidence
CCND3	HGNC:1585	ENSG00000112576	P30281	G1/S-specific cyclin-D3	civic_evidence
FGFR1	HGNC:3688	ENSG00000077782	P11362	Fibroblast growth factor receptor 1	civic_evidence
KRAS	HGNC:6407	ENSG00000133703	P01116	GTPase KRas	civic_evidence
ATM	HGNC:795	ENSG00000149311	Q13315	Serine-protein kinase ATM	civic_evidence
NR1I2	HGNC:7968	ENSG00000144852	O75469	Nuclear receptor subfamily 1 group I member 2	gencc
BCL2	HGNC:990	ENSG00000171791	P10415	Apoptosis regulator Bcl-2	civic_evidence

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
TP53	Cellular tumor antigen p53	Multifunctional transcription factor that induces cell cycle arrest, DNA repair or apoptosis upon binding to its target DNA sequence.
CCND3	G1/S-specific cyclin-D3	Regulatory component of the cyclin D3-CDK4 (DC) complex that phosphorylates and inhibits members of the retinoblastoma (RB) protein family including RB1 and regulates the cell-cycle during G(1)/S transition.
FGFR1	Fibroblast growth factor receptor 1	Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth factors and plays an essential role in the regulation of embryonic development, cell proliferation, differentiation and migration.
KRAS	GTPase KRas	Ras proteins bind GDP/GTP and possess intrinsic GTPase activity.
ATM	Serine-protein kinase ATM	Serine/threonine protein kinase which activates checkpoint signaling upon double strand breaks (DSBs), apoptosis and genotoxic stresses such as ionizing ultraviolet A light (UVA), thereby acting as a DNA damage sensor.
NR1I2	Nuclear receptor subfamily 1 group I member 2	Nuclear receptor that acts as a transcription factor regulating genes involved in the metabolism and excretion of xenobiotics, drugs, and endogenous compounds.
BCL2	Apoptosis regulator Bcl-2	Suppresses apoptosis in a variety of cell systems including factor-dependent lymphohematopoietic and neural cells.

Protein-family classification

Druggable: 4 · Difficult: 1 · Unknown: 2 · Druggable fraction: 0.57

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.

Family	Genes	Fold	FDR
Nuclear receptor	1	55.1×	0.060
Kinase	2	7.9×	0.060
Enzyme (other)	1	1.7×	0.744
Transcription factor	1	1.2×	0.744
Other/Unknown	2	0.5×	0.968

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
TP53	Transcription factor	no		p53_tumour_suppressor, p53-like_TF_DNA-bd_sf, p53_tetrameristn
CCND3	Other/Unknown	no		Cyclin_C-dom, Cyclin_N, Cyclin-like_dom
FGFR1	Kinase	yes	2.7.10.1	Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Ig_sub2
KRAS	Enzyme (other)	yes	3.6.5.2	Small_GTPase, Small_GTP-bd, Small_GTPase_Ras-type
ATM	Kinase	yes	2.7.11.1	PI3/4_kinase_cat_dom, PIK-rel_kinase_FAT, FATC_dom
NR1I2	Nuclear receptor	yes		Nucl_hrmn_rcpt_lig-bd, Znf_hrmn_rcpt, Nuclear_hrmn_rcpt
BCL2	Other/Unknown	no		Bcl2-like, Bcl2_BH4, Bcl2/BclX

Expression context

Cohort genes with no expression data: 0.

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

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	7
unknown	0

Top tissues across cohort

Tissue	Cohort genes
calcaneal tendon	3
ganglionic eminence	1
tendon of biceps brachii	1
ventricular zone	1
blood	1
granulocyte	1
thymus	1
buccal mucosa cell	1
stromal cell of endometrium	1
nipple	1
pylorus	1
trigeminal ganglion	1
colonic epithelium	1
corpus callosum	1
jejunal mucosa	1
mucosa of transverse colon	1
right lobe of liver	1
dorsal motor nucleus of vagus nerve	1
superficial temporal artery	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
TP53	223	ubiquitous	marker	ventricular zone, ganglionic eminence, tendon of biceps brachii
CCND3	287	ubiquitous	marker	granulocyte, thymus, blood
FGFR1	292	ubiquitous	marker	buccal mucosa cell, stromal cell of endometrium, calcaneal tendon
KRAS	298	ubiquitous	marker	trigeminal ganglion, pylorus, nipple
ATM	286	ubiquitous	marker	calcaneal tendon, colonic epithelium, corpus callosum
NR1I2	107	tissue_specific	marker	right lobe of liver, jejunal mucosa, mucosa of transverse colon
BCL2	275	ubiquitous	marker	dorsal motor nucleus of vagus nerve, superficial temporal artery, calcaneal tendon

Protein interactions among cohort

Intra-cohort edges: 4.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
TP53	22,736
KRAS	14,509
BCL2	8,343
ATM	7,383
FGFR1	5,693
CCND3	2,692
NR1I2	2,445

Intra-cohort edges

A	B	Sources
ATM	TP53	biogrid_interaction, string_interaction
BCL2	TP53	intact, string_interaction
KRAS	TP53	string_interaction
NR1I2	TP53	biogrid_interaction

Structural data

PDB: 7 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
KRAS	P01116	511
TP53	P04637	313
FGFR1	P11362	83
NR1I2	O75469	80
BCL2	P10415	55
ATM	Q13315	14
CCND3	P30281	2

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 227. Enrichment computed across 7 evidence-associated genes (7 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 7 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
TP53 Regulates Transcription of Caspase Activators and Caspases	2	271.9×	0.004	TP53, ATM
Stabilization of p53	2	217.5×	0.004	TP53, ATM
TP53 Regulates Transcription of Genes Involved in Cytochrome C Release	2	155.4×	0.004	TP53, ATM
Regulation of TP53 Activity through Methylation	2	155.4×	0.004	TP53, ATM
SHC-mediated cascade:FGFR1	2	141.9×	0.004	FGFR1, KRAS
FRS-mediated FGFR1 signaling	2	130.5×	0.004	FGFR1, KRAS
Signaling by FGFR1 in disease	2	83.7×	0.007	FGFR1, KRAS
Regulation of TP53 Degradation	2	83.7×	0.007	TP53, ATM
NCAM signaling for neurite out-growth	2	77.7×	0.007	FGFR1, KRAS
Autodegradation of the E3 ubiquitin ligase COP1	2	75.9×	0.007	TP53, ATM
Loss of function of TP53 in cancer due to loss of tetramerization ability	1	1631.4×	0.012	TP53
TP53 Regulates Transcription of DNA Repair Genes	2	51.8×	0.012	TP53, ATM
DNA Damage/Telomere Stress Induced Senescence	2	46.6×	0.013	TP53, ATM
Recruitment and ATM-mediated phosphorylation of repair and signaling proteins at DNA double strand breaks	2	41.8×	0.015	TP53, ATM
Signaling by FGFR1 amplification mutants	1	815.7×	0.016	FGFR1
Regulation of TP53 Expression	1	815.7×	0.016	TP53
G2/M Checkpoints	2	38.4×	0.016	TP53, ATM
G2/M DNA damage checkpoint	2	34.4×	0.017	TP53, ATM
Regulation of TP53 Activity through Phosphorylation	2	33.6×	0.017	TP53, ATM
The NLRP1 inflammasome	1	543.8×	0.019	BCL2
Signaling by RAS GAP mutants	1	543.8×	0.019	KRAS
Signaling by RAS GTPase mutants	1	543.8×	0.019	KRAS
Interleukin-4 and Interleukin-13 signaling	2	29.4×	0.019	TP53, BCL2
FGFR1c and Klotho ligand binding and activation	1	407.9×	0.021	FGFR1
Transcriptional activation of cell cycle inhibitor p21	1	407.9×	0.021	TP53
Signaling by plasma membrane FGFR1 fusions	1	407.9×	0.021	FGFR1
Activation of RAS in B cells	1	326.3×	0.025	KRAS
Drug-mediated inhibition of CDK4/CDK6 activity	1	326.3×	0.025	CCND3
Activation of NOXA and translocation to mitochondria	1	271.9×	0.028	TP53
Sensing of DNA Double Strand Breaks	1	271.9×	0.028	ATM

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 7 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
neuron apoptotic process	4	105.8×	1e-05	TP53, KRAS, ATM, BCL2
B cell lineage commitment	2	963.0×	3e-04	TP53, BCL2
positive regulation of cellular senescence	2	370.4×	0.001	TP53, KRAS
replicative senescence	2	283.2×	0.001	TP53, ATM
oocyte development	2	267.5×	0.001	ATM, BCL2
glial cell proliferation	2	253.4×	0.001	TP53, KRAS
positive regulation of gene expression	4	22.1×	0.001	TP53, KRAS, ATM, NR1I2
hematopoietic stem cell differentiation	2	218.9×	0.002	TP53, BCL2
release of cytochrome c from mitochondria	2	200.6×	0.002	TP53, BCL2
cellular response to gamma radiation	2	172.0×	0.002	TP53, ATM
response to gamma radiation	2	166.0×	0.002	TP53, BCL2
cardiac muscle cell proliferation	2	166.0×	0.002	FGFR1, KRAS
gene expression	3	34.2×	0.002	FGFR1, KRAS, NR1I2
intrinsic apoptotic signaling pathway in response to endoplasmic reticulum stress	2	137.6×	0.002	TP53, BCL2
reactive oxygen species metabolic process	2	133.8×	0.002	TP53, BCL2
homeostasis of number of cells within a tissue	2	126.7×	0.002	KRAS, BCL2
determination of adult lifespan	2	123.5×	0.002	TP53, ATM
T cell differentiation in thymus	2	117.4×	0.002	TP53, BCL2
ovarian follicle development	2	112.0×	0.003	ATM, BCL2
somitogenesis	2	107.0×	0.003	TP53, ATM
DNA damage response, signal transduction by p53 class mediator	2	102.4×	0.003	TP53, ATM
cellular response to glucose starvation	2	96.3×	0.003	TP53, BCL2
thymus development	2	96.3×	0.003	ATM, BCL2
intrinsic apoptotic signaling pathway in response to DNA damage	2	92.6×	0.003	ATM, BCL2
response to glucocorticoid	2	92.6×	0.003	KRAS, BCL2
positive regulation of apoptotic process	3	24.3×	0.003	TP53, ATM, BCL2
stem cell proliferation	2	89.2×	0.003	TP53, FGFR1
DNA damage response	3	22.9×	0.003	TP53, ATM, BCL2
cellular senescence	2	84.5×	0.003	TP53, ATM
positive regulation of neuron apoptotic process	2	77.7×	0.003	TP53, ATM

Therapeutics

Drug target analysis

Approved (phase 4): 7 · Phase ≥3: 7 · Phased (≥1): 7 · Undrugged: 0

Druggability breadth: 7 of 7 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.

Symbol	Example approved molecule
TP53	NITROFURANTOIN
CCND3	PALBOCICLIB
FGFR1	PONATINIB
KRAS	VEMURAFENIB
ATM	AMIODARONE HYDROCHLORIDE
NR1I2	BEPRIDIL
BCL2	IXABEPILONE

Top cohort targets by molecule count

Symbol	Molecules	Max phase
NR1I2	257	4
TP53	196	4
FGFR1	93	4
ATM	35	4
CCND3	17	4
BCL2	14	4
KRAS	11	4

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
NITROFURANTOIN	4	TP53
DIOSMIN	4	TP53
VERTEPORFIN	4	TP53
CANDESARTAN CILEXETIL	4	NR1I2, TP53
DIENESTROL	4	TP53
CLOTRIMAZOLE	4	NR1I2, TP53
COLCHICINE	4	TP53
NABUMETONE	4	TP53
SALMETEROL XINAFOATE	4	TP53
AMIODARONE HYDROCHLORIDE	4	ATM, TP53
FURAZOLIDONE	4	ATM, TP53
AMOXAPINE	4	TP53
RALOXIFENE HYDROCHLORIDE	4	TP53
NICARDIPINE HYDROCHLORIDE	4	TP53
SULCONAZOLE NITRATE	4	TP53
PYRITHIONE ZINC	4	TP53
LACTIC ACID	4	TP53
OXYMETHOLONE	4	TP53
CHLOROXINE	4	NR1I2, TP53
PROPIOLACTONE	4	TP53
CLOMIPRAMINE HYDROCHLORIDE	4	TP53
PHENYL AMINOSALICYLATE	4	TP53
THIORIDAZINE HYDROCHLORIDE	4	TP53
AMITRIPTYLINE HYDROCHLORIDE	4	ATM, TP53
ETHOPROPAZINE HYDROCHLORIDE	4	TP53
MECHLORETHAMINE HYDROCHLORIDE	4	TP53
ECONAZOLE NITRATE	4	TP53
TRIFLUPROMAZINE HYDROCHLORIDE	4	TP53
PROCHLORPERAZINE EDISYLATE	4	TP53
DEQUALINIUM CHLORIDE	4	TP53

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 3.

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

Symbol	Assays	Type breakdown
FGFR1	1,465	Binding:1428, Functional:24, ADMET:13
TP53	869	Binding:775, ADMET:83, Functional:10, Toxicity:1
KRAS	861	Binding:829, Functional:32
NR1I2	737	Binding:477, ADMET:201, Functional:30, Toxicity:29
BCL2	446	Binding:418, Functional:23, Toxicity:3, ADMET:2
CCND3	425	Binding:422, ADMET:2, Toxicity:1
ATM	240	Binding:233, Functional:5, ADMET:2

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
FGFR1	2.7.10.1	receptor protein-tyrosine kinase
KRAS	3.6.5.2	small monomeric GTPase
ATM	2.7.11.1	non-specific serine/threonine protein kinase

Cohort genes with high screening signal

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

Symbol	ChEMBL assays
TP53	869
CCND3	425
FGFR1	1,465
KRAS	861
ATM	240
NR1I2	737
BCL2	446

Pharmacogenomics

Cohort genes with a PharmGKB record: 7; with CPIC/DPWG dosing guidelines: 0.

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

Drug repurposing candidates

29 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.

Compound	Max phase	Cohort target (bioactivity)
NITROFURANTOIN	4	TP53
DIOSMIN	4	TP53
VERTEPORFIN	4	TP53
CANDESARTAN CILEXETIL	4	NR1I2, TP53
DIENESTROL	4	TP53
CLOTRIMAZOLE	4	NR1I2, TP53
COLCHICINE	4	TP53
NABUMETONE	4	TP53
SALMETEROL XINAFOATE	4	TP53
AMIODARONE HYDROCHLORIDE	4	ATM, TP53
FURAZOLIDONE	4	ATM, TP53
AMOXAPINE	4	TP53
RALOXIFENE HYDROCHLORIDE	4	TP53
NICARDIPINE HYDROCHLORIDE	4	TP53
SULCONAZOLE NITRATE	4	TP53
PYRITHIONE ZINC	4	TP53
LACTIC ACID	4	TP53
OXYMETHOLONE	4	TP53
CHLOROXINE	4	NR1I2, TP53
PROPIOLACTONE	4	TP53
CLOMIPRAMINE HYDROCHLORIDE	4	TP53
PHENYL AMINOSALICYLATE	4	TP53
THIORIDAZINE HYDROCHLORIDE	4	TP53
AMITRIPTYLINE HYDROCHLORIDE	4	ATM, TP53
ETHOPROPAZINE HYDROCHLORIDE	4	TP53
ECONAZOLE NITRATE	4	TP53
TRIFLUPROMAZINE HYDROCHLORIDE	4	TP53
PROCHLORPERAZINE EDISYLATE	4	TP53
DEQUALINIUM CHLORIDE	4	TP53

Druggability pyramid

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

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	7	TP53, CCND3, FGFR1, KRAS, ATM, NR1I2, BCL2
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	0

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: 2,187.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	678
PHASE1/PHASE2	210
PHASE3	156
PHASE4	22
PHASE1	20
PHASE2/PHASE3	14

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05170399	PHASE4	RECRUITING	Vaccine Responses in Patients With B Cell Malignancies
NCT06049134	PHASE4	ACTIVE_NOT_RECRUITING	Immunogenicity and Clinical Efficacy of 20-valent Pneumococcal Conjugate Vaccine (PCV20) in Lymphoma Survivors After Treatment With Anti-CD20 Therapy
NCT00003398	PHASE4	COMPLETED	Bone Marrow Transplantation in Treating Patients With Hematologic Cancer
NCT00158041	PHASE4	COMPLETED	Subcutaneous Amifostine Safety Study
NCT00361140	PHASE4	COMPLETED	Busulfan Safety/Efficacy as Conditioning Prior to Hematopoietic Cell Transplantation (HCT)
NCT00415103	PHASE4	COMPLETED	AMENO-2: Aprepitant Plus Palonosetron Versus Granisetron in the Prevention of Nausea and the Emesis Induced by Chemotherapy in Patients Treated With Haematopoietic Progenitors
NCT00854581	PHASE4	TERMINATED	Zidovudine, Interferon Alfa-2b, PEG-Interferon Alfa-2b in Patients With HTLV-I Associated Adult T-Cell Leukemia/Lymphoma
NCT01088750	PHASE4	COMPLETED	Surgery Alone or With CYC VBL and PRED or CVP Alone in Stage IA or IIA Nodular Lymphocyte-Predominant Hodgkin Lymphoma
NCT01658280	PHASE4	COMPLETED	Conventional Versus Ultrasound-guided Transbronchial Needle Aspiration for the Diagnosis of Hilar/Mediastinal Lymphadenopathies
NCT01841814	PHASE4	WITHDRAWN	Evolution of Bone Mineral Density (BMD) in Patients With Lymphoma Undergoing Chemotherapy
NCT01909934	PHASE4	COMPLETED	Study of Brentuximab Vedotin in Participants With Relapsed or Refractory Systemic Anaplastic Large Cell Lymphoma
NCT02095951	PHASE4	COMPLETED	Preemptive Ethanol Lock Therapy in Pediatric Bloodstream Infection
NCT02347878	PHASE4	UNKNOWN	Self-control Trial to Evaluate the Role of Aprepitant in the Prophylaxis of Post-lumbar-punture-headache (PLPH)
NCT02805218	PHASE4	COMPLETED	PEG-rhG-CSF in Patients With Lymphoma Receiving Chemotherapy
NCT02905916	PHASE4	UNKNOWN	The Efficacy and Safety of PEG-rhG-CSF in Neutropenia After Chemotherapy
NCT02905942	PHASE4	UNKNOWN	PEG-rhG-CSF in Lymphoma Patients After Autologous Hematopoietic Stem Cell Transplantation
NCT02929615	PHASE4	UNKNOWN	Study of Standard and Individualized Treatment Model for Relapse and Refractory Lymphatic System Malignant Tumors
NCT02933333	PHASE4	UNKNOWN	G-CSF Alone or Combination With GM-CSF on Prevention and Treatment of Infection in Children With Malignant Tumor
NCT03010579	PHASE4	UNKNOWN	Erythropoietin in the Treatment of Anemia After Autologous Hematopoietic Stem Cell Transplantation
NCT04349306	PHASE4	COMPLETED	Evaluate the Efficacy and Safety of Rasburicase (Fasturtec®) in the Prevention and Treatment of Hyperuricemia in Pediatric Patients With Non-Hodgkin’s Lymphoma and Acute Leukemia
NCT05510544	PHASE4	UNKNOWN	Plerixafor for Poorly Mobilized Lymphoma
NCT06026995	PHASE4	UNKNOWN	Clinical Study on PEG-rhG-CSF in Mobilizing Autologous Hematopoietic Stem Cells
NCT00002462	PHASE3	ACTIVE_NOT_RECRUITING	RT or No RT Following Chemotherapy in Treating Patients With Stage III/IV Hodgkin’s Disease
NCT00005584	PHASE3	ACTIVE_NOT_RECRUITING	Combination Chemotherapy With or Without Radiation Therapy in Treating Patients With Hodgkin’s Lymphoma
NCT00379041	PHASE3	ACTIVE_NOT_RECRUITING	Radiation Therapy With or Without Combination Chemotherapy in Treating Patients With Previously Untreated Stage I or Stage II Hodgkin’s Lymphoma
NCT00943423	PHASE3	ACTIVE_NOT_RECRUITING	PET Scan in Planning Treatment in Patients Undergoing Combination Chemotherapy For Stage IA or Stage IIA Hodgkin Lymphoma
NCT03057054	PHASE3	ACTIVE_NOT_RECRUITING	Lactobacillus Plantarum in Preventing Acute Graft Versus Host Disease in Children Undergoing Donor Stem Cell Transplant
NCT03480360	PHASE3	ACTIVE_NOT_RECRUITING	Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators’ Expression
NCT03647072	PHASE3	RECRUITING	PPI Versus Histamine Antagnists as Adjuvant to Chemotherapy
NCT03823534	PHASE3	RECRUITING	Post-Op Pain Control for Prophylactic Intramedullary Nailing.
NCT05006482	PHASE3	RECRUITING	Geriatric Evaluation and Management With Survivorship Health Education for Older Survivors of Cancer, GEM-S Trial
NCT05328258	PHASE3	RECRUITING	Use of GnRHa During Chemotherapy for Fertility Protection
NCT05770037	PHASE2/PHASE3	RECRUITING	DETERMINE Trial Treatment Arm 01: Alectinib in Adult, Paediatric and Teenage/Young Adult Patients With ALK Positive Cancers
NCT05775705	PHASE3	NOT_YET_RECRUITING	L-DEP Regimen Combined With PD-1 Antibody as Induction Therapy for Epstein-Barr Virus-positive LA-HLH
NCT06084936	PHASE3	RECRUITING	A Study to Evaluate Glofitamab as a Single Agent vs. Investigator’s Choice in Participants With Relapsed/Refractory Mantle Cell Lymphoma
NCT06522737	PHASE3	RECRUITING	A Study of Duvelisib Versus Gemcitabine or Bendamustine in Participants With Relapsed/Refractory Nodal T Cell Lymphoma With T Follicular Helper (TFH) Phenotype
NCT00002456	PHASE3	COMPLETED	Graft-Versus-Host Disease Prevention in Treating Patients Who Are Undergoing Bone Marrow Transplantation
NCT00002495	PHASE3	COMPLETED	SWOG-9133 RT w/ or w/o Doxorubicin and Vinblastine in Stage I or Stage II Hodgkin’s Disease
NCT00002565	PHASE3	COMPLETED	Combination Chemotherapy in Treating Patients With Intermediate-Grade or Immunoblastic Non-Hodgkin’s Lymphoma
NCT00002576	PHASE3	COMPLETED	Combination Chemotherapy in Treating Older Patients With Intermediate- or High-Grade Non-Hodgkin’s Lymphoma

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
CYCLOPHOSPHAMIDE ANHYDROUS	4	299
PROCARBAZINE	4	69
VINCRISTINE SULFATE	4	37
2-MERCAPTOETHANESULFONIC ACID	4	36
VINBLASTINE	4	35
BLEOMYCIN SULFATE	4	29
ETOPOSIDE	4	25
FILGRASTIM	4	22
CYTARABINE	4	21
MITOXANTRONE HYDROCHLORIDE	4	19
DOXORUBICIN HYDROCHLORIDE	4	18
THIOTEPA	4	15
PLERIXAFOR	4	12
BUSULFAN	4	11
FLUDARABINE PHOSPHATE	4	9
IFOSFAMIDE	4	8
MECHLORETHAMINE HYDROCHLORIDE	4	7
BRENTUXIMAB VEDOTIN	4	6
IDARUBICIN	4	4
RASBURICASE	4	4
APREPITANT	4	3
CISPLATIN	4	2
GRANISETRON	4	2
VALPROIC ACID	4	2
INTERFERON ALFA-2B	4	1
PALONOSETRON	4	1
PEGINTERFERON ALFA-2B	4	1
PREDNISOLONE	4	1
RESPIRATORY SYNCYTIAL VIRUS PRE-FUSION GLYCOPROTEIN F	4	1
VARICELLA ZOSTER VIRUS ENVELOPE GLYCOPROTEIN E	4	1

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 7 predictive associations from 7 curated evidence items; also 2 predisposing, 1 oncogenic, 1 prognostic.

Molecular subtype	Therapy	Effect	Level	CIViC
ZMYM2::FGFR1 Fusion OR BCR::FGFR1 Fusion OR CEP43::FGFR1 Fusion OR TRIM24::FGFR1 Fusion OR FGFR1 Translocation	Pemigatinib	Sensitivity/Response	CIViC A	EID12242
ATM D2870A or N2875K	Irinotecan	Sensitivity/Response	CIViC D	EID12759
ATM Mutation	Camptothecin	Sensitivity/Response	CIViC D	EID12758
CCND3 Loss	Palbociclib	Sensitivity/Response	CIViC D	EID263
CREBBP R1446C	HDAC Inhibitor OBP-801	Sensitivity/Response	CIViC D	EID12291
BCL2 Overexpression	Venetoclax + Dinaciclib	Resistance	CIViC D	EID9313
KMT2E::ASNS Fusion	Asparaginase	Resistance	CIViC D	EID5926