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Atlas / Gene / PDGFRA

PDGFRA

gene
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Also known as CD140aPDGFR2GAS9

Summary

PDGFRA (platelet derived growth factor receptor alpha, HGNC:8803) is a protein-coding gene on chromosome 4q12, encoding Platelet-derived growth factor receptor alpha (P16234). Tyrosine-protein kinase that acts as a cell-surface receptor for PDGFA, PDGFB and PDGFC and plays an essential role in the regulation of embryonic development, cell proliferation, survival and chemotaxis. In precision oncology, PDGFRA D842V confers sensitivity to Imatinib in Gastrointestinal Stromal Tumor (CIViC Level A); 36 further curated variant–drug associations are listed below.

This gene encodes a cell surface tyrosine kinase receptor for members of the platelet-derived growth factor family. These growth factors are mitogens for cells of mesenchymal origin. The identity of the growth factor bound to a receptor monomer determines whether the functional receptor is a homodimer or a heterodimer, composed of both platelet-derived growth factor receptor alpha and beta polypeptides. Studies suggest that this gene plays a role in organ development, wound healing, and tumor progression. Mutations in this gene have been associated with idiopathic hypereosinophilic syndrome, somatic and familial gastrointestinal stromal tumors, and a variety of other cancers.

Source: NCBI Gene 5156 — RefSeq curated summary.

At a glance

  • Gene–disease (curated): gastrointestinal stromal tumor (Definitive, ClinGen) — +4 more curated relationships
  • GWAS associations: 34
  • Clinical variants (ClinVar): 3,917 total — 7 pathogenic
  • Phenotypes (HPO): 55
  • Druggable target: yes — 77 molecules with ChEMBL bioactivity
  • Precision-oncology evidence (CIViC): 37 curated variant–drug associations
  • Cancer driver (intOGen): activating (oncogene-like) across 7 cancer types
  • Dosage sensitivity (ClinGen): haploinsufficiency no evidence, triplosensitivity no evidence
  • MANE Select transcript: NM_006206

Identifiers

Gene identifiers

FieldValue
HGNC IDHGNC:8803
Approved symbolPDGFRA
Nameplatelet derived growth factor receptor alpha
Location4q12
Locus typegene with protein product
StatusApproved
AliasesCD140a, PDGFR2, GAS9
Ensembl geneENSG00000134853
Ensembl biotypeprotein_coding
OMIM173490
Entrez5156

Gene structure

Transcript identifiers

Ensembl transcripts: 23 — 19 protein_coding, 3 retained_intron, 1 nonsense_mediated_decay

ENST00000257290, ENST00000461294, ENST00000503856, ENST00000504461, ENST00000507536, ENST00000508170, ENST00000509092, ENST00000509490, ENST00000512143, ENST00000512522, ENST00000870889, ENST00000870890, ENST00000958745, ENST00000958746, ENST00000958747, ENST00000958748, ENST00000958749, ENST00000958750, ENST00000958751, ENST00000958752, ENST00000958753, ENST00000958754, ENST00000958755

RefSeq mRNA: 5 — MANE Select: NM_006206 NM_001347827, NM_001347828, NM_001347829, NM_001347830, NM_006206

CCDS: CCDS3495

Canonical transcript exons

ENST00000257290 — 23 exons

ExonStartEnd
ENSE000012263185429512554298245
ENSE000020280615422929354229415
ENSE000034628975428900954289114
ENSE000034640465427836254278515
ENSE000034718355429031354290554
ENSE000034821295425875754258817
ENSE000034837725428537154285486
ENSE000035290695427789654278006
ENSE000035470095426755254267741
ENSE000035489325427453154274625
ENSE000035669725426728954267460
ENSE000035700525428584154285963
ENSE000035717375427484154274973
ENSE000035809395426491954265049
ENSE000035849295427353754273730
ENSE000035869815428879954288898
ENSE000036265265427239454272520
ENSE000036343685426366754263927
ENSE000036478735428031654280482
ENSE000036736045427738854277492
ENSE000036744705427063354270748
ENSE000036816415428743054287541
ENSE000037852235426109554261412

Expression profiles

Bgee: expression breadth ubiquitous, 289 present calls, max score 99.38.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 34.1851 / max 610.7707, expressed in 1119 samples.

FANTOM5 promoters (13 alternative TSS)

Promoter IDTPM avgSamples expressed
4763721.0494875
476366.3735758
476442.3539725
476381.1781491
476350.5677339
476400.5548242
476430.5311320
476460.4616155
476410.3160195
476470.2525109

Top tissues by expression

296 total, by Bgee expression score (0-100, higher = more expressed):

TissueAnatomy IDExpression scoreQuality
tibiaUBERON:000097999.38gold quality
deciduaUBERON:000245099.29gold quality
synovial jointUBERON:000221799.19gold quality
pericardiumUBERON:000240799.04gold quality
lower lobe of lungUBERON:000894998.85gold quality
pylorusUBERON:000116698.75gold quality
stromal cell of endometriumCL:000225598.74gold quality
parietal pleuraUBERON:000240098.67gold quality
caput epididymisUBERON:000435898.58gold quality
left ovaryUBERON:000211998.56gold quality
pleuraUBERON:000097798.43gold quality
cauda epididymisUBERON:000436098.42gold quality
right ovaryUBERON:000211898.29gold quality
layer of synovial tissueUBERON:000761698.11gold quality
visceral pleuraUBERON:000240198.06gold quality
germinal epithelium of ovaryUBERON:000130497.97gold quality
urethraUBERON:000005797.96gold quality
ovaryUBERON:000099297.96gold quality
mucosa of urinary bladderUBERON:000125997.96gold quality
mammary ductUBERON:000176597.90gold quality
skin of hipUBERON:000155497.84gold quality
superficial temporal arteryUBERON:000161497.82gold quality
calcaneal tendonUBERON:000370197.81gold quality
tendon of biceps brachiiUBERON:000818897.78gold quality
epithelium of mammary glandUBERON:000324497.69gold quality
heart right ventricleUBERON:000208097.56gold quality
cardia of stomachUBERON:000116297.54gold quality
corpus epididymisUBERON:000435997.47gold quality
jejunal mucosaUBERON:000039997.30gold quality
tendonUBERON:000004397.28gold quality

Single-cell (SCXA)

Detected in 19 experiment(s), a significant marker in 16.

ExperimentMarker?Max mean expression
E-MTAB-9435yes6405.27
E-MTAB-9906yes1346.80
E-MTAB-8410yes1153.67
E-GEOD-84465yes1100.06
E-HCAD-56yes469.12
E-GEOD-109979yes294.20
E-MTAB-10287yes120.59
E-HCAD-10yes66.64
E-GEOD-135922yes59.67
E-HCAD-35yes55.65
E-GEOD-134144yes38.69
E-MTAB-6678yes28.01
E-CURD-46yes22.15
E-MTAB-9543yes20.19
E-MTAB-9388yes16.03

Regulation

Is transcription factor: no

Upstream regulators (CollecTRI, top): ASCL1, ATF4, CEBPB, CEBPD, CEBPG, CREB1, E2F1, FOS, FOXO1, GATA4, GATA6, GLI1, GLI2, HOXC6, MIXL1, NFKB, PAX1, PAX3, POU5F1, PPARG, SOX10, SP1, SP3, YY1, ZNF148, ZNF354C

miRNA regulators (miRDB)

192 targeting PDGFRA, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNAMax scoreAvg scoremiRNA target_count
HSA-MIR-8485100.0077.574731
HSA-MIR-4262100.0073.263931
HSA-MIR-3613-3P100.0076.367965
HSA-MIR-4795-3P100.0074.624024
HSA-MIR-196A-5P100.0068.16684
HSA-MIR-196B-5P100.0068.16681
HSA-MIR-29A-3P100.0073.111835
HSA-MIR-29B-3P100.0073.181833
HSA-MIR-29C-3P100.0073.151833
HSA-MIR-126-5P100.0072.713180
HSA-MIR-450A-1-3P100.0069.331837
HSA-MIR-432-3P100.0067.86705
HSA-MIR-4713-3P100.0065.92505
HSA-MIR-340-5P100.0072.504437
HSA-MIR-6833-3P100.0070.633197
HSA-MIR-5011-5P100.0083.465820
HSA-MIR-4481100.0066.421669
HSA-MIR-190A-3P100.0080.355520
HSA-MIR-4768-5P100.0069.492861
HSA-MIR-33A-5P99.9968.621055
HSA-MIR-33B-5P99.9968.581062
HSA-MIR-34A-5P99.9971.211784
HSA-MIR-449A99.9971.051776
HSA-MIR-181A-5P99.9972.962995
HSA-MIR-181B-5P99.9972.972996
HSA-MIR-181C-5P99.9972.952996
HSA-MIR-181D-5P99.9973.042997
HSA-MIR-548C-3P99.9974.017587
HSA-MIR-3617-3P99.9867.86918
HSA-MIR-569699.9872.364487

Functional genomics

ClinGen dosage: haploinsufficiency 0 (no evidence), triplosensitivity 0 (no evidence). ClinGen Gene Dosage Map

Literature-anchored findings (GeneRIF, showing 40)

  • high expression associated with long survival in patients with grade 2 astrocytomas and oligoastrocytomas (PMID:12023424)
  • PDGFRA is endogenenously activated in medulloblastoma tumor cell lines (PMID:12176024)
  • KIT and PDGFRA mutations appear to be alternative and mutually exclusive oncogenic mechanisms in gastrointestinal stromal tumors (PMID:12522257)
  • The hypereosinophilic syndrome may result from a novel fusion tyrosine kinase - FIP1L1-PDGFRalpha - that is a consequence of an interstitial chromosomal deletion. (PMID:12660384)
  • observations suggest that the FIP1L1-PDGFRA rearrangement occurs in an early hematopoietic progenitor and suggests that the molecular pathogenesis for a subset of SMCD patients is similar to that of HES (PMID:12842979)
  • results indicate that the fusion of FIP1L1 to PDGFRA occurs rarely in leukemia cell lines (PMID:14630792)
  • Result from our case-control study demonstrated that the frequencies of haplotypes with low transcription activity were significantly higher in NTD mothers than that observed in control mothers (odds ratio=2.2, 95% CI=1.0-4.6). (PMID:14741194)
  • results confirm the high frequency of BCR-ABL kinase domain mutations in patients with secondary resistance to imatinib and exclude mutations of the activation loops of KIT, PDGFRA and PDGFRB as causes of resistance in patients without ABL mutations (PMID:14745431)
  • PDGFRa expression is regulated by C-EBP (PMID:14766209)
  • PDGFRA mutations were found in 7 (25.0%)gastrointestinal stromal tumors (GISTs) , carrying either D842 V mutations or exon 18 deletions. (PMID:15154005)
  • FIP1L1-PDGFRA is a relatively infrequent but treatment-relevant mutation in primary eosinophilia that is indicative of an underlying systemic mastocytosis. (PMID:15284118)
  • gene expression profiling of 26 gastrointesstinsal stromsal tumors with known KIT and PDGFRA mutational status (PMID:15326474)
  • These results suggest that myxoid epithelioid GISTs are a distinct subtype of GISTs that are closely correlated with the PDGFRA gene mutation (PMID:15492989)
  • link between single nucleotide polymorphisms (SNPs) within the PDGFRalpha gene promoter and the occurrence of brain tumours (PMID:15635072)
  • The Receptor, PDGF alpha (PDGFRA)is implicated in the pathogenesis of AML, and the mechanisms of cytogenetic progression and oncoprotein-driven function may be similar in AML expressing oncogenic forms of PDGFRA. (PMID:15674355)
  • High sensitivity of the imatinib-resistant KIT -T670I and KIT -V654A and of PDGFRA -D842V mutants to PKC412 in gastrointestinal stromal tumors. (PMID:15685537)
  • study demonstrates that platelet-derived growth factor receptor alpha and beta, and c-kit protein are expressed in a high percentage of epithelial ovarian cancers (PMID:15791568)
  • Western blot showed a high amount of PDGFRalpha and beta proteins in primary stromal cells that could not be detected in prostatic cancer cells. (PMID:15862965)
  • In human ASMCs, IFNgamma again stimulated a transient STAT1-binding to the PDGF-Ralpha promoter. (PMID:15871904)
  • single example of Gastrointestinal Stromal Tumor with activating mutation and immunoreactivity without CD117 positivity (PMID:15894928)
  • The point mutations of c-kit and platelet- derived growth factor receptor alpha genes may play a limited role in the tumorigenesis of type 1 neurofibromatosis-associated gastrointestinal stromal tumors. (PMID:15897742)
  • PDGFRA mutations are associated with gastrointestinal stromal tumors (PMID:15928335)
  • The hypereosinophilic syndrome may result from a novel fusion tyrosine kinase - FIP1L1-PDGFRalpha - that is a consequence of an interstitial chromosomal deletion. (PMID:16116920)
  • The current identification of cis-acting elements in the PDGFRA promoter and the transcription factors that bind them, provides a new strategy for the identification of genes that are potentially involved in neural tube defects. (PMID:16126374)
  • An intronic insertion IVS17-50insA at exon 18 in all sequenced cases. None of these genetic alterations was correlated with PDGFR-alpha expression in breast cancer. (PMID:16168125)
  • Two-basepair deletion in the C terminus of PDGFRA (exon 23) found in glioblastomas. (PMID:16186508)
  • PDGFRA P1 promoter region haplotypes do not have a major role in the development of spina bifida meningomyelocele . (PMID:16283668)
  • data describe percentage of bone marrow cells expressing receptors for interleukin-1, platelet-derived growth factor, fibroblast growth factor, transforming growth factor-beta, epidermal growth factor and c-Fos and c-Myc in untreated lung & breast cancer (PMID:16305343)
  • Specific PDGFR inhibition by siRNA or a neutralizing antibody to the receptor inhibited PDGF-stimulated receptor activation and cell proliferation, suggesting that receptor targeting has a role in ovarian cancer treatment (PMID:16331269)
  • describes frequence mutation of PDGFRA in malignant periphaerl nerve sheath tumors often associated with coamplification of KIT (PMID:16357008)
  • activating mutations of PDGFR-alpha, c-kit and B-RAF are absent in gliosarcomas (PMID:16373964)
  • Demonstrates the utility of screening for PDGFRA kinase domain overexpression in patients with IHES and has identified KIF5B as a third PDGFRA fusion partner in chronic myeloproliferative disorders. (PMID:16498388)
  • Fusion protein is detected in hypereosinophilic syndrome and chronic eosinophilic leukemia. (PMID:16502585)
  • The most intense expression of PDGFRalpha was observed in fibroblasts in the lung carcinoma outer rim. (PMID:16675559)
  • Six of the 20 gastrointestinal stomach tumors lacking KIT mutations had mutations in exon 18 of PDGFRA, and 1 had a mutation in exon 12 in this receptor. (PMID:16685437)
  • FIP1L1-PDGFRalpha activation requires disruption of the juxtamembrane domain of PDGFRalpha and is FIP1L1-independent (PMID:16690743)
  • Gastrointestinal stromal tumours (GIST) with platelet-derived growth factor receptor mutations often have reduced expression of the KIT protein in immunohistochemistry (IHC), suggesting that IHC may be potentially useful in identification of such GISTs. (PMID:16785193)
  • 77% of the examined primary central nervous system lymphoma specimens showed expression of PDGFRalpha. Tumours expressing survivin frequently co-expressed PDGFRalpha. (PMID:16850112)
  • PDGFRalpha mutations have been identified as alternative oncogenic mechanism in gastrointestinal stromal tumors. (PMID:16892550)
  • In midgut carcinoid tumor stroma PDGFRalpha was expressed in 35%, PDGFRbeta in 94% and EGFR in 9%. (PMID:17047316)

Cross-species orthologs

3 orthologs

OrganismSymbolGene ID
danio_reriopdgfraENSDARG00000070494
mus_musculusPdgfraENSMUSG00000029231
rattus_norvegicusPdgfraENSRNOG00000002244

Paralogs (53): INSRR (ENSG00000027644), MUSK (ENSG00000030304), FLT4 (ENSG00000037280), EPHA3 (ENSG00000044524), ROS1 (ENSG00000047936), LTK (ENSG00000062524), ERBB3 (ENSG00000065361), TIE1 (ENSG00000066056), FGFR2 (ENSG00000066468), FGFR3 (ENSG00000068078), EPHA8 (ENSG00000070886), FGFR1 (ENSG00000077782), EPHA6 (ENSG00000080224), TYRO3 (ENSG00000092445), FLT1 (ENSG00000102755), MET (ENSG00000105976), EPHB6 (ENSG00000106123), PDGFRB (ENSG00000113721), EPHA4 (ENSG00000116106), TEK (ENSG00000120156), FLT3 (ENSG00000122025), KDR (ENSG00000128052), EPHB2 (ENSG00000133216), EPHA7 (ENSG00000135333), IGF1R (ENSG00000140443), NTRK3 (ENSG00000140538), ERBB2 (ENSG00000141736), EPHA2 (ENSG00000142627), EPHA5 (ENSG00000145242), EGFR (ENSG00000146648), EPHA1 (ENSG00000146904), NTRK2 (ENSG00000148053), MERTK (ENSG00000153208), EPHB1 (ENSG00000154928), KIT (ENSG00000157404), FGFR4 (ENSG00000160867), DDR2 (ENSG00000162733), RYK (ENSG00000163785), MST1R (ENSG00000164078), LMTK2 (ENSG00000164715)

Protein

Protein identifiers

Platelet-derived growth factor receptor alpha — P16234 (reviewed: P16234)

Alternative names: Alpha platelet-derived growth factor receptor, Alpha-type platelet-derived growth factor receptor, CD140 antigen-like family member A, CD140a antigen, Platelet-derived growth factor alpha receptor, Platelet-derived growth factor receptor 2

All UniProt accessions (5): D6RDX0, D6RG11, D6RIG5, D6RJH0, P16234

UniProt curated annotations — full annotation on UniProt →

Function. Tyrosine-protein kinase that acts as a cell-surface receptor for PDGFA, PDGFB and PDGFC and plays an essential role in the regulation of embryonic development, cell proliferation, survival and chemotaxis. Depending on the context, promotes or inhibits cell proliferation and cell migration. Plays an important role in the differentiation of bone marrow-derived mesenchymal stem cells. Required for normal skeleton development and cephalic closure during embryonic development. Required for normal development of the mucosa lining the gastrointestinal tract, and for recruitment of mesenchymal cells and normal development of intestinal villi. Plays a role in cell migration and chemotaxis in wound healing. Plays a role in platelet activation, secretion of agonists from platelet granules, and in thrombin-induced platelet aggregation. Binding of its cognate ligands - homodimeric PDGFA, homodimeric PDGFB, heterodimers formed by PDGFA and PDGFB or homodimeric PDGFC -leads to the activation of several signaling cascades; the response depends on the nature of the bound ligand and is modulated by the formation of heterodimers between PDGFRA and PDGFRB. Phosphorylates PIK3R1, PLCG1, and PTPN11. Activation of PLCG1 leads to the production of the cellular signaling molecules diacylglycerol and inositol 1,4,5-trisphosphate, mobilization of cytosolic Ca(2+) and the activation of protein kinase C. Phosphorylates PIK3R1, the regulatory subunit of phosphatidylinositol 3-kinase, and thereby mediates activation of the AKT1 signaling pathway. Mediates activation of HRAS and of the MAP kinases MAPK1/ERK2 and/or MAPK3/ERK1. Promotes activation of STAT family members STAT1, STAT3 and STAT5A and/or STAT5B. Receptor signaling is down-regulated by protein phosphatases that dephosphorylate the receptor and its down-stream effectors, and by rapid internalization of the activated receptor.

Subunit / interactions. Interacts with homodimeric PDGFA, PDGFB and PDGFC, and with heterodimers formed by PDGFA and PDGFB. Monomer in the absence of bound ligand. Interaction with dimeric PDGFA, PDGFB and/or PDGFC leads to receptor dimerization, where both PDGFRA homodimers and heterodimers with PDGFRB are observed. Interacts (tyrosine phosphorylated) with SHB (via SH2 domain). Interacts (tyrosine phosphorylated) with SHF (via SH2 domain). Interacts (tyrosine phosphorylated) with SRC (via SH2 domain). Interacts (tyrosine phosphorylated) with PIK3R1. Interacts (tyrosine phosphorylated) with PLCG1 (via SH2 domain). Interacts (tyrosine phosphorylated) with CRK, GRB2 and GRB7. Interacts with CD248; this interaction promotes PDGF receptor signaling pathway. (Microbial infection) Interacts with human cytomegalovirus/HHV-5 envelope glycoprotein B/gB. Also interacts with the trimeric complex gH-gL-gO. Trimer-PDGFRA interaction has an inhibitory effect on PDGFRA signaling.

Subcellular location. Cell membrane. Cell projection. Cilium. Golgi apparatus.

Tissue specificity. Detected in platelets (at protein level). Widely expressed. Detected in brain, fibroblasts, smooth muscle, heart, and embryo. Expressed in primary and metastatic colon tumors and in normal colon tissue.

Post-translational modifications. N-glycosylated. Ubiquitinated, leading to its internalization and degradation. Autophosphorylated on tyrosine residues upon ligand binding. Autophosphorylation occurs in trans, i.e. one subunit of the dimeric receptor phosphorylates tyrosine residues on the other subunit. Phosphorylation at Tyr-731 and Tyr-742 is important for interaction with PIK3R1. Phosphorylation at Tyr-720 and Tyr-754 is important for interaction with PTPN11. Phosphorylation at Tyr-762 is important for interaction with CRK. Phosphorylation at Tyr-572 and Tyr-574 is important for interaction with SRC and SRC family members. Phosphorylation at Tyr-988 and Tyr-1018 is important for interaction with PLCG1.

Disease relevance. A chromosomal aberration involving PDGFRA is found in some cases of hypereosinophilic syndrome. Interstitial chromosomal deletion del(4)(q12q12) causes the fusion of FIP1L1 and PDGFRA (FIP1L1-PDGFRA). Mutations that cause overexpression and/or constitutive activation of PDGFRA may be a cause of hypereosinophilic syndrome. Gastrointestinal stromal tumor (GIST) [MIM:606764] Common mesenchymal neoplasms arising in the gastrointestinal tract, most often in the stomach. They are histologically, immunohistochemically, and genetically different from typical leiomyomas, leiomyosarcomas, and schwannomas. Most GISTs are composed of a fairly uniform population of spindle-shaped cells. Some tumors are dominated by epithelioid cells or contain a mixture of spindle and epithelioid morphologies. Primary GISTs in the gastrointestinal tract commonly metastasize in the omentum and mesenteries, often as multiple nodules. However, primary tumors may also occur outside of the gastrointestinal tract, in other intra-abdominal locations, especially in the omentum and mesentery. The gene represented in this entry may be involved in disease pathogenesis. Mutations causing PDGFRA constitutive activation have been found in gastrointestinal stromal tumors lacking KIT mutations. GIST-plus syndrome (GISTPS) [MIM:175510] A disorder characterized by multiple mesenchymal tumors of the gastrointestinal tract, including gastrointestinal stromal tumor, inflammatory fibroid polyps, and fibroid tumors. Additional features are coarse facies and skin, broad hands and feet, and premature tooth loss. GISTPS is an autosomal dominant disease with incomplete penetrance. Gastrointestinal stromal tumor and inflammatory fibroid polyps may also occur in isolation. The disease is caused by variants affecting the gene represented in this entry.

Activity regulation. Present in an inactive conformation in the absence of bound ligand. Binding of PDGFA and/or PDGFB leads to dimerization and activation by autophosphorylation on tyrosine residues. Inhibited by imatinib, nilotinib and sorafenib.

Similarity. Belongs to the protein kinase superfamily. Tyr protein kinase family. CSF-1/PDGF receptor subfamily.

Isoforms (3)

UniProt IDNamesCanonical?
P16234-11yes
P16234-22
P16234-33

RefSeq proteins (5): NP_001334756, NP_001334757, NP_001334758, NP_001334759, NP_006197* (*=MANE)

Domains & families (InterPro)

IDNameType
IPR000719Prot_kinase_domDomain
IPR001245Ser-Thr/Tyr_kinase_cat_domDomain
IPR001824Tyr_kinase_rcpt_3_CSConserved_site
IPR003598Ig_sub2Domain
IPR003599Ig_subDomain
IPR007110Ig-like_domDomain
IPR008266Tyr_kinase_ASActive_site
IPR011009Kinase-like_dom_sfHomologous_superfamily
IPR013098Ig_I-setDomain
IPR013783Ig-like_foldHomologous_superfamily
IPR017441Protein_kinase_ATP_BSBinding_site
IPR020635Tyr_kinase_cat_domDomain
IPR027290PDGFRAFamily
IPR036179Ig-like_dom_sfHomologous_superfamily
IPR050122RTKFamily

Pfam: PF07679, PF07714, PF25305

Enzyme classification (BRENDA):

  • EC 2.7.10.1 — receptor protein-tyrosine kinase (BRENDA: 44 organisms, 214 substrates, 574 inhibitors, 11 Km, 0 kcat entries)

Substrate kinetics (BRENDA)

4 substrates with measured Km, best-characterized 4. Km ranges are aggregated across organisms/conditions.

SubstrateKm (mM)Measurements
ATP0.0011–0.1294
AC-DYFE-6-CHLORO-W-NHME0.00511
AC-DYFGW-NHME0.071
YFEW0.2321

Catalyzed reactions (Rhea), 1 shown:

  • L-tyrosyl-[protein] + ATP = O-phospho-L-tyrosyl-[protein] + ADP + H(+) (RHEA:10596)

UniProt features (150 total): strand 47, sequence variant 26, helix 22, modified residue 11, glycosylation site 8, domain 6, mutagenesis site 6, turn 4, disulfide bond 4, splice variant 4, compositionally biased region 2, binding site 2, topological domain 2, signal peptide 1, chain 1, region of interest 1, active site 1, site 1, transmembrane region 1

Structure

Experimental structures (PDB)

15 structures.

PDBMethodResolution (Å)
5GRNX-RAY DIFFRACTION1.77
8PQJX-RAY DIFFRACTION1.82
6A32X-RAY DIFFRACTION1.87
6JOLX-RAY DIFFRACTION1.9
8PQKX-RAY DIFFRACTION2
5K5XX-RAY DIFFRACTION2.17
1GQ5X-RAY DIFFRACTION2.2
9GZHX-RAY DIFFRACTION2.2
8PQHX-RAY DIFFRACTION2.5
6JOJX-RAY DIFFRACTION2.6
8PQIX-RAY DIFFRACTION2.6
7LBFELECTRON MICROSCOPY2.8
6JOIX-RAY DIFFRACTION3.1
7RAMELECTRON MICROSCOPY3.43
6JOKX-RAY DIFFRACTION3.8

Predicted structure (AlphaFold)

ModelpLDDTFraction very-high
AF-P16234-F172.910.25

Antibody-complex structures (SAbDab): 1 — 7LBF

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Catalytic / active sites (2): 818 (proton acceptor); 578–579 (breakpoint for interstitial deletion to form the fip1l1-pdgfra fusion protein)

Ligand- & substrate-binding residues (2): 599–607; 627

Post-translational modifications (11): 572, 574, 720, 731, 742, 754, 762, 768, 849, 988, 1018

Disulfide bonds (4): 49–100, 150–189, 235–290, 435–501

Glycosylation sites (8): 42, 76, 103, 179, 353, 359, 458, 468

Mutagenesis-validated functional residues (6):

PositionPhenotype
572abolishes interaction with src-family members and impairs internalization of the activated receptor; when associated wit
574abolishes interaction with src-family members and impairs internalization of the activated receptor; when associated wit
720strongly reduced interaction with ptpn11 and grb2.
731no effect on autophosphorylation and phosphorylation of plcg1. abolishes activation of phosphatidylinositol 3-kinase.
742no effect on autophosphorylation and phosphorylation of plcg1. abolishes activation of phosphatidylinositol 3-kinase.
762abolishes interaction with crk.

Function

Pathways and Gene Ontology

Reactome pathways

13 pathways

IDPathway
R-HSA-1257604PIP3 activates AKT signaling
R-HSA-186763Downstream signal transduction
R-HSA-186797Signaling by PDGF
R-HSA-2219530Constitutive Signaling by Aberrant PI3K in Cancer
R-HSA-5673001RAF/MAP kinase cascade
R-HSA-6811558PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling
R-HSA-9673767Signaling by PDGFRA transmembrane, juxtamembrane and kinase domain mutants
R-HSA-9673770Signaling by PDGFRA extracellular domain mutants
R-HSA-9674396Imatinib-resistant PDGFR mutants
R-HSA-9674401Sunitinib-resistant PDGFR mutants
R-HSA-9674403Regorafenib-resistant PDGFR mutants
R-HSA-9674404Sorafenib-resistant PDGFR mutants
R-HSA-9674428PDGFR mutants bind TKIs

MSigDB gene sets: 747 (showing top): GOBP_PLATELET_DERIVED_GROWTH_FACTOR_RECEPTOR_SIGNALING_PATHWAY, GOBP_REGULATION_OF_CELL_ACTIVATION, GOBP_HEMATOPOIETIC_PROGENITOR_CELL_DIFFERENTIATION, MODULE_52, GOBP_EMBRYO_DEVELOPMENT_ENDING_IN_BIRTH_OR_EGG_HATCHING, ACTACCT_MIR196A_MIR196B, GOBP_BODY_MORPHOGENESIS, GOBP_MUSCLE_TISSUE_DEVELOPMENT, BENPORATH_ES_WITH_H3K27ME3, IIZUKA_LIVER_CANCER_EARLY_RECURRENCE, GOBP_VASCULAR_ENDOTHELIAL_GROWTH_FACTOR_SIGNALING_PATHWAY, GOBP_REGULATION_OF_CALCIUM_MEDIATED_SIGNALING, GOBP_METANEPHROS_DEVELOPMENT, GOBP_REGULATION_OF_WOUND_HEALING, GOBP_CELL_CHEMOTAXIS

GO Biological Process (49): luteinization (GO:0001553), in utero embryonic development (GO:0001701), cell activation (GO:0001775), hematopoietic progenitor cell differentiation (GO:0002244), cell surface receptor protein tyrosine kinase signaling pathway (GO:0007169), estrogen metabolic process (GO:0008210), positive regulation of cell population proliferation (GO:0008284), negative regulation of platelet activation (GO:0010544), cell migration (GO:0016477), peptidyl-tyrosine phosphorylation (GO:0018108), signal transduction involved in regulation of gene expression (GO:0023019), extracellular matrix organization (GO:0030198), lung development (GO:0030324), adrenal gland development (GO:0030325), positive regulation of cell migration (GO:0030335), male genitalia development (GO:0030539), regulation of actin cytoskeleton organization (GO:0032956), Leydig cell differentiation (GO:0033327), cellular response to reactive oxygen species (GO:0034614), platelet-derived growth factor receptor-alpha signaling pathway (GO:0035790), positive regulation of cell proliferation by VEGF-activated platelet derived growth factor receptor signaling pathway (GO:0038091), wound healing (GO:0042060), odontogenesis of dentin-containing tooth (GO:0042475), protein autophosphorylation (GO:0046777), platelet-derived growth factor receptor signaling pathway (GO:0048008), positive regulation of fibroblast proliferation (GO:0048146), embryonic digestive tract morphogenesis (GO:0048557), embryonic cranial skeleton morphogenesis (GO:0048701), embryonic skeletal system morphogenesis (GO:0048704), positive regulation of calcium-mediated signaling (GO:0050850), white fat cell differentiation (GO:0050872), positive regulation of chemotaxis (GO:0050921), positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction (GO:0051897), cardiac myofibril assembly (GO:0055003), roof of mouth development (GO:0060021), face morphogenesis (GO:0060325), cell chemotaxis (GO:0060326), retina vasculature development in camera-type eye (GO:0061298), positive regulation of ERK1 and ERK2 cascade (GO:0070374), platelet aggregation (GO:0070527)

GO Molecular Function (16): protein kinase activity (GO:0004672), transmembrane receptor protein tyrosine kinase activity (GO:0004714), platelet-derived growth factor alpha-receptor activity (GO:0005018), vascular endothelial growth factor receptor activity (GO:0005021), platelet-derived growth factor receptor binding (GO:0005161), ATP binding (GO:0005524), vascular endothelial growth factor binding (GO:0038085), protein homodimerization activity (GO:0042803), protein-containing complex binding (GO:0044877), platelet-derived growth factor binding (GO:0048407), phospholipase C activator activity (GO:0160185), nucleotide binding (GO:0000166), protein tyrosine kinase activity (GO:0004713), protein binding (GO:0005515), kinase activity (GO:0016301), transferase activity (GO:0016740)

GO Cellular Component (18): nucleus (GO:0005634), nucleoplasm (GO:0005654), cytoplasm (GO:0005737), endoplasmic reticulum membrane (GO:0005789), Golgi apparatus (GO:0005794), cytosol (GO:0005829), plasma membrane (GO:0005886), microvillus (GO:0005902), cilium (GO:0005929), external side of plasma membrane (GO:0009897), membrane (GO:0016020), nuclear body (GO:0016604), cell junction (GO:0030054), protein-containing complex (GO:0032991), signaling receptor complex (GO:0043235), platelet-derived growth factor receptor-ligand complex (GO:1990270), cell surface (GO:0009986), cell projection (GO:0042995)

Reactome top-level categories

Rollup of top-8 pathways:

CategoryPathways
Drug resistance of PDGFR mutants4
Signaling by PDGFR in disease3
Intracellular signaling by second messengers1
Signaling by PDGF1
Signaling by Receptor Tyrosine Kinases1
PI3K/AKT Signaling in Cancer1
MAPK1/MAPK3 signaling1
Negative regulation of the PI3K/AKT network1

GO top-level categories

Rollup of top GO terms by namespace:

CategoryTerms
cellular anatomical structure7
cell differentiation2
growth factor binding2
binding2
intracellular membrane-bounded organelle2
cytoplasm2
female gonad development1
ovulation cycle process1
chordate embryonic development1
cellular process1
multicellular organismal process1
hemopoiesis1
enzyme-linked receptor protein signaling pathway1
steroid metabolic process1
hormone metabolic process1
cell population proliferation1
regulation of cell population proliferation1
positive regulation of cellular process1
regulation of platelet activation1
platelet activation1
negative regulation of blood coagulation1
negative regulation of cell activation1
cell motility1
protein phosphorylation1
peptidyl-tyrosine modification1
signal transduction1
regulation of gene expression1
extracellular structure organization1
external encapsulating structure organization1
respiratory tube development1
animal organ development1
respiratory system development1
endocrine system development1
gland development1
cell migration1
regulation of cell migration1
positive regulation of cell motility1
male sex differentiation1
genitalia development1
reproductive system development1

Protein interactions and networks

STRING

4664 interactions, top by confidence (×1000):

Protein AProtein BPartner UniProtScore
PDGFRAPDGFAP04085998
PDGFRAPDGFCQ9NRA1998
PDGFRAPDGFBP01127996
PDGFRAPDGFDQ9GZP0991
PDGFRAFIP1L1Q6UN15981
PDGFRAFGF2P09038941
PDGFRAPTPN11Q06124889
PDGFRAEGFP01133877
PDGFRAPIK3CAP42336867
PDGFRAKITLGP21583809
PDGFRAOLIG2Q13516809
PDGFRAANO1Q5XXA6804
PDGFRANF1P21359803
PDGFRAPIK3R1P27986796
PDGFRAPTENP60484796

IntAct

89 interactions, top by confidence:

ABTypeScore
PIK3R3PIK3CDpsi-mi:“MI:0914”(association)0.800
PDGFRBPDGFRApsi-mi:“MI:0914”(association)0.770
PDGFRBPDGFRApsi-mi:“MI:2364”(proximity)0.770
PDGFRAPDGFBpsi-mi:“MI:0915”(physical association)0.730
PDGFBPDGFRApsi-mi:“MI:0407”(direct interaction)0.730
PDGFRAPDGFBpsi-mi:“MI:0407”(direct interaction)0.730
PDGFAPDGFRApsi-mi:“MI:2364”(proximity)0.690
PDGFRAPDGFApsi-mi:“MI:0407”(direct interaction)0.690
EGFRPDGFRApsi-mi:“MI:0915”(physical association)0.680
PDGFRAEGFRpsi-mi:“MI:0915”(physical association)0.680
PDGFRACRKpsi-mi:“MI:2364”(proximity)0.670
CRKPDGFRApsi-mi:“MI:0914”(association)0.670
PDGFCPDGFRApsi-mi:“MI:0407”(direct interaction)0.660
PDGFRBPIK3R2psi-mi:“MI:0914”(association)0.610
CRKLPDGFRApsi-mi:“MI:2364”(proximity)0.570
CRKLPDGFRApsi-mi:“MI:0914”(association)0.570

BioGRID (433): CCDC155 (Two-hybrid), PDGFRA (Two-hybrid), PDGFRA (Affinity Capture-MS), CLU (Affinity Capture-MS), DSG2 (Affinity Capture-MS), H3F3A (Affinity Capture-MS), MYO1C (Affinity Capture-MS), NRD1 (Affinity Capture-MS), PDGFRA (Affinity Capture-MS), TFRC (Affinity Capture-MS), TNK1 (Affinity Capture-MS), FARP1 (Affinity Capture-MS), FLOT1 (Affinity Capture-MS), RNPS1 (Affinity Capture-MS), SCFD1 (Affinity Capture-MS)

ESM2 similar proteins: A7MB46, F8W3X3, O14917, O35902, O54800, O97799, P05622, P16234, P20786, P26618, P26619, P32926, P35546, P55286, P55289, P79749, P97291, Q05030, Q08DJ5, Q13634, Q14126, Q28889, Q5RJH3, Q68SP4, Q6KEQ9, Q6W3B0, Q6WXV7, Q6WYY1, Q6X862, Q71M42, Q7TMD7, Q7TSF0, Q7YRU7, Q80TF3, Q86SJ6, Q8AXC6, Q8AXC7, Q8BIZ0, Q8N6Y1, Q8TAB3

Diamond homologs: D2IYS2, G3V9H8, O08775, O42127, O73791, O73798, O97799, P00529, P00545, P04048, P05532, P05622, P07333, P07949, P09581, P09619, P10721, P11362, P13369, P16092, P16234, P17948, P18460, P18461, P20786, P21802, P21803, P21804, P22182, P22455, P22607, P23049, P26618, P26619, P33497, P35546, P35590, P35916, P35917, P35918

SIGNOR signaling

33 interactions.

AEffectBMechanism
PDGFB“up-regulates activity”PDGFRAbinding
PDGFAup-regulatesPDGFRAbinding
sunitinibdown-regulatesPDGFRA“chemical inhibition”
PDGFRAup-regulatesCRKbinding
N-(1,3-benzodioxol-5-ylmethyl)-4-(4-benzofuro[3,2-d]pyrimidinyl)-1-piperazinecarbothioamidedown-regulatesPDGFRA“chemical inhibition”
Crenolanibdown-regulatesPDGFRA“chemical inhibition”
linifanibdown-regulatesPDGFRA“chemical inhibition”
PDGFRA“up-regulates activity”SRCphosphorylation
PDGFRAup-regulatesAKT1
PDGFRAup-regulatesERK1/2
imatinib“down-regulates activity”PDGFRA“chemical inhibition”
regorafenib“down-regulates activity”PDGFRA“chemical inhibition”
PDGFRA“up-regulates activity”PTK2phosphorylation
PDGFRAup-regulatesPLCG1phosphorylation
CBL“down-regulates quantity by destabilization”PDGFRAubiquitination
PDGFRA“up-regulates activity”PDGFRAphosphorylation
PTPRG“up-regulates activity”PDGFRAdephosphorylation
nintedanib“down-regulates activity”PDGFRA“chemical inhibition”
axitinib“down-regulates activity”PDGFRA“chemical inhibition”
“2-Hydroxy-3-[N-[4-[methyl-[2-(4-methylpiperazin-1-yl)acetyl]amino]phenyl]-C-phenylcarbonimidoyl]-1H-indole-6-carboxylic acid”“down-regulates activity”PDGFRA“chemical inhibition”
canertinib“down-regulates activity”PDGFRA“chemical inhibition”
masitinib“down-regulates activity”PDGFRA“chemical inhibition”
nilotinib“down-regulates activity”PDGFRA“chemical inhibition”
tandutinib“down-regulates activity”PDGFRA“chemical inhibition”

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 52 IntAct physical interaction partners (hypergeometric vs the genome-wide background, BH-FDR, gene-set size 15–500, ranked by fold). A functional readout of the neighbourhood — distinct from this gene’s own memberships above, and biased toward well-studied / hub proteins, so read it as themes rather than proof.

Reactome pathways:

PathwayPartnersFoldFDR
Downstream signal transduction1088.5×2e-15
Regulation of signaling by CBL669.3×2e-08
Signaling by phosphorylated juxtamembrane, extracellular and kinase domain KIT mutants560.4×7e-07
Signaling by CSF1 (M-CSF) in myeloid cells648.3×1e-07
Interleukin receptor SHC signaling547.4×2e-06
Interleukin-7 signaling536.9×6e-06
RET signaling636.2×7e-07
Signaling by PDGF635.4×7e-07

GO biological processes:

GO termPartnersFoldFDR
platelet-derived growth factor receptor signaling pathway559.8×4e-06
phosphatidylinositol 3-kinase/protein kinase B signal transduction940.3×8e-10
positive regulation of miRNA transcription530.9×6e-05
positive regulation of ERK1 and ERK2 cascade916.3×1e-06
positive regulation of angiogenesis614.7×3e-04
actin filament organization512.6×1e-03
positive regulation of cell migration911.8×9e-06
positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction711.7×2e-04

Disease & clinical

Cancer significance

From CIViC — curated cancer-variant interpretation:

Commonly mutated in GI tract tumors, PDGFR family genes (mutually exclusive to KIT mutations) are a hallmark of gastrointestinal stromal tumors. Gene fusions involving the PDGFRA kinase domain are highly correlated with eosinophilia, and the WHO classifies myeloid and lymphoid neoplasms with these characteristics as a distinct disorder. Mutations in the 842 region of PDGFRA have been often found to confer resistance to the tyrosine kinase inhibitor, imatinib.

From intOGen — cancer-driver classification: activating (oncogene-like) across 7 cancer types — CSCC, GB, GBM, HGGNOS, LGGNOS, LUSC, PAST.

Clinical variants and AI predictions

ClinVar

3917 variants total. Per-class counts are floors (≥ shown; pagination cap):

ClassificationCount (floor)
Pathogenic7
Likely pathogenic0
Uncertain significance2051
Likely benign1402
Benign92

Top pathogenic / likely-pathogenic (7)

Variant IDHGVSClassification
13545NM_006206.6(PDGFRA):c.2533_2544del (p.His845_Asn848del)Pathogenic
13547NM_006206.6(PDGFRA):c.1681_1682insAGAGGG (p.Arg560_Val561insGluArg)Pathogenic
13548NM_006206.6(PDGFRA):c.1679_1693del (p.Arg560_Ser564del)Pathogenic
13549NM_006206.6(PDGFRA):c.1696_1713del (p.Ser566_Glu571del)Pathogenic
13552NM_006206.6(PDGFRA):c.1664A>G (p.Tyr555Cys)Pathogenic
635777NM_006206.6(PDGFRA):c.1957_1958delinsTT (p.Pro653Leu)Pathogenic
635778NM_006206.6(PDGFRA):c.2537A>T (p.Asp846Val)Pathogenic

SpliceAI

4137 predictions. Top by Δscore:

VariantEffectΔscore
4:54251430:G:GTdonor_gain1.0000
4:54258755:A:AGacceptor_gain1.0000
4:54258756:G:GAacceptor_gain1.0000
4:54261409:CCAGG:Cdonor_loss1.0000
4:54261410:CAGG:Cdonor_loss1.0000
4:54261410:CAGGT:Cdonor_loss1.0000
4:54261411:AGGTG:Adonor_loss1.0000
4:54261412:GGT:Gdonor_loss1.0000
4:54261413:G:Cdonor_loss1.0000
4:54261413:G:Tdonor_loss1.0000
4:54261414:T:Adonor_loss1.0000
4:54263665:A:AGacceptor_gain1.0000
4:54263666:G:GGacceptor_gain1.0000
4:54263800:G:GAdonor_gain1.0000
4:54263884:A:Tdonor_gain1.0000
4:54263887:G:GTdonor_gain1.0000
4:54267421:G:Tdonor_gain1.0000
4:54272518:G:GTdonor_gain1.0000
4:54272518:GAA:Gdonor_gain1.0000
4:54272521:G:GGdonor_gain1.0000
4:54272534:A:Gdonor_gain1.0000
4:54273679:GC:Gdonor_gain1.0000
4:54273731:G:GGdonor_gain1.0000
4:54273764:C:Gdonor_gain1.0000
4:54274969:GCTTG:Gdonor_gain1.0000
4:54274971:TTGGT:Tdonor_loss1.0000
4:54274973:GGTA:Gdonor_loss1.0000
4:54274974:G:Adonor_loss1.0000
4:54274975:T:Gdonor_loss1.0000
4:54277493:G:GGdonor_gain1.0000

AlphaMissense

7179 scored. Top likely-pathogenic:

VariantProtein changeam_pathogenicity
4:54263747:T:CC150R1.000
4:54274862:T:AW559R1.000
4:54274862:T:CW559R1.000
4:54274864:G:CW559C1.000
4:54274864:G:TW559C1.000
4:54274869:T:AV561D1.000
4:54274872:T:AI562N1.000
4:54274926:T:AL580Q1.000
4:54274926:T:CL580P1.000
4:54274943:T:AW586R1.000
4:54274943:T:CW586R1.000
4:54274965:T:CL593P1.000
4:54277399:G:AG600R1.000
4:54277399:G:CG600R1.000
4:54277399:G:TG600W1.000
4:54277400:G:AG600E1.000
4:54277405:G:AG602R1.000
4:54277405:G:CG602R1.000
4:54277406:G:AG602E1.000
4:54277411:T:AF604I1.000
4:54277411:T:CF604L1.000
4:54277411:T:GF604V1.000
4:54277412:T:CF604S1.000
4:54277412:T:GF604C1.000
4:54277413:T:AF604L1.000
4:54277413:T:GF604L1.000
4:54277414:G:AG605R1.000
4:54277414:G:CG605R1.000
4:54277414:G:TG605W1.000
4:54277415:G:AG605E1.000

dbSNP variants (sampled 300 via entrez): RS1000015262 (4:54274373 G>A), RS1000046285 (4:54229341 G>T), RS1000083286 (4:54245482 G>C,T), RS1000172289 (4:54294432 A>G), RS1000270553 (4:54294177 G>A,C), RS10003055 (4:54291499 T>G), RS1000315091 (4:54243748 G>A), RS1000335790 (4:54260536 A>G), RS1000342682 (4:54229195 G>T), RS1000357961 (4:54229623 G>A), RS1000386316 (4:54238948 G>T), RS1000438653 (4:54235001 A>G), RS1000444302 (4:54254626 T>C), RS10004857 (4:54289813 G>A,C), RS1000493457 (4:54288413 G>T)

Disease associations

OMIM: gene MIM:173490 | disease phenotypes: MIM:606764, MIM:175510, MIM:167000, MIM:607685, MIM:119540

GenCC curated gene-disease

DiseaseClassificationInheritance
polyps, multiple and recurrent inflammatory fibroid, gastrointestinalDefinitiveAutosomal dominant
gastrointestinal stromal tumorDefinitiveAutosomal dominant
colobomaStrongAutosomal dominant
congenital heart diseaseLimitedAutosomal dominant
isolated cleft palateLimitedUnknown

ClinGen Gene-Disease Validity (2)

Expert-panel classifications — Definitive > Strong > Moderate > Limited > Disputed > Refuted.

DiseaseClassificationInheritance
gastrointestinal stromal tumorDefinitiveAD
congenital heart diseaseLimitedAD

Mondo (12): gastrointestinal stromal tumor (MONDO:0011719), hereditary neoplastic syndrome (MONDO:0015356), polyps, multiple and recurrent inflammatory fibroid, gastrointestinal (MONDO:0008285), ovarian cancer (MONDO:0008170), idiopathic hypereosinophilic syndrome (MONDO:0011895), myeloproliferative neoplasm, unclassifiable (MONDO:0019452), colon carcinoma (MONDO:0002032), squamous cell lung carcinoma (MONDO:0005097), isolated cleft palate (MONDO:0007336), lung sarcomatoid carcinoma (MONDO:0006279), congenital heart disease (MONDO:0005453), coloboma (MONDO:0001476)

Orphanet (6): Inherited cancer-predisposing syndrome (Orphanet:140162), Gastrointestinal stromal tumor (Orphanet:44890), Rare ovarian cancer (Orphanet:213500), Idiopathic hypereosinophilic syndrome (Orphanet:3260), Chronic myeloproliferative disease, unclassifiable (Orphanet:86830), Cleft palate (Orphanet:2014)

HPO phenotypes

55 total (30 of 55 shown, HPO-id order):

HPOTerm
HP:0000006Autosomal dominant inheritance
HP:0000175Cleft palate
HP:0000202Orofacial cleft
HP:0000220Velopharyngeal insufficiency
HP:0000327Hypoplasia of the maxilla
HP:0000403Recurrent otitis media
HP:0000405Conductive hearing impairment
HP:0000689Dental malocclusion
HP:0000707Abnormality of the nervous system
HP:0000750Delayed speech and language development
HP:0000988Skin rash
HP:0000989Pruritus
HP:0001392Abnormality of the liver
HP:0001442Typified by somatic mosaicism
HP:0001611Hypernasal speech
HP:0001723Restrictive cardiomyopathy
HP:0001744Splenomegaly
HP:0001880Increased total eosinophil count
HP:0001903Anemia
HP:0002015Dysphagia
HP:0002017Nausea and vomiting
HP:0002019Constipation
HP:0002033Poor suck
HP:0002113Pulmonary infiltrates
HP:0002239Gastrointestinal hemorrhage
HP:0002240Hepatomegaly
HP:0002576Intussusception
HP:0003326Myalgia
HP:0003745Sporadic
HP:0004395Malnutrition

GWAS associations

34 associations (top):

StudyTraitp-value
GCST000136_2Height4.000000e-07
GCST000585_9Mean corpuscular volume2.000000e-29
GCST000587_9Mean corpuscular hemoglobin3.000000e-25
GCST000588_4Red blood cell count2.000000e-17
GCST001101_2Corneal curvature1.000000e-09
GCST001339_4Corneal astigmatism8.000000e-09
GCST001803_1Corneal curvature5.000000e-14
GCST002479_6Lupus nephritis in systemic lupus erythematosus5.000000e-07
GCST002502_4Corneal curvature8.000000e-09
GCST002698_1Serum VEGFR2 concentration5.000000e-25
GCST002936_29Cadmium levels1.000000e-06
GCST003264_1630Post bronchodilator FEV1/FVC ratio1.000000e-06
GCST003264_477Post bronchodilator FEV1/FVC ratio3.000000e-06
GCST003831_12Asthma1.000000e-06
GCST003833_22Adult asthma1.000000e-06
GCST004332_3Red blood cell count4.000000e-10
GCST005803_1Corneal astigmatism6.000000e-09
GCST005991_10Platelet count7.000000e-09
GCST005993_74Mean corpuscular hemoglobin6.000000e-109
GCST005994_20Hematocrit2.000000e-20
GCST005995_8Hemoglobin6.000000e-16
GCST005996_60Red blood cell count1.000000e-93
GCST006011_105Mean corpuscular volume3.000000e-126
GCST006462_24Uterine fibroids9.000000e-10
GCST006585_2488Blood protein levels1.000000e-07
GCST006976_71Macular thickness8.000000e-10
GCST007267_264Systolic blood pressure2.000000e-09
GCST007269_94Pulse pressure3.000000e-29
GCST008413_1002Core binding factor acute myeloid leukemia3.000000e-18
GCST008413_1003Core binding factor acute myeloid leukemia6.000000e-08

EFO canonical traits (11, from GWAS)

EFO IDTrait name
EFO:0004509hemoglobin measurement
EFO:0004527mean corpuscular hemoglobin
EFO:0004305erythrocyte count
EFO:0004345corneal topography
EFO:0004713FEV/FVC ratio
EFO:1002040Corneal astigmatism
EFO:0004309platelet count
EFO:0004348hematocrit
EFO:0006335systolic blood pressure
EFO:0005763pulse pressure measurement
EFO:0006939cup-to-disc ratio measurement

MeSH disease descriptors (6)

DescriptorNameTree numbers
D003103ColobomaC11.250.110; C11.270.147; C16.131.384.282
D046152Gastrointestinal Stromal TumorsC04.557.450.565.370; C06.301.371.308; C06.405.249.308
D006330Heart Defects, CongenitalC14.240.400; C14.280.400; C16.131.240.400
D009386Neoplastic Syndromes, HereditaryC04.700; C16.320.700
D010051Ovarian NeoplasmsC04.588.322.455; C12.050.351.500.056.630.705; C12.050.351.937.418.685; C12.100.250.056.630.705; C12.900.418.685; C19.344.410; C19.391.630.705
C566774Polyps, Multiple And Recurrent Inflammatory Fibroid, Gastrointestinal (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (3): CHEMBL2007 (SINGLE PROTEIN), CHEMBL2095189 (PROTEIN COMPLEX), CHEMBL4630733 (PROTEIN-PROTEIN INTERACTION)

Molecules with ChEMBL bioactivity

77 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 345,222 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

MoleculeNamePhasePatents
CHEMBL1171837PONATINIB48,955
CHEMBL1287853FEDRATINIB43,554
CHEMBL1289494TIVOZANIB44,455
CHEMBL1289601LENVATINIB48,784
CHEMBL1289926AXITINIB415,732
CHEMBL1336SORAFENIB486,060
CHEMBL1642IMATINIB MESYLATE470,143
CHEMBL1834657INFIGRATINIB PHOSPHATE4285
CHEMBL1852688INFIGRATINIB42,209
CHEMBL1946170REGORAFENIB412,678
CHEMBL2403108CERITINIB48,551
CHEMBL24828VANDETANIB442,230
CHEMBL255863NILOTINIB438,627
CHEMBL288441BOSUTINIB412,255
CHEMBL3039504NINTEDANIB ESYLATE4659
CHEMBL3813873PEXIDARTINIB43,586
CHEMBL4204794AVAPRITINIB41,306
CHEMBL4216467RIPRETINIB41,068
CHEMBL477772PAZOPANIB415,540
CHEMBL502835NINTEDANIB48,545
CHEMBL535SUNITINIB4
CHEMBL5416410DASATINIB4
CHEMBL553ERLOTINIB4
CHEMBL576982QUIZARTINIB4
CHEMBL608533MIDOSTAURIN4
CHEMBL941IMATINIB4
CHEMBL101253VATALANIB3
CHEMBL1908391MASITINIB3
CHEMBL2105728CRENOLANIB3
CHEMBL217092SARACATINIB3

Clinical evidence (CIViC)

Drug × variant × indication: 37 predictive associations from 45 curated evidence items; also 1 diagnostic.

VariantTherapyIndicationEffectLevelCIViC
PDGFRA D842VImatinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC AEID11545
PDGFRA Exon 18 MutationAvapritinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC AEID7809
PDGFRA MutationRipretinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC AEID11333
PDGFRA MutationImatinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC BEID11331 +1
PDGFRA D842VAvapritinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC BEID7479
PDGFRA D842VImatinibGastrointestinal Stromal TumorResistanceCIViC BEID15 +4
PDGFRA D842VSunitinibGastrointestinal Stromal TumorResistanceCIViC BEID4058 +2
PDGFRA Exon 18 MutationImatinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC CEID2486
PDGFRA V561DImatinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC CEID2474
FIP1L1::PDGFRA Fusion AND PDGFRA T674IImatinibMyeloid And Lymphoid Neoplasms With Eosinophilia And Abnormalities Of PDGFRA, PDGFRB, And FGFR1ResistanceCIViC CEID1446 +1
FIP1L1::PDGFRA Fusion AND PDGFRA T674IPonatinibMyeloid And Lymphoid Neoplasms With Eosinophilia And Abnormalities Of PDGFRA, PDGFRB, And FGFR1Sensitivity/ResponseCIViC DEID1779
PDGFRA AmplificationTrametinibLung Large Cell CarcinomaSensitivity/ResponseCIViC DEID12457
PDGFRA AmplificationSunitinibLung Non-small Cell CarcinomaSensitivity/ResponseCIViC DEID1771
PDGFRA AmplificationImatinib + SunitinibLung Squamous Cell CarcinomaSensitivity/ResponseCIViC DEID7989
PDGFRA D842ICrenolanibGastrointestinal Stromal TumorSensitivity/ResponseCIViC DEID43
PDGFRA D842VDasatinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC DEID4057
PDGFRA D842VCrenolanibGastrointestinal Stromal TumorSensitivity/ResponseCIViC DEID44
PDGFRA D842VBosutinibChronic Myeloid LeukemiaSensitivity/ResponseCIViC DEID7514
PDGFRA D842YCrenolanibGastrointestinal Stromal TumorSensitivity/ResponseCIViC DEID45
PDGFRA D842_I843delinsVMCrenolanibGastrointestinal Stromal TumorSensitivity/ResponseCIViC DEID47
PDGFRA G853DCrenolanib + ImatinibMelanomaSensitivity/ResponseCIViC DEID1977
PDGFRA H845YCrenolanib + ImatinibMelanomaSensitivity/ResponseCIViC DEID1976
PDGFRA I843DELImatinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC DEID1309
PDGFRA I843DELCrenolanibGastrointestinal Stromal TumorSensitivity/ResponseCIViC DEID46
PDGFRA OverexpressionDasatinibHigh Grade GliomaSensitivity/ResponseCIViC DEID7934
PDGFRA OverexpressionSunitinibRhabdomyosarcomaSensitivity/ResponseCIViC DEID7988
PDGFRA P577SImatinib + CrenolanibMelanomaSensitivity/ResponseCIViC DEID1974
PDGFRA R841KImatinib + CrenolanibMelanomaSensitivity/ResponseCIViC DEID1975
PDGFRA V561AImatinibGastrointestinal Stromal TumorSensitivity/ResponseCIViC DEID652
FIP1L1::PDGFRA Fusion AND PDGFRA T674IDasatinibMyeloid And Lymphoid Neoplasms With Eosinophilia And Abnormalities Of PDGFRA, PDGFRB, And FGFR1ResistanceCIViC DEID4420

+7 more predictive associations (showing top 30 by evidence level).

PharmGKB: 1 entry (VIP=true, CPIC=false)

PharmGKB variants

6 variants.

VariantGenesLevelScore#Clin annotsDrugs
rs35597368PDGFRA0.000
rs1800810PDGFRA0.000
rs1800812PDGFRA0.000
rs1800813PDGFRA0.000
rs2228230PDGFRA0.000
rs121908585PDGFRA0.000

GtoPdb / IUPHAR curated pharmacology

(IUPHAR/BPS Guide to Pharmacology — expert-curated)

Target class: catalytic receptor — Type III RTKs: PDGFR, CSFR, Kit, FLT3 receptor family

Most potent curated ligand interactions (25 total), top 25:

LigandActionAffinityParameter
olaratumabBinding10.4pKd
ibcasertibInhibition9.0pIC50
AC710Inhibition8.89pKd
compound 8h [PMID: 21561767]Inhibition8.89pIC50
PP121Inhibition8.7pIC50
compound 8i [PMID: 22765894]Inhibition8.7pIC50
crenolanibInhibition8.68pKd
compound 8h [PMID: 22765894]Inhibition8.52pIC50
elenestinibInhibition8.35pIC50
toceranibInhibition8.3pKi
CP-673451Inhibition8.0pIC50
quizartinibInhibition7.96pKd
ilorasertibInhibition7.96pIC50
lucitanibInhibition7.89pIC50
CHMFL-KIT-64Inhibition7.6pIC50
sitravatinibInhibition7.52pIC50
cediranibInhibition7.44pIC50
velzatinibInhibition7.3pIC50
BPR1R024Inhibition7.28pIC50
ENMD-2076Inhibition7.25pIC50
nintedanibInhibition7.23pIC50
dovitinibInhibition6.68pIC50
masitinibInhibition6.27pIC50
vimseltinibInhibition6.0pIC50
PDGF receptor tyrosine kinase inhibitor IIInhibition5.96pIC50

Binding affinities (BindingDB)

280 measured of 392 human assays (392 total across all organisms); most potent 50 below. Values come from heterogeneous assays and are not directly comparable.

LigandMeasureValuePatent
[1-(4-chloro-2,3,5,6-tetradeuteriophenyl)-2,2,2-trideuterioethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC500.016 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
1-[2-(4-methylphenyl)-5-tert-butyl-pyrazol-3-yl]-3-[4-(2-morpholin-4-ylethoxy)naphthalen-1-yl]ureaKD0.37 nM
[(1R)-1-(4-chlorophenyl)ethyl] 2,2,3,3,5,5,6,6-octadeuterio-4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC500.56 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
StaurosporineKD1.7 nM
FORETINIBIC501.9 nM
2-benzyl-5-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]-1,3,4-thiadiazoleIC502.31 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
[(1R)-1-(3,4-difluorophenyl)ethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC502.69 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
4-(6-methoxy-3,4-dihydro-2H-quinolin-1-yl)-2,6-dimethylfuro[2,3-d]pyrimidineIC503.4 nMUS-10189853: Conformationally restricted 4-substituted-2,6-dimethylfuro[2,3-d]pyrimidines as anti-tumor agents
cyclohexyl-[2-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]pyrimidin-5-yl]methanolIC505.26 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
[2-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]pyrimidin-5-yl]-phenylmethanolIC505.27 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]-N-[[4-(trifluoromethyl)phenyl]methyl]piperazine-1-sulfonamideIC505.96 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
[(1R)-1-phenylethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC507.17 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
N-[4-methyl-3-[(4-pyridin-3-ylpyrimidin-2-yl)amino]phenyl]-2-prop-2-enoyl-3,4-dihydro-1H-isoquinoline-6-carboxamideIC507.9 nMUS-8586600: Heterocyclic compounds and uses thereof
3-[(4-bromo-2,6-difluorophenyl)methoxy]-5-({[4-(pyrrolidin-1-yl)butyl]carbamoyl}amino)-1,2-thiazole-4-carboxamideIC5010.1 nMUS-9446026: Ocular formulations for drug-delivery to the posterior segment of the eye
4-[(2,5-dioxopyrrol-1-yl)methyl]-N-[4-methyl-3-[(4-pyridin-3-ylpyrimidin-2-yl)amino]phenyl]benzamideIC5010.7 nMUS-8586600: Heterocyclic compounds and uses thereof
[(1R)-1-[4-(trifluoromethoxy)phenyl]ethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5011.8 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
1-[3-fluoro-4-(trifluoromethyl)phenyl]ethyl 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5013 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
[(1R)-1-(3-fluorophenyl)ethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5016.1 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
1-(5-methyl-2-pyridinyl)ethyl 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5017.1 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
1-(6-methyl-3-pyridinyl)ethyl 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5017.3 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
N-[3-[2-[4-amino-1-(1-methylpiperidin-4-yl)pyrazolo[3,4-d]pyrimidin-3-yl]ethynyl]-4-methylphenyl]-3-(trifluoromethyl)benzamideIC5018 nMUS-10266537: 3-acetylenyl-pyrazole-pyrimidine derivative, and preparation method therefor and uses thereof
4-(ethenylsulfonylamino)-N-[4-methyl-3-[(4-pyridin-3-ylpyrimidin-2-yl)amino]phenyl]benzamideIC5018.8 nMUS-8586600: Heterocyclic compounds and uses thereof
(+/-)Bicyclo[2.2.1]hept-2-yl-(6,7-dimethoxy-quinoxalin-2-yl)-amineIC5020 nM
CHEMBL2112463IC5025 nM
[(4-fluorophenyl)-[2-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]pyrimidin-5-yl]methyl] acetateIC5026.8 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
BMS-354825KD27 nM
N-[4-methyl-3-[(4-pyridin-3-ylpyrimidin-2-yl)amino]phenyl]-4-[(prop-2-enoylamino)methyl]benzamideIC5029.8 nMUS-8586600: Heterocyclic compounds and uses thereof
(R)-[2-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]pyrimidin-5-yl]-(oxan-3-yl)methanolIC5031 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
4-[7-(1-methylpyrazol-4-yl)imidazo[1,2-b]pyridazin-3-yl]-N-[[4-(trifluoromethyl)phenyl]methyl]piperazine-1-carboxamideIC5031.7 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
4-methyl-N-[3-(4-methyl-1H-imidazol-1-yl)-5-(trifluoromethyl)phenyl]-3-[(4-pyridin-3-ylpyrimidin-2-yl)amino]benzamideIC5033 nM
[(1S)-2,2,2-trifluoro-1-phenylethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5038.4 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
[(1R)-1-(3,4-dichlorophenyl)ethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5039.1 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
4-[(4-methylpiperazin-1-yl)methyl]-N-[4-methyl-3-[(4-pyridin-3-yl-1,3-thiazol-2-yl)amino]phenyl]benzamideIC5045 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
N-[(1R)-1-(4-chlorophenyl)ethyl]-4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxamideIC5049 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
N-[(4-chlorophenyl)methyl]-4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-sulfonamideIC5050 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
3-[2-(cyclopropanecarbonylamino)-[1,3]thiazolo[5,4-b]pyridin-5-yl]-N-[4-[(4-ethylpiperazin-1-yl)methyl]-3-(trifluoromethyl)phenyl]benzamideKD57 nMUS-8765747: Fused 2-aminothiazole compounds
2-benzyl-5-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]-1,3,4-oxadiazoleIC5058.6 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]-N-[[4-(trifluoromethyl)phenyl]methyl]piperazine-1-carboxamideIC5058.9 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
[(1R)-1-phenylethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyrazin-3-yl]piperazine-1-carboxylateIC5059.5 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
(5-chlorothiophen-3-yl)methyl 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5065.3 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
N-[(4-chlorophenyl)methyl]-4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxamideIC5067 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
N-(4-methoxyphenyl)-N,2,6-trimethylfuro[2,3-d]pyrimidin-4-amineIC5068.2 nMUS-10189853: Conformationally restricted 4-substituted-2,6-dimethylfuro[2,3-d]pyrimidines as anti-tumor agents
(S)-[2-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]pyrimidin-5-yl]-(oxan-3-yl)methanolIC5069.1 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
2-ethyl-1-[2-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]pyrimidin-5-yl]butan-1-olIC5075.3 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
2-(4-fluorophenyl)-2-[2-[4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazin-1-yl]pyrimidin-5-yl]ethanolIC5079.8 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
6-(1-methylpyrazol-4-yl)-3-[4-[5-(pyridin-2-ylmethyl)-2-pyridinyl]piperazin-1-yl]pyrazolo[1,5-a]pyridineIC5080.4 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
N-[4-methyl-3-[(4-pyridin-3-ylpyrimidin-2-yl)amino]phenyl]-4-(prop-2-enoylamino)-3-(trifluoromethyl)benzamideIC5083 nMUS-8586600: Heterocyclic compounds and uses thereof
N-(3,4-dimethoxyphenyl)-N,2,6-trimethylfuro[2,3-d]pyrimidin-4-amineIC5090.3 nMUS-10189853: Conformationally restricted 4-substituted-2,6-dimethylfuro[2,3-d]pyrimidines as anti-tumor agents
[(1R)-1-(2-fluorophenyl)ethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylateIC5097.2 nMUS-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
N,2,6-trimethyl-N-(4-propan-2-yloxyphenyl)furo[2,3-d]pyrimidin-4-amineIC50102 nMUS-10189853: Conformationally restricted 4-substituted-2,6-dimethylfuro[2,3-d]pyrimidines as anti-tumor agents

ChEMBL bioactivities

1422 potent at pChembl≥5 of 1494 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChemblTypeValueUnitMolecule
10.10Ki0.07943nMCHEMBL196363
10.05IC500.09nMCHEMBL3904768
9.74IC500.18nMCHEMBL3979322
9.74IC500.18nMCHEMBL6150730
9.72IC500.19nMCHEMBL3940697
9.70IC500.2nMCHEMBL3915941
9.66IC500.22nMCHEMBL6162898
9.62IC500.24nMAVAPRITINIB
9.62IC500.24nMCHEMBL1668411
9.55IC500.28nMCHEMBL6114929
9.52IC500.3nMCHEMBL6173067
9.49IC500.32nMCHEMBL3913766
9.39Kd0.41nMCEDIRANIB
9.38IC500.418nMSTAUROSPORINE
9.37IC500.43nMSTAUROSPORINE
9.35IC500.45nMSTAUROSPORINE
9.33Kd0.47nMDASATINIB
9.31Kd0.49nMCHEMBL1908396
9.29Kd0.51nMAXITINIB
9.28IC500.53nMCHEMBL3975580
9.24IC500.58nMSTAUROSPORINE
9.24IC500.577nMSTAUROSPORINE
9.22IC500.6nMSTAUROSPORINE
9.22IC500.6nMRIPRETINIB
9.22IC500.6nMPONATINIB
9.15IC500.7nMRIPRETINIB
9.14IC500.73nMCHEMBL3642837
9.12IC500.75nMCHEMBL3950278
9.10Ki0.7943nMILORASERTIB
9.10IC500.793nMSTAUROSPORINE
9.10Kd0.79nMSUNITINIB
9.10IC500.8nMCHEMBL6177679
9.09IC500.81nMTAS-115
9.02IC500.96nMCHEMBL3985689
9.01IC500.98nMCHEMBL1173411
9.00IC501nMCHEMBL2071201
9.00IC501nMCHEMBL4216473
9.00Kd1nMCHEMBL1079545
8.96IC501.1nMPONATINIB
8.96IC501.1nMCHEMBL5176837
8.96IC501.1nMSTAUROSPORINE
8.96Kd1.1nMSU-014813
8.92IC501.2nMCHEMBL2347053
8.92IC501.2nMCHEMBL3947262
8.92IC501.2nMCHEMBL6177746
8.90Ki1.259nMCHEMBL1999414
8.89Kd1.3nMCHEMBL2206278
8.89IC501.3nMCHEMBL3955987
8.89IC501.3nMCHEMBL3895824
8.89Kd1.3nMCHEMBL4741283

PubChem BioAssay actives

931 with measured affinity, of 3238 total; 50 most potent distinct compounds. Largely complementary to BindingDB; screening values are coarse (µM, 4 dp), so sub-nM hits tie at the floor.

CompoundAssayTypeValueUnit
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-7-(1H-pyrazol-5-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0001uM
(2S,3R,4R,6R)-3-methoxy-2-methyl-4-(methylamino)-29-oxa-1,7,17-triazaoctacyclo[12.12.2.12,6.07,28.08,13.015,19.020,27.021,26]nonacosa-8,10,12,14,19,21,23,25,27-nonaen-16-one350286: Inhibition of PDGFRalphaic500.0001uM
N-[5-[(3,4-difluorophenyl)methylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0001uM
(4S,6Z,9S,10R,12E)-9,10,18-trihydroxy-16-methoxy-4-methyl-3-oxabicyclo[12.4.0]octadeca-1(14),6,12,15,17-pentaene-2,8-dione568303: Inhibition of human recombinant PDGFRalpha in cell free system after 60 minsic500.0002uM
Avapritinib2141012: Inhibition of PDGFRA (unknown origin)ic500.0002uM
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0002uM
N-[5-[2-(2,2-dimethylpyrrolidin-1-yl)ethylcarbamoyl]-2-fluorophenyl]-7-(1-methylpyrazol-4-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0002uM
N-[5-[2-(2,2-dimethylpyrrolidin-1-yl)ethylcarbamoyl]-2-methyl-3-pyridinyl]-6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0002uM
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-7-(1-methylpyrazol-4-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0003uM
4-[(4-fluoro-2-methyl-1H-indol-5-yl)oxy]-6-methoxy-7-(3-pyrrolidin-1-ylpropoxy)quinazoline625034: Binding constant for PDGFRA kinase domainkd0.0004uM
N-(2-chloro-6-methylphenyl)-2-[[6-[4-(2-hydroxyethyl)piperazin-1-yl]-2-methylpyrimidin-4-yl]amino]-1,3-thiazole-5-carboxamide;hydrate435827: Binding constant for PDGFRA kinase domainkd0.0005uM
N-[5-[(5,5-dimethyloxolan-2-yl)methylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0005uM
Axitinib625034: Binding constant for PDGFRA kinase domainkd0.0005uM
1-[4-(6,7-dimethoxyquinolin-4-yl)oxy-2-methoxyphenyl]-3-[1-(1,3-thiazol-2-yl)ethyl]urea625034: Binding constant for PDGFRA kinase domainkd0.0005uM
4-[2-fluoro-4-[(2-phenylacetyl)carbamothioylamino]phenoxy]-7-methoxy-N-methylquinoline-6-carboxamide2095663: Inhibition of wild type human PDGFRalpha transfected with CHO cells by ADP-Glo assayic500.0008uM
1-[4-[4-amino-7-[1-(2-hydroxyethyl)pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl]phenyl]-3-(3-fluorophenyl)urea673707: Inhibition of PDGFRAki0.0008uM
Sunitinib435827: Binding constant for PDGFRA kinase domainkd0.0008uM
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-7-pyridin-3-ylimidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0008uM
N-[4-[(5-tert-butyl-1,2-oxazol-3-yl)carbamoylamino]phenyl]-3-hydroxypyridine-2-carboxamide469532: Binding affinity to PDGFRalphakd0.0010uM
5-[(Z)-[6-[[3-(4-methoxyanilino)-3-oxopropanoyl]amino]-2-oxo-1H-indol-3-ylidene]methyl]-2,4-dimethyl-1H-pyrrole-3-carboxylic acid1184098: Inhibition of PDGFRalpha (unknown origin) using EAIYAAPFAKKK substrate by radioisotope-based P81 filter-binding assayic500.0010uM
N’-[(3Z)-3-[[3,5-dimethyl-4-(2-pyrrolidin-1-ylethylcarbamoyl)-1H-pyrrol-2-yl]methylidene]-2-oxo-1H-indol-6-yl]-N-(4-methoxyphenyl)propanediamide1184098: Inhibition of PDGFRalpha (unknown origin) using EAIYAAPFAKKK substrate by radioisotope-based P81 filter-binding assayic500.0010uM
N-[4-[[5-fluoro-7-(2-methoxyethoxy)quinazolin-4-yl]amino]phenyl]-2-(4-propan-2-yltriazol-1-yl)acetamide1934913: Inhibition of PDGFRalpha (unknown origin)ic500.0010uM
5-[(Z)-[6-[(4-methoxyphenyl)carbamoylamino]-2-oxo-1H-indol-3-ylidene]methyl]-4-methyl-1H-pyrrole-3-carboxylic acid491704: Inhibition of PDGFRalphaic500.0010uM
(1S,2S,3R,4R)-3-[[5-bromo-2-[(1-methylpyrazol-4-yl)amino]pyrimidin-4-yl]amino]bicyclo[2.2.1]hept-5-ene-2-carboxamide677881: Inhibition of PDGFRalpha by TR-FRET analysisic500.0010uM
N-[2-methyl-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]-3-pyridinyl]-6-(2-methylpyrazol-3-yl)pyrazolo[1,5-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0010uM
5-[(Z)-(5-fluoro-2-oxo-1H-indol-3-ylidene)methyl]-N-[(2S)-2-hydroxy-3-morpholin-4-ylpropyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide435827: Binding constant for PDGFRA kinase domainkd0.0011uM
6-[7-methoxy-6-(1-methylpyrazol-4-yl)imidazo[1,2-a]pyridin-3-yl]-N-pyrrolidin-3-ylpyridin-2-amine1894591: Inhibition of PDGFR alpha (unknown origin)ic500.0011uM
Ponatinib1716407: Binding affinity to human PDGFRalpha using poly[Glu:Tyr] (4:1) as substrate by radiometric hotspot kinase assayic500.0011uM
(3Z)-3-[[5-(2-nitrophenyl)-1H-pyrazol-4-yl]methylidene]-1H-indol-2-one739576: Inhibition of PDGFRalpha (unknown origin) after 10 mins by mobility shift assayic500.0012uM
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-6-(2-methylpyrazol-3-yl)pyrazolo[1,5-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0012uM
N-[5-[2-(2,2-dimethylpyrrolidin-1-yl)ethylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0013uM
trans-(1S,2S)-2-[[6-[(2-amino-3-chloro-4-pyridinyl)methoxy]-1,3-benzothiazol-2-yl]amino]cyclohexan-1-ol1723267: Binding affinity to human wild type PDGFRalpha (V575 to D1002 residues) expressed in mammalian expression system by kinome scan assaykd0.0013uM
5-[2-[5-[[4-[[4-(2-hydroxyethyl)piperazin-1-yl]methyl]-3-(trifluoromethyl)phenyl]carbamoyl]-2-methylphenyl]ethynyl]-N,1-dimethylimidazole-2-carboxamide601222: Inhibition of human PDGFRalpha using poly[Glu:Tyr] by Hotspot assayic500.0013uM
N-[4-[(5-tert-butyl-1,2-oxazol-3-yl)carbamoylamino]phenyl]-5-(1-ethyl-2,2,6,6-tetramethylpiperidin-4-yl)oxypyridine-2-carboxamide712315: Binding affinity to PDGFRalpha expressed in HEK-293 cellskd0.0013uM
N-[5-[2-[(2R,6S)-2,6-dimethylpiperidin-1-yl]ethylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0013uM
1-(2,3-difluorophenyl)-3-[4-(6,7-dimethoxyquinolin-4-yl)oxyphenyl]urea241088: Inhibition of platelet-derived growth factor receptor alphaic500.0016uM
N-[5-[2-[tert-butyl(ethyl)amino]ethylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresisic500.0016uM
Quizartinib526666: Binding affinity to PDGFRalphakd0.0016uM
methyl 2-hydroxy-3-[N-[4-[methyl-[2-(4-methylpiperazin-1-yl)acetyl]amino]phenyl]-C-phenylcarbonimidoyl]-1H-indole-6-carboxylate1427161: Inhibition of PDGFRalpha (unknown origin) preincubated for 10 mins followed by FAM-labeled peptide substrate addition measured after 1 hr by mobility shift assayic500.0019uM
1-[4-(6,7-dimethoxyquinolin-4-yl)oxyphenyl]-3-(3-methoxyphenyl)urea241088: Inhibition of platelet-derived growth factor receptor alphaic500.0020uM
1-[4-(6,7-dimethoxyquinolin-4-yl)oxyphenyl]-3-(4-methoxyphenyl)urea241088: Inhibition of platelet-derived growth factor receptor alphaic500.0020uM
N-[3-chloro-4-(4,6-dihydro-1H-pyrrolo[3,4-d]pyrazol-5-ylmethyl)phenyl]-3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methylbenzamide1648813: Inhibition of PDGFRalpha (unknown origin) assessed as residual activity incubated for 5 mins in presence of [gamma-33ATP] by scintillation counting based radiometry assayic500.0020uM
Imatinib767455: Inhibition of PDGFRA (unknown origin)ic500.0020uM
5-[(1-ethylpiperidin-4-yl)amino]-3-[(3-fluorophenyl)-(5-methyl-1H-imidazol-2-yl)methylidene]-1H-indol-2-one684155: Inhibition of N-terminal GST-tagged PDGFRalpha after 2 hrs by luciferase-luciferin coupled chemiluminescence assayic500.0020uM
N-[4-(1,3a,4,5,7,7a-hexahydropyrazolo[3,4-c]pyridin-6-ylmethyl)-3-chlorophenyl]-3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methylbenzamide1648813: Inhibition of PDGFRalpha (unknown origin) assessed as residual activity incubated for 5 mins in presence of [gamma-33ATP] by scintillation counting based radiometry assayic500.0021uM
4-methyl-N-[4-[(4-methylpiperazin-1-yl)methyl]-3-(trifluoromethyl)phenyl]-3-[2-(1H-pyrazolo[5,4-b]pyridin-5-yl)ethynyl]benzamide1937938: Inhibition of PDGFRalpha (unknown origin) incubated for 1.5 hrs in the presence of ATP by Z’-LYTE kinase assayic500.0021uM
N-[4-[(5-tert-butyl-1,2-oxazol-3-yl)carbamoylamino]phenyl]-5-(1-ethylpiperidin-4-yl)oxypyridine-2-carboxamide712315: Binding affinity to PDGFRalpha expressed in HEK-293 cellskd0.0023uM
Tivozanib1206830: Inhibition of recombinant PDGFRalpha (unknown origin) by cell-free assayic500.0023uM
(4S,6Z,9S,10S)-9,10,18-trihydroxy-16-methoxy-4-methyl-3-oxabicyclo[12.4.0]octadeca-1(14),6,15,17-tetraene-2,8-dione553092: Inhibition of PDGFRalpha V561D mutant by TR-FRET based LanthaScreen assayic500.0024uM
2-N-[4-(4-ethylpiperazin-1-yl)-3-methoxyphenyl]-4-N-(1H-indazol-6-yl)-5-methylpyrimidine-2,4-diamine1903974: Inhibition of PDGFRalpha (unknown origin) by caliper mobility shift assayic500.0024uM

CTD chemical–gene interactions

91 total (human), top 30 by PubMed support.

ChemicalActions (top 5)PubMed papers
Valproic Acidaffects cotreatment, increases expression, affects expression7
bisphenol Aaffects methylation, increases expression, affects cotreatment3
trichostatin Aaffects cotreatment, increases expression3
ponatinibdecreases activity, decreases phosphorylation3
Imatinib Mesylateaffects binding, increases response to substance, decreases expression, increases reaction3
Cadmium Chlorideincreases expression, increases phosphorylation, decreases reaction, decreases expression, increases abundance3
perfluorooctane sulfonic aciddecreases expression, affects expression, affects cotreatment2
monomethylarsonous acidincreases expression2
bisphenol Saffects methylation, decreases expression2
Sunitinibdecreases expression, decreases phosphorylation2
Fulvestrantincreases phosphorylation, decreases methylation, decreases reaction2
Vorinostataffects cotreatment, increases expression2
Panobinostataffects cotreatment, increases expression2
Benzo(a)pyrenedecreases expression, decreases methylation, affects methylation2
Cadmiumdecreases expression, increases abundance, increases phosphorylation, decreases reaction2
Leadincreases abundance, increases expression, decreases expression2
Phenylmercuric Acetateaffects cotreatment, increases expression2
Progesteroneaffects binding, increases phosphorylation, increases secretion, decreases expression, increases reaction2
Tretinoinincreases expression2
Aflatoxin B1decreases expression, decreases methylation2
bisphenol Faffects cotreatment, increases expression1
ammonium 2,3,3,3-tetrafluoro-2-(heptafluoropropoxy)-propanoateaffects cotreatment, affects expression1
TL8-506affects cotreatment, increases expression1
methylmercuric chlorideincreases expression1
propionaldehydeincreases expression1
pirinixic acidincreases activity, affects binding, decreases expression1
lead acetateincreases abundance, increases expression1
N(4)-hydroxycytidinedecreases expression1
2-methyl-4-isothiazolin-3-oneincreases expression1
arseniteincreases methylation1

ChEMBL screening assays

1172 unique, capped per target: 1160 binding, 8 functional, 4 admet

Representative assays (with source publication via chembl_document):

Assay IDTypeDescriptionSource paper
CHEMBL1009914BindingInhibition of PDGFRalpha D842V mutantFragment-based discovery of the pyrazol-4-yl urea (AT9283), a multitargeted kinase inhibitor with potent aurora kinase activity. — J Med Chem
CHEMBL1963805FunctionalPUBCHEM_BIOASSAY: Navigating the Kinome. (Class of assay: other) Panel member name: PDGFRAPubChem BioAssay data set
CHEMBL4044106ADMETInhibition of wild-type human partial length PDGFRA (V575 to D1002 residues) expressed in HEK293 cells assessed as residual activity at 1000 nM after 30 mins by Kinomescan method relative to control2-Oxo-3, 4-dihydropyrimido[4, 5-d]pyrimidinyl derivatives as new irreversible pan fibroblast growth factor receptor (FGFR) inhibitors. — Eur J Med Chem

Cellosaurus cell lines

28 cell lines: 20 cancer cell line, 5 induced pluripotent stem cell, 2 factor-dependent cell line, 1 transformed cell line

First 10 cell lines (id-ordered, not curated):

CellosaurusNameCategorySex
CVCL_0258EoL-1Cancer cell lineMale
CVCL_0D47NZM087Cancer cell lineMale
CVCL_0D59NZM099Cancer cell lineSex unspecified
CVCL_9973SUP-T13Cancer cell lineFemale
CVCL_A5MZUKAi007-AInduced pluripotent stem cellMale
CVCL_A5NAUKAi007-BInduced pluripotent stem cellMale
CVCL_A5NBUKAi008-AInduced pluripotent stem cellMale
CVCL_A5NCUKAi008-BInduced pluripotent stem cellMale
CVCL_A5NDUKAi008-CInduced pluripotent stem cellMale
CVCL_B0YHAbcam SH-SY5Y PDGFRA KOCancer cell lineFemale

Clinical trials (associated diseases)

600 trials via MONDO — disease-level, not drug-specific.

TrialPhaseStatusTitle
NCT00668824PHASE4UNKNOWNImproved Diagnosis of Congenital Heart Disease by Magnetic Resonance Imaging Using Vasovist
NCT01368705PHASE4COMPLETEDNitrogen Balance in Infants After Post Cardiothoracic Surgery
NCT01619982PHASE4COMPLETEDPre-operative Prophylaxis With Vancomycin and Cefazolin in Pediatric Cardiovascular Surgery Patients
NCT02122679PHASE4WITHDRAWNTranexamic Acid Effect on Platelet Aggregation Following Infant Cardiopulmonary Bypass
NCT02527811PHASE4UNKNOWNUlinastatin Injection in in Pediatric Patients Undergoing Open Heart Surgery
NCT03014700PHASE4COMPLETEDFibrinogen Concentrate vs Cryoprecipitate
NCT03408340PHASE4TERMINATEDParavertebral Nerve Blocks in Neonates
NCT03630796PHASE4UNKNOWNEffect of Sevoflurane in Postoperative Troponin I Levels in Children Undergoing Congenital Heart Defects Surgery
NCT03667703PHASE4COMPLETEDStress Ulcer Prophylaxis Versus Placebo in Critically Ill Infants With Congenital Heart Disease
NCT04453761PHASE4UNKNOWNThiamine Influenced on Substrate Energy Effectiveness in Indonesian Children Undergoing Cardiopulmonary Bypass
NCT06668389PHASE4RECRUITINGSodium-Glucose Cotransporter 2 Inhibitors for Repaired Tetralogy of Fallot Patients for Enhancement of Cardio-Pulmonary Status Trial
NCT07499154PHASE4NOT_YET_RECRUITINGPerioperative Lidocaine for Lung Protection in Infants Undergoing Cardiac Surgery
NCT00171977PHASE4COMPLETEDPost-Marketing Clinical Study of Postoperative Adjuvant Therapy With Imatinib Mesylate in Patients With Gastrointestinal Stromal Tumors (GIST)
NCT00510354PHASE4COMPLETEDTreatment of Patients With Everolimus and Imatinib Mesylate Who Have Progressive Gastro Intestinal Stromal Tumors (GIST) and Are Resistant to Imatinib Mesylate
NCT00756509PHASE4COMPLETEDTreatment of Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumors in First Line With Nilotinib
NCT00777504PHASE4UNKNOWNStudy to the Optimal Duration of Therapy With Oral Angiogenesis Inhibitors
NCT02800330PHASE4COMPLETEDThe Effects of the Proton Pump Inhibitor Esomeprazole on the Bioavailability of Regorafenib
NCT04825574PHASE4COMPLETEDStudy for Patients Previously Treated in Avapritinib Clinical Trials
NCT00000470PHASE3COMPLETEDInfant Heart Surgery: Central Nervous System Sequelae of Circulatory Arrest
NCT00000494PHASE3COMPLETEDManagement of Patent Ductus in Premature Infants
NCT01134302PHASE3UNKNOWNHybrid Versus Norwood Management Strategies in Infants Undergoing Single Ventricle Palliation
NCT01607983PHASE3WITHDRAWNEffects of Pulmonary Vasodilation Upon VA Coupling in Fontan Patients
NCT01662011PHASE3UNKNOWNApplication of Neurally Adjusted Ventilatory Assist to Children After Congenital Cardiac Surgery
NCT02320669PHASE3COMPLETEDPhase 3 Triiodothyronine Supplementation for Infants After Cardiopulmonary Bypass
NCT02615262PHASE3COMPLETEDIntraoperative Dexamethasone in Pediatric Cardiac Surgery
NCT03153137PHASE3COMPLETEDClinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects
NCT03154476PHASE3COMPLETEDRole of Sildenafil for Fontan Associated Liver Disease (SiFALD) Study
NCT04536194PHASE3COMPLETEDDopamine Versus Norepinephrine Under General Anesthesia
NCT04702373PHASE3ACTIVE_NOT_RECRUITINGTraining in Exercise Activities and Motion for Growth (TEAM 4 Growth) RCT
NCT05049590PHASE3COMPLETEDAcute Normovolemic Hemodilution in Complex Cardiac Surgery
NCT06406517PHASE3UNKNOWNComparative Effectiveness of Gadopiclenol for Evaluation of Adult Congenital Heart Anatomy and Hemodynamics
NCT06693674PHASE3RECRUITINGEffect of Sacubitril-Valsartan on Cardiac Structure and Function
NCT06955260PHASE3NOT_YET_RECRUITINGSGLT2 Inhibition With Empagliflozin in Fontan Circulatory Failure
NCT00009906PHASE3TERMINATEDComparison of Two Different Doses of STI571 in Treating Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumor
NCT00041197PHASE3COMPLETEDImatinib Mesylate in Treating Patients With Primary Gastrointestinal Stromal Tumor That Has Been Completely Removed By Surgery
NCT00075218PHASE3COMPLETEDA Study To Assess The Safety And Efficacy Of SU11248 In Patients With Gastrointestinal Stromal Tumor(GIST)
NCT00103168PHASE3COMPLETEDImatinib Mesylate or Observation Only in Treating Patients Who Have Undergone Surgery for Localized Gastrointestinal Stromal Tumor
NCT00293124PHASE3COMPLETEDOpen-label Trial of GlivecWith Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumors
NCT00324987PHASE3TERMINATEDImatinib Mesylate With or Without Bevacizumab in Treating Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumor
NCT00372567PHASE3TERMINATEDSafety And Effectiveness Of Daily Dosing With Sunitinib Or Imatinib In Patients With Gastrointestinal Stromal Tumors

About this page

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

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Sources 81

This page pulls from 81 datasets indexed by BioBTree:

alphafoldalphamissenseantibodybgeebgee_evidencebindingdbbiogrid_interactionbrendabrenda_kineticsccdscellosauruschembl_activitychembl_assaychembl_documentchembl_moleculechembl_targetciviccivic_evidenceclingen_dosageclingen_gene_validityclinical_trialsclinvarcollectrictd_gene_interactiondbsnpdepmapdiamond_similarityefoensemblentrezesm2_similarityexonfantom5_genefantom5_promotergenccgenerifgogoparentgtopdbgtopdb_interactiongwasgwas_studyhgnchpointactinterprointogenjasparmeshmimmirdbmondomsigdborphanetorthologparalogpatentpatent_compoundpdbpfampharmgkb_clinicalpharmgkb_genepharmgkb_guidelinepharmgkb_variantpubchempubchem_activitypubchem_assaypubmedreactomereactomeparentrefseqrhearnacentralscxa_expressionscxa_gene_experimentsignorspliceaistring_interactiontranscriptufeatureuniprot