CSF1R

Summary

CSF1R (colony stimulating factor 1 receptor, HGNC:2433) is a protein-coding gene on chromosome 5q32, encoding Macrophage colony-stimulating factor 1 receptor (P07333). Tyrosine-protein kinase that acts as a cell-surface receptor for CSF1 and IL34 and plays an essential role in the regulation of survival, proliferation and differentiation of hematopoietic precursor cells, especially mononuclear phagocytes, such as macrophages and monocytes.

The protein encoded by this gene is the receptor for colony stimulating factor 1, a cytokine which controls the production, differentiation, and function of macrophages. This receptor mediates most if not all of the biological effects of this cytokine. Ligand binding activates the receptor kinase through a process of oligomerization and transphosphorylation. The encoded protein is a tyrosine kinase transmembrane receptor and member of the CSF1/PDGF receptor family of tyrosine-protein kinases. Mutations in this gene have been associated with a predisposition to myeloid malignancy. The first intron of this gene contains a transcriptionally inactive ribosomal protein L7 processed pseudogene oriented in the opposite direction. Alternative splicing results in multiple transcript variants. Expression of a splice variant from an LTR promoter has been found in Hodgkin lymphoma (HL), HL cell lines and anaplastic large cell lymphoma.

Source: NCBI Gene 1436 — RefSeq curated summary.

At a glance

• Gene–disease (curated): leukoencephalopathy, diffuse hereditary, with spheroids 1 (Definitive, ClinGen) — +3 more curated relationships
• GWAS associations: 17
• Clinical variants (ClinVar): 1,025 total — 42 pathogenic, 34 likely-pathogenic
• Phenotypes (HPO): 79
• Druggable target: yes — 79 molecules with ChEMBL bioactivity
• Cancer driver (intOGen): loss-of-function (tumor-suppressor-like) across 2 cancer types
• MANE Select transcript: NM_001288705

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:2433
Approved symbol	CSF1R
Name	colony stimulating factor 1 receptor
Location	5q32
Locus type	gene with protein product
Status	Approved
Aliases	C-FMS, CSFR, CD115
Ensembl gene	ENSG00000182578
Ensembl biotype	protein_coding
OMIM	164770
Entrez	1436

Gene structure

Transcript identifiers

Ensembl transcripts: 19 — 13 protein_coding, 3 retained_intron, 2 nonsense_mediated_decay, 1 protein_coding_CDS_not_defined

ENST00000286301, ENST00000502660, ENST00000504875, ENST00000509861, ENST00000511344, ENST00000513609, ENST00000515068, ENST00000515239, ENST00000543093, ENST00000675795, ENST00000911110, ENST00000911111, ENST00000911112, ENST00000911113, ENST00000911114, ENST00000911115, ENST00000911116, ENST00000960687, ENST00000960688

RefSeq mRNA: 5 — MANE Select: NM_001288705 NM_001288705, NM_001349736, NM_001375320, NM_001375321, NM_005211

CCDS: CCDS4302

Canonical transcript exons

ENST00000675795 — 21 exons

Exon	Start	End
ENSE00001023081	150077276	150077435
ENSE00001023090	150068215	150068330
ENSE00001023092	150070456	150070571
ENSE00001023094	150069873	150070063
ENSE00001023096	150059700	150059862
ENSE00001023097	150070182	150070302
ENSE00001023101	150073301	150073493
ENSE00003479789	150080767	150081024
ENSE00003495350	150078112	150078248
ENSE00003499486	150057287	150057384
ENSE00003511616	150056219	150056341
ENSE00003547588	150061723	150061849
ENSE00003582642	150057504	150057592
ENSE00003586812	150060862	150060972
ENSE00003595569	150061491	150061595
ENSE00003623917	150080052	150080336
ENSE00003625490	150056026	150056137
ENSE00003647852	150054322	150054430
ENSE00003691987	150055237	150055336
ENSE00003902156	150086379	150086554
ENSE00003902731	150053295	150054224

Expression profiles

Bgee: expression breadth ubiquitous, 245 present calls, max score 98.10.

FANTOM5 (CAGE): breadth broad, TPM avg 23.0415 / max 958.1040, expressed in 574 samples.

FANTOM5 promoters (8 alternative TSS)

Promoter ID	TPM avg	Samples expressed
64153	20.7264	447
64154	1.2490	299
64157	0.5127	60
64158	0.2138	44
64159	0.1869	33
64156	0.0706	31
64161	0.0412	12
64160	0.0407	14

Top tissues by expression

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

Tissue	Anatomy ID	Expression score	Quality
granulocyte	CL:0000094	98.10	gold quality
monocyte	CL:0000576	98.02	gold quality
leukocyte	CL:0000738	97.90	gold quality
mononuclear cell	CL:0000842	97.88	gold quality
spleen	UBERON:0002106	97.23	gold quality
right coronary artery	UBERON:0001625	95.43	gold quality
placenta	UBERON:0001987	94.86	gold quality
inferior vagus X ganglion	UBERON:0005363	94.75	gold quality
pigmented layer of retina	UBERON:0001782	94.72	gold quality
retina	UBERON:0000966	94.70	gold quality
gall bladder	UBERON:0002110	94.66	gold quality
vermiform appendix	UBERON:0001154	94.25	gold quality
lymph node	UBERON:0000029	92.58	gold quality
decidua	UBERON:0002450	92.39	gold quality
C1 segment of cervical spinal cord	UBERON:0006469	92.33	gold quality
spinal cord	UBERON:0002240	92.15	gold quality
synovial joint	UBERON:0002217	91.49	gold quality
oocyte	CL:0000023	91.13	gold quality
ventral tegmental area	UBERON:0002691	90.99	gold quality
superior vestibular nucleus	UBERON:0007227	90.69	gold quality
blood	UBERON:0000178	90.24	gold quality
secondary oocyte	CL:0000655	90.23	gold quality
subthalamic nucleus	UBERON:0001906	90.17	gold quality
superficial temporal artery	UBERON:0001614	89.66	gold quality
caecum	UBERON:0001153	89.58	gold quality
tibial nerve	UBERON:0001323	89.31	gold quality
smooth muscle tissue	UBERON:0001135	89.14	gold quality
dorsal root ganglion	UBERON:0000044	89.01	gold quality
sural nerve	UBERON:0015488	88.93	gold quality
right adrenal gland cortex	UBERON:0035827	88.68	gold quality

Single-cell (SCXA)

Detected in 19 experiment(s), a significant marker in 18.

Experiment	Marker?	Max mean expression
E-GEOD-84465	yes	1800.35
E-MTAB-6678	yes	1586.56
E-MTAB-9906	yes	1188.05
E-GEOD-149689	yes	343.41
E-MTAB-6701	yes	64.58
E-HCAD-10	yes	62.42
E-MTAB-8410	yes	42.58
E-CURD-122	yes	39.65
E-HCAD-35	yes	31.97
E-GEOD-134144	yes	27.82
E-MTAB-10287	yes	24.72
E-CURD-88	yes	20.19
E-CURD-112	yes	17.13
E-MTAB-8498	yes	12.25
E-MTAB-9067	yes	10.55

Regulation

Is transcription factor: yes

Downstream targets (CollecTRI)

2 targets.

Target	Regulation
RUNX1	Repression
SOCS1	Activation

Upstream regulators (CollecTRI, top): ATF4, BCL6, CEBPB, CEBPD, EGR1, ELF1, EPAS1, ETS2, EWSR1, FOS, FOXP1, GFI1, HDLBP, HSF1, IRF6, JDP2, JUN, MECOM, MYC, NR3C1, PAX5, RUNX1, RUNX2, SPDEF, SPI1

miRNA regulators (miRDB)

41 targeting CSF1R, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-6870-5P	99.99	68.55	2115
HSA-MIR-34A-5P	99.99	71.21	1784
HSA-MIR-449A	99.99	71.05	1776
HSA-MIR-4723-5P	99.97	68.70	2034
HSA-MIR-5698	99.97	68.49	2029
HSA-MIR-7111-5P	99.97	68.48	2062
HSA-MIR-34C-5P	99.97	70.45	1577
HSA-MIR-449B-5P	99.97	70.26	1580
HSA-MIR-22-3P	99.93	68.13	917
HSA-MIR-218-5P	99.93	72.22	2103
HSA-MIR-4728-5P	99.85	69.39	4718
HSA-MIR-765	99.84	68.24	2442
HSA-MIR-6785-5P	99.82	68.68	4428
HSA-MIR-6842-5P	99.80	67.54	1587
HSA-MIR-7110-5P	99.80	67.84	1712
HSA-MIR-3934-3P	99.76	65.51	1351
HSA-MIR-11181-3P	99.75	66.38	2205
HSA-MIR-149-3P	99.72	68.22	3963
HSA-MIR-6883-5P	99.69	68.05	3785
HSA-MIR-766-5P	99.47	67.91	2225
HSA-MIR-1275	99.47	67.90	2749
HSA-MIR-20A-3P	99.44	69.10	1575
HSA-MIR-155-5P	99.35	70.16	1509
HSA-MIR-6852-5P	99.17	66.69	2073
HSA-MIR-6734-3P	99.15	66.27	1627
HSA-MIR-455-3P	98.94	67.68	878
HSA-MIR-6894-5P	98.70	63.78	809
HSA-MIR-5581-3P	98.55	70.31	1161
HSA-MIR-4450	98.26	68.35	725
HSA-MIR-4436B-3P	98.25	65.26	1494

Literature-anchored findings (GeneRIF, showing 40)

• AP-1 proteins, GR and associated co-factors regulate transcription from the c-fms first promoter in breast carcinoma cells. (PMID:11891846)
• upregulated significantly in acute myeloid leukemia patients with a white blood cell count higher than 30 x 10(9)/L cells (PMID:12031912)
• DNA binding to and transactivation of the M-CSF receptor promoter were deficient in acute myeloid leukemia PU.1 mutants that affected the DNA-binding domain, contibuting to the block in differentiation in AML. (PMID:12130514)
• CSF-1 and its receptor are regulated by 1,25(OH)(2)D(3) and its analogue tacalcitol in human monocytes which parallels the inhibition of differentiation into dendritic cells without altering survival (PMID:12372416)
• These observations correlate CSF-1R expression with changes in the growth and development of the normal and neoplastic prostate (PMID:12381783)
• C-fms expression correlates with monocytic differentiation in PML-RAR alpha+ acute promyelocytic leukemias (PMID:12529666)
• In t(8;21) leukaemic cells expressing the aberrant fusion protein AML1-ETO, we demonstrate that this protein is part of a transcription factor complex binding to extended sequences of the c-FMS intronic regulatory region rather than the promoter. (PMID:12773394)
• findings of co-expression of KIT and/or FMS with their respective ligands implies these receptors might contribute to leukemogenesis in some patients with AML through autocrine, paracrine, or intracrine interactive stimulation. (PMID:14654075)
• Lower expression of CSF1R is associated with acute myeloid leukemia-M2 and higher expression of CSF1R is associated with acute myeloid leukemia-M5 (PMID:14738146)
• data suggest that colony-stimulating factor receptor-1R (CSF-1R) disrupts cell adhesion by uncoupling adherens junction complexes from the cytoskeleton and promoting cadherin internalization through a Src-dependent mechanism (PMID:15117969)
• CSF-1-mediated wild-type (WT)-CSF-1R phosphorylation was not markedly affected by Src-family kinases (SFK) inhibition, indicating that lack of SFK binding is not responsible for diminished Y559F phosphorylation. (PMID:15297464)
• HIV-1 Nef interferes with M-CSF receptor signaling through Hck activation and thereby inhibits M-CSF functions in monocytes/macrophages. (PMID:15626739)
• In c-fms transgenic mice it is determined that although the c-fms promoter is inactive in dendritic cell (DC) precursors, it is up-regulated in all DC subsets during differentiation (PMID:16034075)
• the CSF1R mutation of Asp-802 to Val confers resistance to kinase inhibitor imatinib (PMID:16170366)
• Inhibitory activity of lead compounds and drug candidates, such as ABT-869, against the CSF-1R protein in situ. (PMID:16648572)
• No evidence that CSF1R 2033T variant was a major risk factor for Crohn’s disease in New Zealand. (PMID:16708222)
• Creation of a conditional allele of the Csf1r by placing LoxP sites around Exon 5 of the Csf1r gene in mice. (PMID:16823860)
• 2.7 A resolution crystal structure of the cytosolic domain of c-Fms that comprises the kinase domain and the juxtamembrane domain (PMID:17292918)
• method revealed the presence of an activated colony-stimulating factor 1 receptor (CSF1R) kinase in the acute megakaryoblastic leukemia (AMKL) cell line MKPL-1, essential for the growth and survival of MKPL-1 cells. (PMID:17360941)
• tyrosine 559 is the major mediator of receptor activation and cell death, intracellular signaling, and cell proliferation and that the tyrosine residues at positions 697 and 807 play lesser roles in these events (PMID:17420255)
• We thus conclude that Ox-LDL induces monocyte differentiation to macrophages in vivo and this phenomenon involves activation of the M-CSF-receptor. (PMID:17675037)
• The results suggest that the growth factor M-CSF might have an important role in ovarian function. (PMID:17880962)
• Nef perturbs the intracellular maturation and the trafficking of nascent Fms, through a unique mechanism that required both the activation of Hck and the aberrant spatial regulation of the active Hck (PMID:17893228)
• c-KIT but not CSF1R mutations play a role in the leukemogenesis of childhood CBF-AML. (PMID:17960171)
• analysis of the transcriptional regulation of the Colony-Stimulating Factor 1 Receptor (csf1r) gene during hematopoiesis [review] (PMID:17981568)
• CSF-1 receptor RIPping can be activated by various intracellular signal transduction pathways and that RIPping is likely to play an important role during macrophage activation. (PMID:18294963)
• Expression of M-CSF, CSF-1R and CD3 is a significant prognostic factor in primary prostatic cancers by predicting the development of metastases. (PMID:18510570)
• VEGF expression was statistically correlated to c-fms and COX-2 expression in high-grade CIN. (PMID:18565574)
• c-Fms signals are blocked by imatinib mesylate inhibition of osteoclasts, which suppresses bone metastases of breast cancer (PMID:18814279)
• mutant CSF1R was constitutively phosphorylated, but rapidly dephosphorylated on exposure to imatinib, in cell line derived from a patient with an atypical myeloproliferative neoplasms, as being responsive to imatinib. (PMID:18971950)
• CSF1R may be a critical factor facilitating hTERT immortalization of epithelial cells. (PMID:18997822)
• Calorimetric data indicate that M-CSF cannot dimerize FMS without receptor-receptor interactions mediated by FMS domains D4 and D5 (PMID:19017797)
• findings suggest that HuR plays a supportive role for c-fms in breast cancer progression by binding a 69-nt element in its 3’UTR, thus regulating its expression (PMID:19151756)
• Genes for Fra2, Id2, and CSF1-receptor are deregulated, regardless of whether the in anaplastic large cell lymphoma contains the t(2;5). (PMID:19321746)
• study evaluated the prevalence of the activating mutations of C-FMS in a series of 85 patients with chronic myelomonocytic leukemia; no mutations in codons 301 and 969 of the C-FMS gene were detected in any of the 85 samples (PMID:19375163)
• CSF1R plays a relevant role in clear cell renal cell carcinoma carcinogenesis (PMID:19377443)
• This finding establishes an intriguing link between the pathogenesis of Nef and a newly emerging concept that the Golgi-localized Src kinases regulate the Golgi function. (PMID:19585521)
• These results reveal a novel proteolytic regulation of M-CSF receptor expression in monocytes to control M-CSF signaling and monocytic differentiation into macrophage/osteoclast-lineage cells or dendritic cells. (PMID:19762488)
• c-Fms plays a central role in the pathogenesis of rheumatoid arthritis by mediating the differentiation and priming of monocyte lineage cells (PMID:20181277)
• The different spatiotemporal expression of IL-34 and CSF-1 allows for complementary activation of the CSF-1R in developing and adult tissues. (PMID:20504948)

Cross-species orthologs

5 orthologs

Organism	Symbol	Gene ID
danio_rerio	csf1rb	ENSDARG00000053624
danio_rerio	si:zfos-1069f5.1	ENSDARG00000100890
danio_rerio	csf1ra	ENSDARG00000102986
mus_musculus	Csf1r	ENSMUSG00000024621
rattus_norvegicus	Csf1r	ENSRNOG00000018414

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), PDGFRA (ENSG00000134853), 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)

Protein

Protein identifiers

Macrophage colony-stimulating factor 1 receptor — P07333 (reviewed: P07333)

Alternative names: CSF-1 receptor, Proto-oncogene c-Fms

All UniProt accessions (4): D6RGW1, E9PEK4, P07333, H0YAL5

UniProt curated annotations — full annotation on UniProt →

Function. Tyrosine-protein kinase that acts as a cell-surface receptor for CSF1 and IL34 and plays an essential role in the regulation of survival, proliferation and differentiation of hematopoietic precursor cells, especially mononuclear phagocytes, such as macrophages and monocytes. Promotes the release of pro-inflammatory chemokines in response to IL34 and CSF1, and thereby plays an important role in innate immunity and in inflammatory processes. Plays an important role in the regulation of osteoclast proliferation and differentiation, the regulation of bone resorption, and is required for normal bone and tooth development. Required for normal male and female fertility, and for normal development of milk ducts and acinar structures in the mammary gland during pregnancy. Promotes reorganization of the actin cytoskeleton, regulates formation of membrane ruffles, cell adhesion and cell migration, and promotes cancer cell invasion. Activates several signaling pathways in response to ligand binding, including the ERK1/2 and the JNK pathway. Phosphorylates PIK3R1, PLCG2, GRB2, SLA2 and CBL. Activation of PLCG2 leads to the production of the cellular signaling molecules diacylglycerol and inositol 1,4,5-trisphosphate, that then lead to the activation of protein kinase C family members, especially PRKCD. Phosphorylation of PIK3R1, the regulatory subunit of phosphatidylinositol 3-kinase, leads to activation of the AKT1 signaling pathway. Activated CSF1R also mediates activation of the MAP kinases MAPK1/ERK2 and/or MAPK3/ERK1, and of the SRC family kinases SRC, FYN and YES1. Activated CSF1R transmits signals both via proteins that directly interact with phosphorylated tyrosine residues in its intracellular domain, or via adapter proteins, such as GRB2. Promotes activation of STAT family members STAT3, STAT5A and/or STAT5B. Promotes tyrosine phosphorylation of SHC1 and INPP5D/SHIP-1. Receptor signaling is down-regulated by protein phosphatases, such as INPP5D/SHIP-1, that dephosphorylate the receptor and its downstream effectors, and by rapid internalization of the activated receptor. In the central nervous system, may play a role in the development of microglia macrophages.

Subunit / interactions. Interacts with INPPL1/SHIP2 and THOC5. Monomer. Homodimer. Interacts with CSF1 and IL34. Interaction with dimeric CSF1 or IL34 leads to receptor homodimerization. Interacts (tyrosine phosphorylated) with PLCG2 (via SH2 domain). Interacts (tyrosine phosphorylated) with PIK3R1 (via SH2 domain). Interacts (tyrosine phosphorylated) with FYN, YES1 and SRC (via SH2 domain). Interacts (tyrosine phosphorylated) with CBL, GRB2 and SLA2.

Subcellular location. Cell membrane.

Tissue specificity. Expressed in bone marrow and in differentiated blood mononuclear cells.

Post-translational modifications. Autophosphorylated in response to CSF1 or IL34 binding. Phosphorylation at Tyr-561 is important for normal down-regulation of signaling by ubiquitination, internalization and degradation. Phosphorylation at Tyr-561 and Tyr-809 is important for interaction with SRC family members, including FYN, YES1 and SRC, and for subsequent activation of these protein kinases. Phosphorylation at Tyr-699 and Tyr-923 is important for interaction with GRB2. Phosphorylation at Tyr-723 is important for interaction with PIK3R1. Phosphorylation at Tyr-708 is important for normal receptor degradation. Phosphorylation at Tyr-723 and Tyr-809 is important for interaction with PLCG2. Phosphorylation at Tyr-969 is important for interaction with CBL. Dephosphorylation by PTPN2 negatively regulates downstream signaling and macrophage differentiation. Ubiquitinated. Becomes rapidly polyubiquitinated after autophosphorylation, leading to its degradation.

Disease relevance. Aberrant expression of CSF1 or CSF1R can promote cancer cell proliferation, invasion and formation of metastases. Overexpression of CSF1 or CSF1R is observed in a significant percentage of breast, ovarian, prostate, and endometrial cancers. Aberrant expression of CSF1 or CSF1R may play a role in inflammatory diseases, such as rheumatoid arthritis, glomerulonephritis, atherosclerosis, and allograft rejection. Leukoencephalopathy, hereditary diffuse, with spheroids 1 (HDLS1) [MIM:221820] An autosomal dominant adult-onset rapidly progressive neurodegenerative disorder characterized by variable behavioral, cognitive, and motor changes. Patients often die of dementia within 6 years of onset. Brain imaging shows patchy abnormalities in the cerebral white matter, predominantly affecting the frontal and parietal lobes. The disease is caused by variants affecting the gene represented in this entry. Brain abnormalities, neurodegeneration, and dysosteosclerosis (BANDDOS) [MIM:618476] An autosomal recessive disease with variable manifestations. Main features are brain malformations with calcifying leukoencephalopathy, progressive neurodegeneration, and bone sclerotic features. The age at onset ranges from infancy to early adulthood. Neurologic features include loss of previous motor and language skills, cognitive impairment, spasticity, and focal seizures. Brain imaging shows periventricular white matter abnormalities and calcifications, large cisterna magna or Dandy-Walker malformation, and sometimes agenesis of the corpus callosum. 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. CSF1 or IL34 binding leads to dimerization and activation by autophosphorylation on tyrosine residues. Inhibited by imatinib/STI-571 (Gleevec), dasatinib, sunitinib/SU11248, lestaurtinib/CEP-701, midostaurin/PKC-412, Ki20227, linifanib/ABT-869, Axitinib/AG013736, sorafenib/BAY 43-9006 and GW2580.

Domain organisation. The juxtamembrane domain functions as autoinhibitory region. Phosphorylation of tyrosine residues in this region leads to a conformation change and activation of the kinase. The activation loop plays an important role in the regulation of kinase activity. Phosphorylation of tyrosine residues in this region leads to a conformation change and activation of the kinase.

Induction. Up-regulated by glucocorticoids.

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

Isoforms (2)

UniProt ID	Names	Canonical?
P07333-1	1	yes
P07333-2	2

RefSeq proteins (5): NP_001275634, NP_001336665, NP_001362249, NP_001362250, NP_005202 (=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR000719	Prot_kinase_dom	Domain
IPR001245	Ser-Thr/Tyr_kinase_cat_dom	Domain
IPR001824	Tyr_kinase_rcpt_3_CS	Conserved_site
IPR003598	Ig_sub2	Domain
IPR003599	Ig_sub	Domain
IPR007110	Ig-like_dom	Domain
IPR008266	Tyr_kinase_AS	Active_site
IPR011009	Kinase-like_dom_sf	Homologous_superfamily
IPR013151	Immunoglobulin_dom	Domain
IPR013783	Ig-like_fold	Homologous_superfamily
IPR017441	Protein_kinase_ATP_BS	Binding_site
IPR020635	Tyr_kinase_cat_dom	Domain
IPR030658	CSF-1_receptor	Family
IPR036179	Ig-like_dom_sf	Homologous_superfamily
IPR050122	RTK	Family

Pfam: PF00047, 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.

Substrate	Km (mM)	Measurements
ATP	0.0011–0.129	4
AC-DYFE-6-CHLORO-W-NHME	0.0051	1
AC-DYFGW-NHME	0.07	1
YFEW	0.232	1

Catalyzed reactions (Rhea), 1 shown:

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

UniProt features (175 total): strand 55, sequence variant 37, helix 22, glycosylation site 11, modified residue 9, turn 8, domain 6, mutagenesis site 5, disulfide bond 4, sequence conflict 4, region of interest 3, binding site 2, topological domain 2, splice variant 2, signal peptide 1, chain 1, compositionally biased region 1, active site 1, transmembrane region 1

Structure

Experimental structures (PDB)

26 structures.

PDB	Method	Resolution (Å)
8JOT	X-RAY DIFFRACTION	1.69
6T2W	X-RAY DIFFRACTION	1.7
2I1M	X-RAY DIFFRACTION	1.8
6IG8	X-RAY DIFFRACTION	1.8
2I0Y	X-RAY DIFFRACTION	1.9
8CGC	X-RAY DIFFRACTION	1.93
3DPK	X-RAY DIFFRACTION	1.95
3BEA	X-RAY DIFFRACTION	2.02
3KRJ	X-RAY DIFFRACTION	2.1
6N33	X-RAY DIFFRACTION	2.25
8W1L	X-RAY DIFFRACTION	2.26
3KRL	X-RAY DIFFRACTION	2.4
3LCD	X-RAY DIFFRACTION	2.5
4LIQ	X-RAY DIFFRACTION	2.6
6WXJ	X-RAY DIFFRACTION	2.62
2OGV	X-RAY DIFFRACTION	2.7
7TNH	X-RAY DIFFRACTION	2.7
4R7I	X-RAY DIFFRACTION	2.75
2I0V	X-RAY DIFFRACTION	2.8
7MFC	X-RAY DIFFRACTION	2.8
4R7H	X-RAY DIFFRACTION	2.8
4WRL	X-RAY DIFFRACTION	2.8
4HW7	X-RAY DIFFRACTION	2.9
4DKD	X-RAY DIFFRACTION	3
3LCO	X-RAY DIFFRACTION	3.4
4WRM	X-RAY DIFFRACTION	6.85

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-P07333-F1	78.08	0.47

Antibody-complex structures (SAbDab): 1 — 4LIQ

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 (1): 778 (proton acceptor)

Ligand- & substrate-binding residues (2): 588–596; 616

Post-translational modifications (9): 546, 561, 699, 708, 713, 723, 809, 923, 969

Disulfide bonds (4): 42–84, 127–177, 224–278, 419–485

Glycosylation sites (11): 45, 73, 153, 240, 275, 302, 335, 353, 412, 428, 480

Mutagenesis-validated functional residues (5):

Position	Phenotype
301	constitutive kinase activity.
708	impairs degradation of activated csf1r.
802	constitutive kinase activity. loss of inhibition by imatinib.
809	reduced kinase activity. reduced interaction with src, fyn and yes1.
969	abolishes down-regulation of activated csf1r.

Function

Pathways and Gene Ontology

Reactome pathways

3 pathways

ID	Pathway
R-HSA-449836	Other interleukin signaling
R-HSA-8853884	Transcriptional Regulation by VENTX
R-HSA-9680350	Signaling by CSF1 (M-CSF) in myeloid cells

MSigDB gene sets: 596 (showing top): GOBP_MYELOID_CELL_DIFFERENTIATION, GSE45365_NK_CELL_VS_CD8A_DC_MCMV_INFECTION_DN, FERRANDO_TAL1_NEIGHBORS, GOBP_MORPHOGENESIS_OF_AN_EPITHELIUM, CREL_01, GOBP_REGULATION_OF_PROTEIN_TYROSINE_KINASE_ACTIVITY, GOBP_REGULATION_OF_LEUKOCYTE_PROLIFERATION, GOBP_EPITHELIUM_DEVELOPMENT, GOBP_MAMMARY_GLAND_MORPHOGENESIS, GOBP_GLAND_MORPHOGENESIS, MCLACHLAN_DENTAL_CARIES_UP, GOBP_REGULATION_OF_CELL_MORPHOGENESIS, GOBP_MYELOID_LEUKOCYTE_MIGRATION, GOBP_CELL_CHEMOTAXIS, GOBP_REGULATION_OF_PHOSPHORYLATION

GO Biological Process (53): positive regulation of protein phosphorylation (GO:0001934), response to ischemia (GO:0002931), inflammatory response (GO:0006954), signal transduction (GO:0007165), cell surface receptor protein tyrosine kinase signaling pathway (GO:0007169), axon guidance (GO:0007411), cell population proliferation (GO:0008283), positive regulation of cell population proliferation (GO:0008284), negative regulation of cell population proliferation (GO:0008285), regulation of cell shape (GO:0008360), positive regulation of macrophage chemotaxis (GO:0010759), cell migration (GO:0016477), peptidyl-tyrosine phosphorylation (GO:0018108), cytokine-mediated signaling pathway (GO:0019221), olfactory bulb development (GO:0021772), forebrain neuron differentiation (GO:0021879), hemopoiesis (GO:0030097), monocyte differentiation (GO:0030224), macrophage differentiation (GO:0030225), osteoclast differentiation (GO:0030316), positive regulation of cell migration (GO:0030335), ruffle organization (GO:0031529), positive regulation of chemokine production (GO:0032722), regulation of actin cytoskeleton organization (GO:0032956), cellular response to macrophage colony-stimulating factor stimulus (GO:0036006), macrophage colony-stimulating factor signaling pathway (GO:0038145), positive regulation of tyrosine phosphorylation of STAT protein (GO:0042531), negative regulation of apoptotic process (GO:0043066), regulation of MAPK cascade (GO:0043408), host-mediated activation of viral process (GO:0044794), innate immune response (GO:0045087), regulation of bone resorption (GO:0045124), cell-cell junction maintenance (GO:0045217), protein autophosphorylation (GO:0046777), positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction (GO:0051897), mammary gland duct morphogenesis (GO:0060603), positive regulation of protein tyrosine kinase activity (GO:0061098), microglial cell proliferation (GO:0061518), positive regulation of ERK1 and ERK2 cascade (GO:0070374), cellular response to cytokine stimulus (GO:0071345)

GO Molecular Function (14): protein tyrosine kinase activity (GO:0004713), macrophage colony-stimulating factor receptor activity (GO:0005011), ATP binding (GO:0005524), growth factor binding (GO:0019838), protein phosphatase binding (GO:0019903), cytokine binding (GO:0019955), protein homodimerization activity (GO:0042803), nucleotide binding (GO:0000166), protein kinase activity (GO:0004672), transmembrane receptor protein tyrosine kinase activity (GO:0004714), protein binding (GO:0005515), kinase activity (GO:0016301), transferase activity (GO:0016740), metal ion binding (GO:0046872)

GO Cellular Component (6): nucleoplasm (GO:0005654), plasma membrane (GO:0005886), cell surface (GO:0009986), signaling receptor complex (GO:0043235), CSF1-CSF1R complex (GO:1990682), membrane (GO:0016020)

Reactome top-level categories

Rollup of top-3 pathways:

Category	Pathways
Signaling by Interleukins	1
Generic Transcription Pathway	1
Cytokine Signaling in Immune system	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
myeloid leukocyte differentiation	3
cellular anatomical structure	3
protein phosphorylation	2
cellular process	2
cell population proliferation	2
regulation of cell population proliferation	2
mononuclear cell differentiation	2
protein binding	2
protein-containing complex	2
regulation of protein phosphorylation	1
positive regulation of protein modification process	1
positive regulation of phosphorylation	1
response to stress	1
defense response	1
cell communication	1
signaling	1
regulation of cellular process	1
cellular response to stimulus	1
enzyme-linked receptor protein signaling pathway	1
axonogenesis	1
neuron projection guidance	1
positive regulation of cellular process	1
negative regulation of cellular process	1
regulation of cell morphogenesis	1
regulation of biological quality	1
positive regulation of leukocyte chemotaxis	1
regulation of macrophage chemotaxis	1
macrophage chemotaxis	1
regulation of granulocyte chemotaxis	1
positive regulation of macrophage migration	1
cell motility	1
peptidyl-tyrosine modification	1
cell surface receptor signaling pathway	1
cellular response to cytokine stimulus	1
olfactory lobe development	1
anatomical structure development	1
forebrain generation of neurons	1
central nervous system neuron differentiation	1
cell development	1
protein kinase activity	1

Protein interactions and networks

STRING

4243 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
CSF1R	CSF1	P09603	999
CSF1R	IL34	Q6ZMJ4	998
CSF1R	TNFSF11	O14788	987
CSF1R	CSF2	P04141	974
CSF1R	CSF3	P09919	873
CSF1R	CXCL12	P48061	866
CSF1R	SPI1	P17947	859
CSF1R	CCL2	P13500	849
CSF1R	KITLG	P21583	847
CSF1R	IL3	P08700	838
CSF1R	CCL5	P13501	821
CSF1R	TYROBP	O43914	816
CSF1R	TREM2	Q9NZC2	805
CSF1R	ITGAM	P11215	803
CSF1R	ITGAX	P20702	799

IntAct

59 interactions, top by confidence:

A	B	Type	Score
CSF1	CSF1R	psi-mi:“MI:0407”(direct interaction)	0.920
CSF1R	CSF1	psi-mi:“MI:0407”(direct interaction)	0.920
IL34	CSF1R	psi-mi:“MI:0407”(direct interaction)	0.790
CSF1R	EPHB2	psi-mi:“MI:0914”(association)	0.500
EPHA7	CSF1R	psi-mi:“MI:0915”(physical association)	0.500
CSF1R	EPHB2	psi-mi:“MI:0915”(physical association)	0.500
Plcg2	CSF1R	psi-mi:“MI:0915”(physical association)	0.480
Plcg2	CSF1R	psi-mi:“MI:0914”(association)	0.480
CSF1R	CSF1R	psi-mi:“MI:0407”(direct interaction)	0.440
CSF1R	Csf1	psi-mi:“MI:0407”(direct interaction)	0.440

BioGRID (90): CSF1R (Affinity Capture-Western), SOCS1 (Two-hybrid), PIK3R2 (Reconstituted Complex), CSF1R (Co-localization), CSF1R (Co-localization), GRB2 (Two-hybrid), CTDSP1 (Two-hybrid), CTDSP2 (Two-hybrid), KIT (Affinity Capture-Western), KIT (Biochemical Activity), CSF1R (Biochemical Activity), PIK3CA (Reconstituted Complex), PIK3CA (Two-hybrid), CSF1R (Affinity Capture-Western), CSF1R (Protein-peptide)

ESM2 similar proteins: A0JM20, F1LW30, O73875, O73878, P00545, P07333, P11362, P13369, P16092, P18460, P21709, P21803, P21804, P22182, P22455, P22607, P29317, P54755, P54756, P54757, P54759, P54761, P55144, P55146, P57097, Q03142, Q04589, Q06418, Q06806, Q12866, Q15375, Q1KL86, Q498D6, Q60629, Q60750, Q60805, Q61772, Q61851, Q6UXZ4, Q8K1S2

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

27 interactions.

A	Effect	B	Mechanism
CSF1R	up-regulates	CSF1R	phosphorylation
CSF1R	down-regulates	CSF1R	phosphorylation
“pazopanib hydrochloride”	down-regulates	CSF1R	“chemical inhibition”
CSF1	“up-regulates activity”	CSF1R	binding
IL34	“up-regulates activity”	CSF1R	binding
CSF1R	up-regulates	PLCG2
CSF1R	up-regulates	ERK1/2
CSF1R	“up-regulates quantity by expression”	SOCS1	“transcriptional regulation”
pazopanib	“down-regulates activity”	CSF1R	“chemical inhibition”
sunitinib	“down-regulates activity”	CSF1R	“chemical inhibition”
HDLBP	“down-regulates quantity by repression”	CSF1R	“transcriptional regulation”
FOXP1	“down-regulates quantity by repression”	CSF1R	“transcriptional regulation”
CSF1R	“up-regulates quantity by stabilization”	CTNND1	phosphorylation
5-(3-Methoxy-4-((4-methoxybenzyl)oxy)benzyl)pyrimidine-2,4-diamine	“down-regulates activity”	CSF1R	“chemical inhibition”
1-(4-((6,7-Dimethoxyquinolin-4-yl)oxy)-2-methoxyphenyl)-3-(1-(thiazol-2-yl)ethyl)urea	“down-regulates activity”	CSF1R	“chemical inhibition”
“JNJ-28312141 free base”	“down-regulates activity”	CSF1R	“chemical inhibition”
linifanib	“down-regulates activity”	CSF1R	“chemical inhibition”

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 30 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:

Pathway	Partners	Fold	FDR
Signaling by CSF1 (M-CSF) in myeloid cells	5	91.1×	6e-07

GO biological processes:

GO term	Partners	Fold	FDR
protein stabilization	6	20.1×	9e-05
positive regulation of gene expression	7	13.6×	9e-05
negative regulation of apoptotic process	5	8.7×	5e-03

Disease & clinical

Cancer significance

From intOGen — cancer-driver classification: loss-of-function (tumor-suppressor-like) across 2 cancer types — BRCA, THYM.

Clinical variants and AI predictions

ClinVar

1025 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	42
Likely pathogenic	34
Uncertain significance	413
Likely benign	317
Benign	105

Top pathogenic / likely-pathogenic (30)

Variant ID	HGVS	Classification
1029946	NM_001288705.3(CSF1R):c.1620T>A (p.Tyr540Ter)	Pathogenic
120322	NM_001288705.3(CSF1R):c.2060dup (p.Ser688fs)	Pathogenic
120323	NM_001288705.3(CSF1R):c.2442+1G>T	Pathogenic
120324	NM_001288705.3(CSF1R):c.2342C>A (p.Ala781Glu)	Pathogenic
1322164	NM_001288705.3(CSF1R):c.2026C>T (p.Arg676Ter)	Pathogenic
1356688	NM_001288705.3(CSF1R):c.161dup (p.Ser55fs)	Pathogenic
1444078	NM_001288705.3(CSF1R):c.1276del (p.Gln426fs)	Pathogenic
1458272	NM_001288705.3(CSF1R):c.2539G>A (p.Glu847Lys)	Pathogenic
1483886	NM_001288705.3(CSF1R):c.1876G>T (p.Glu626Ter)	Pathogenic
1488995	NM_001288705.3(CSF1R):c.1100_1103del (p.Ser367fs)	Pathogenic
1499735	NM_001288705.3(CSF1R):c.2370_2377del (p.His790fs)	Pathogenic
162109	NM_001288705.3(CSF1R):c.1699del (p.Thr567fs)	Pathogenic
162111	NM_001288705.3(CSF1R):c.2330G>A (p.Arg777Gln)	Pathogenic
162117	NM_001288705.3(CSF1R):c.2655-2A>G	Pathogenic
162118	NM_001288705.3(CSF1R):c.2342C>T (p.Ala781Val)	Pathogenic
162126	NM_005211.3(CSF1R):c.2467C>T	Pathogenic
1685676	NM_001288705.3(CSF1R):c.2498C>A (p.Thr833Lys)	Pathogenic
2024481	NM_001288705.3(CSF1R):c.834C>A (p.Cys278Ter)	Pathogenic
2052730	NM_001288705.3(CSF1R):c.242_264del (p.Thr81fs)	Pathogenic
2194866	NM_001288705.3(CSF1R):c.1924C>T (p.Gln642Ter)	Pathogenic
2230074	NM_001288705.3(CSF1R):c.554del (p.Lys185fs)	Pathogenic
2575888	NM_001288705.3(CSF1R):c.2471C>T (p.Pro824Leu)	Pathogenic
2703602	NM_001288705.3(CSF1R):c.173G>A (p.Trp58Ter)	Pathogenic
2763158	NM_001288705.3(CSF1R):c.2056dup (p.Ser686fs)	Pathogenic
2790058	NM_001288705.3(CSF1R):c.1047del (p.Lys350fs)	Pathogenic
29810	NM_001288705.3(CSF1R):c.2624T>C (p.Met875Thr)	Pathogenic
29812	NM_001288705.3(CSF1R):c.1754-2A>G	Pathogenic
29814	NM_001288705.3(CSF1R):c.2509G>T (p.Asp837Tyr)	Pathogenic
3006756	NM_001288705.3(CSF1R):c.2117del (p.Lys706fs)	Pathogenic
3026935	NM_001288705.3(CSF1R):c.1682_1683insAA (p.Tyr561Ter)	Pathogenic

SpliceAI

4213 predictions. Top by Δscore:

Variant	Effect	Δscore
5:150054220:TAGTC:T	acceptor_gain	1.0000
5:150054221:AGTC:A	acceptor_gain	1.0000
5:150054222:GTC:G	acceptor_gain	1.0000
5:150054223:TC:T	acceptor_gain	1.0000
5:150054224:CC:C	acceptor_gain	1.0000
5:150054224:CCT:C	acceptor_loss	1.0000
5:150054225:C:CC	acceptor_gain	1.0000
5:150054231:G:C	acceptor_gain	1.0000
5:150054231:G:GC	acceptor_gain	1.0000
5:150054317:CTCA:C	donor_loss	1.0000
5:150054318:TCA:T	donor_loss	1.0000
5:150054319:CACCC:C	donor_loss	1.0000
5:150054320:A:AC	donor_gain	1.0000
5:150054320:AC:A	donor_gain	1.0000
5:150054321:C:CC	donor_gain	1.0000
5:150054321:CC:C	donor_gain	1.0000
5:150054321:CCCG:C	donor_gain	1.0000
5:150054330:T:A	donor_gain	1.0000
5:150054430:TCTGG:T	acceptor_loss	1.0000
5:150054431:C:CA	acceptor_loss	1.0000
5:150054431:C:CC	acceptor_gain	1.0000
5:150055235:A:AC	donor_gain	1.0000
5:150055236:C:CC	donor_gain	1.0000
5:150055236:CAT:C	donor_gain	1.0000
5:150055236:CATA:C	donor_gain	1.0000
5:150055239:A:AC	donor_gain	1.0000
5:150055239:ATT:A	donor_gain	1.0000
5:150055332:CAGCC:C	acceptor_gain	1.0000
5:150055334:GCC:G	acceptor_gain	1.0000
5:150055335:CC:C	acceptor_gain	1.0000

AlphaMissense

0 scored. Top likely-pathogenic:

dbSNP variants (sampled 300 via entrez): RS1000009490 (5:150108627 G>A), RS1000057293 (5:150110977 C>A,T), RS1000063045 (5:150070389 G>A,C), RS1000063587 (5:150095092 C>A,T), RS1000248291 (5:150109980 C>G), RS1000260405 (5:150070680 T>C), RS1000351780 (5:150078895 T>C), RS1000414437 (5:150053840 G>A,C,T), RS1000442814 (5:150065207 A>G), RS1000492429 (5:150076600 A>G), RS1000518857 (5:150053791 T>C), RS1000595624 (5:150084242 A>T), RS1000634351 (5:150071832 T>TGGCCAGAAAG), RS1000688062 (5:150058864 G>T), RS1000868076 (5:150080433 C>T)

Disease associations

OMIM: gene MIM:164770 | disease phenotypes: MIM:618476, MIM:221820, MIM:123100

GenCC curated gene-disease

Disease	Classification	Inheritance
hereditary diffuse leukoencephalopathy with axonal spheroids and pigmented glia	Definitive	Autosomal dominant
brain abnormalities, neurodegeneration, and dysosteosclerosis	Definitive	Autosomal recessive
leukoencephalopathy, diffuse hereditary, with spheroids 1	Strong	Autosomal dominant
early-onset calcifying leukoencephalopathy-skeletal dysplasia	Supportive	Autosomal recessive

ClinGen Gene-Disease Validity (1)

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

Disease	Classification	Inheritance
leukoencephalopathy, diffuse hereditary, with spheroids 1	Definitive	AD

Mondo (12): brain abnormalities, neurodegeneration, and dysosteosclerosis (MONDO:0032772), leukoencephalopathy, diffuse hereditary, with spheroids 1 (MONDO:0800027), leukoencephalopathy, hereditary diffuse, with spheroids (MONDO:0030796), CSF1R-related disorder (MONDO:0100632), parkinsonian disorder (MONDO:0021095), neurodegenerative disease (MONDO:0005559), craniosynostosis (MONDO:0015469), cerebral arterial disease (MONDO:0006693), Alzheimer disease (MONDO:0004975), frontotemporal dementia (MONDO:0017276), (MONDO:0009096), early-onset calcifying leukoencephalopathy-skeletal dysplasia (MONDO:0034143)

Orphanet (5): Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (Orphanet:313808), Craniosynostosis (Orphanet:1531), Early-onset autosomal dominant Alzheimer disease (Orphanet:1020), Frontotemporal dementia (Orphanet:282), NON RARE IN EUROPE: Alzheimer disease (Orphanet:238616)

HPO phenotypes

79 total (30 of 79 shown, HPO-id order):

HPO	Term
HP:0000006	Autosomal dominant inheritance
HP:0000007	Autosomal recessive inheritance
HP:0000238	Hydrocephalus
HP:0000256	Macrocephaly
HP:0000286	Epicanthus
HP:0000414	Bulbous nose
HP:0000505	Visual impairment
HP:0000527	Long eyelashes
HP:0000639	Nystagmus
HP:0000648	Optic atrophy
HP:0000708	Atypical behavior
HP:0000716	Depression
HP:0000727	Frontal lobe dementia
HP:0000746	Delusion
HP:0000748	Inappropriate laughter
HP:0000768	Pectus carinatum
HP:0000926	Platyspondyly
HP:0001249	Intellectual disability
HP:0001250	Seizure
HP:0001251	Ataxia
HP:0001257	Spasticity
HP:0001260	Dysarthria
HP:0001263	Global developmental delay
HP:0001268	Mental deterioration
HP:0001274	Agenesis of corpus callosum
HP:0001288	Gait disturbance
HP:0001300	Parkinsonism
HP:0001305	Dandy-Walker malformation
HP:0001321	Cerebellar hypoplasia
HP:0001347	Hyperreflexia

GWAS associations

17 associations (top):

Study	Trait	p-value
GCST004608_16	Granulocyte percentage of myeloid white cells	8.000000e-21
GCST004608_17	Granulocyte percentage of myeloid white cells	2.000000e-19
GCST004609_55	Monocyte percentage of white cells	3.000000e-20
GCST004609_56	Monocyte percentage of white cells	8.000000e-25
GCST004625_15	Monocyte count	1.000000e-12
GCST004625_16	Monocyte count	4.000000e-23
GCST006585_859	Blood protein levels	8.000000e-06
GCST012146_11	Hemoglobin levels	2.000000e-06
GCST90002389_268	Lymphocyte percentage of white cells	2.000000e-10
GCST90002393_104	Monocyte count	5.000000e-67
GCST90002393_105	Monocyte count	6.000000e-12
GCST90002393_106	Monocyte count	2.000000e-63
GCST90002394_28	Monocyte percentage of white cells	2.000000e-19
GCST90002394_29	Monocyte percentage of white cells	9.000000e-10
GCST90002394_30	Monocyte percentage of white cells	2.000000e-84
GCST90002399_428	Neutrophil percentage of white cells	1.000000e-17
GCST90011899_118	Aspartate aminotransferase levels	1.000000e-09

EFO canonical traits (7, from GWAS)

EFO ID	Trait name
EFO:0007997	granulocyte percentage of myeloid white cells
EFO:0007989	monocyte percentage of leukocytes
EFO:0005091	monocyte count
EFO:0004509	hemoglobin measurement
EFO:0007993	lymphocyte percentage of leukocytes
EFO:0007990	neutrophil percentage of leukocytes
EFO:0004736	aspartate aminotransferase measurement

MeSH disease descriptors (7)

Descriptor	Name	Tree numbers
D000544	Alzheimer Disease	C10.228.140.380.100; C10.574.945.249; F03.615.400.100
D002539	Cerebral Arterial Diseases	C10.228.140.300.510.200; C14.907.253.560.200
D003398	Craniosynostoses	C05.116.099.370.894.232; C05.660.207.240; C05.660.906.364; C16.131.621.207.240; C16.131.621.906.364
D057180	Frontotemporal Dementia	C10.228.140.380.266.299; C10.574.950.300.299; C18.452.845.800.300.299; F03.615.400.380.299
D019636	Neurodegenerative Diseases	C10.574
D020734	Parkinsonian Disorders	C10.228.140.079.862; C10.228.662.600
C580150	Hereditary Diffuse Leukoencephalopathy with Spheroids (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (1): CHEMBL1844 (SINGLE PROTEIN)

Molecules with ChEMBL bioactivity

79 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 402,715 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

Molecule	Name	Phase	Patents
CHEMBL1171837	PONATINIB	4	8,955
CHEMBL1287853	FEDRATINIB	4	3,554
CHEMBL1289926	AXITINIB	4	15,732
CHEMBL1336	SORAFENIB	4	86,060
CHEMBL1421	DASATINIB ANHYDROUS	4	55,003
CHEMBL1567	SUNITINIB MALATE	4	161
CHEMBL180022	NERATINIB	4	9,404
CHEMBL1873475	IBRUTINIB	4	7,994
CHEMBL1983268	ENTRECTINIB	4	3,510
CHEMBL2035187	PACRITINIB	4	3,345
CHEMBL24828	VANDETANIB	4	42,230
CHEMBL255863	NILOTINIB	4	38,627
CHEMBL288441	BOSUTINIB	4	12,255
CHEMBL3301607	FILGOTINIB	4	2,905
CHEMBL3545311	BRIGATINIB	4	5,634
CHEMBL3813873	PEXIDARTINIB	4	3,586
CHEMBL477772	PAZOPANIB	4	15,540
CHEMBL502835	NINTEDANIB	4	8,545
CHEMBL535	SUNITINIB	4	79,020
CHEMBL5416410	DASATINIB	4	655
CHEMBL576982	QUIZARTINIB	4
CHEMBL601719	CRIZOTINIB	4
CHEMBL608533	MIDOSTAURIN	4
CHEMBL941	IMATINIB	4
CHEMBL101253	VATALANIB	3
CHEMBL1879463	DACTOLISIB	3
CHEMBL1908391	MASITINIB	3
CHEMBL223360	LINIFANIB	3
CHEMBL276711	SEMAXANIB	3
CHEMBL377300	BRIVANIB	3

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

PharmGKB variants

1 variants.

Variant	Genes	Level	Score	#Clin annots	Drugs
rs55712339	CSF1R, PDGFRB		0.00	0

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 (40 total), top 25:

Ligand	Action	Affinity	Parameter
axatilimab	Binding	11.02	pKd
LY3022855	Binding	10.05	pEC50
elzovantinib	Inhibition	9.92	pIC50
emactuzumab	Binding	9.54	pKd
BPR1R024	Inhibition	9.28	pIC50
AMG820	Binding	9.26	pKd
compound 29 [PMID: 31934767]	Inhibition	9.15	pIC50
cabiralizumab	Binding	9.0	pKd
AC710	Inhibition	8.8	pKd
IACS-9439	Inhibition	8.77	pIC50
GR-389988	Inhibition	8.7	pIC50
Ki-20227	Inhibition	8.7	pIC50
GW-2580	Inhibition	8.66	pKd
linifanib	Inhibition	8.52	pIC50
dovitinib	Inhibition	8.52	pIC50
ilorasertib	Inhibition	8.52	pIC50
JNJ-28312141	Inhibition	8.49	pKd
edicotinib	Inhibition	8.49	pIC50
sotuletinib	Inhibition	8.47	pKd
segigratinib	Inhibition	8.42	pIC50
cerdulatinib	Inhibition	8.3	pIC50
velzatinib	Inhibition	8.26	pIC50
AZD6495	Inhibition	8.22	pIC50
ibcasertib	Inhibition	8.15	pIC50
ARRY-382	Inhibition	8.05	pIC50

Binding affinities (BindingDB)

809 measured of 920 human assays (935 total across all organisms); most potent 50 below. Values come from heterogeneous assays and are not directly comparable.

Ligand	Measure	Value	Patent
1-ethyl-N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-6-oxopyrimidine-5-carboxamide	IC50	0.003 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-[[1-(difluoromethyl)pyrazol-3-yl]amino]-4-pyridinyl]oxy]-2-pyridinyl]-4-oxo-7,8-dihydro-6H-pyrrolo[1,2-a]pyrimidine-3-carboxamide	IC50	0.003 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-6-oxo-1-propan-2-ylpyrimidine-5-carboxamide	IC50	0.004 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-N-[5-[[2-[(1-methylpyrazol-3-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-6-oxopyrimidine-5-carboxamide	IC50	0.005 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-[(2-methylpyrazol-3-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-4-oxo-7,8-dihydro-6H-pyrrolo[1,2-a]pyrimidine-3-carboxamide	IC50	0.005 nM	US-12435062: Amide compounds and uses thereof
1-methyl-N-[5-[[2-[(2-methylpyrazol-3-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-2-oxopyridine-3-carboxamide	IC50	0.005 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-6-oxo-N-[5-[[2-(pyridin-2-ylamino)-4-pyridinyl]oxy]-2-pyridinyl]pyrimidine-5-carboxamide	IC50	0.005 nM	US-12435062: Amide compounds and uses thereof
5-fluoro-1,6-dimethyl-N-[5-[[2-[(2-methylpyrazol-3-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-2-oxopyridine-3-carboxamide	IC50	0.005 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-N-[5-[[2-[(2-methylpyrazol-3-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-6-oxopyrimidine-5-carboxamide	IC50	0.006 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-[(1-methylpyrazol-3-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-4-oxo-7,8-dihydro-6H-pyrrolo[1,2-a]pyrimidine-3-carboxamide	IC50	0.007 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-[[1-(difluoromethyl)pyrazol-3-yl]amino]-4-pyridinyl]oxy]-2-pyridinyl]-1,2-dimethyl-6-oxopyrimidine-5-carboxamide	IC50	0.007 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-3-oxo-2-propan-2-ylpyridazine-4-carboxamide	IC50	0.008 nM	US-12435062: Amide compounds and uses thereof
5-fluoro-1,6-dimethyl-N-[5-[[2-[(1-methylpyrazol-4-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-2-oxopyridine-3-carboxamide	IC50	0.008 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-[(1-methylpyrazol-4-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-4-oxo-7,8-dihydro-6H-pyrrolo[1,2-a]pyrimidine-3-carboxamide	IC50	0.009 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-N-[5-[[2-[(1-methylpyrazol-4-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-6-oxopyrimidine-5-carboxamide	IC50	0.009 nM	US-12435062: Amide compounds and uses thereof
2-cyclopropyl-1-methyl-N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-6-oxopyrimidine-5-carboxamide	IC50	0.009 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-(1-methylpyrazol-3-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2-oxo-1-propan-2-ylpyridine-3-carboxamide	IC50	0.01 nM	US-12435062: Amide compounds and uses thereof
1-methyl-N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-6-oxo-2-propan-2-ylpyrimidine-5-carboxamide	IC50	0.011 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-(1-ethylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-1,2-dimethyl-6-oxopyrimidine-5-carboxamide	IC50	0.012 nM	US-12435062: Amide compounds and uses thereof
1-methyl-N-[5-[[2-[(1-methylpyrazol-3-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-2-oxopyridine-3-carboxamide	IC50	0.012 nM	US-12435062: Amide compounds and uses thereof
1-methyl-N-[5-[[2-(1-methylpyrazol-3-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2-oxoquinoline-3-carboxamide	IC50	0.014 nM	US-12435062: Amide compounds and uses thereof
1,3-dimethyl-N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2,4-dioxopyrimidine-5-carboxamide	IC50	0.016 nM	US-12435062: Amide compounds and uses thereof
N-[5-[(2-imidazo[1,2-a]pyridin-6-yl-4-pyridinyl)oxy]-2-pyridinyl]-1,2-dimethyl-6-oxopyrimidine-5-carboxamide	IC50	0.016 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-N-(5-((2-(1-methyl-1H-pyrazol-4-yl)pyridin-4-yl)methyl)pyridin-2-yl)-6-oxo-1,6-dihydropyrimidine-5-carboxamide	IC50	0.019 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-N-[5-[[2-(2-methyl-1,3-thiazol-5-yl)-4-pyridinyl]oxy]-2-pyridinyl]-6-oxopyrimidine-5-carboxamide	IC50	0.02 nM	US-12435062: Amide compounds and uses thereof
N-(5-((2-(1H-pyrazol-4-yl)pyridin-4-yl)oxy)pyridin-2-yl)-1,2-dimethyl-6-oxo-1,6-dihydropyrimidine-5-carboxamide	IC50	0.021 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-[1-(difluoromethyl)pyrazol-4-yl]-4-pyridinyl]oxy]-2-pyridinyl]-1,2-dimethyl-6-oxopyrimidine-5-carboxamide	IC50	0.023 nM	US-12435062: Amide compounds and uses thereof
5-chloro-N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2-oxo-1-propan-2-ylpyridine-3-carboxamide	IC50	0.024 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-6-oxo-N-[5-[(2-pyridin-4-yl-4-pyridinyl)oxy]-2-pyridinyl]pyrimidine-5-carboxamide	IC50	0.027 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-(2-methylpyrazol-3-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2-oxo-1-propan-2-ylpyridine-3-carboxamide	IC50	0.032 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-6-oxo-N-[5-[[2-(1-propan-2-ylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]pyrimidine-5-carboxamide	IC50	0.033 nM	US-12435062: Amide compounds and uses thereof
1-methyl-N-[5-[[2-[(1-methylpyrazol-4-yl)amino]-4-pyridinyl]oxy]-2-pyridinyl]-2-oxopyridine-3-carboxamide	IC50	0.033 nM	US-12435062: Amide compounds and uses thereof
1-(difluoromethyl)-N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2-oxopyridine-3-carboxamide	IC50	0.045 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-N-[5-[[2-(2-methyl-1,3-thiazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-6-oxopyrimidine-5-carboxamide	IC50	0.052 nM	US-12435062: Amide compounds and uses thereof
N-[5-[(2-imidazo[1,2-a]pyridin-7-yl-4-pyridinyl)oxy]-2-pyridinyl]-1,2-dimethyl-6-oxopyrimidine-5-carboxamide	IC50	0.054 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-N-[5-[[2-(5-methylfuran-2-yl)-4-pyridinyl]oxy]-2-pyridinyl]-6-oxopyrimidine-5-carboxamide	IC50	0.055 nM	US-12435062: Amide compounds and uses thereof
1,2-dimethyl-6-oxo-N-[5-[(2-phenyl-4-pyridinyl)oxy]-2-pyridinyl]pyrimidine-5-carboxamide	IC50	0.056 nM	US-12435062: Amide compounds and uses thereof
4-methyl-N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-3-oxopyrazine-2-carboxamide	IC50	0.069 nM	US-12435062: Amide compounds and uses thereof
N-[5-[[2-(4-fluorophenyl)-4-pyridinyl]oxy]-2-pyridinyl]-1,2-dimethyl-6-oxopyrimidine-5-carboxamide	IC50	0.078 nM	US-12435062: Amide compounds and uses thereof
3-[[3-methoxy-4-[(4-methoxyphenyl)methoxy]phenyl]methyl]-6-(1-methylpyrazol-4-yl)imidazo[4,5-b]pyridin-2-amine	IC50	0.1 nM	US-9067914: Tropomyosin-related kinase (TRK) inhibitors
N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-3-oxo-4-propan-2-ylpyrazine-2-carboxamide	IC50	0.121 nM	US-12435062: Amide compounds and uses thereof
N-[2-methoxy-4-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]phenyl]-1,2-dimethyl-6-oxopyrimidine-5-carboxamide	IC50	0.135 nM	US-12435062: Amide compounds and uses thereof
6-(6-dimethylphosphoryl-3-pyridinyl)-3-[[3-methoxy-4-[(4-methoxyphenyl)methoxy]phenyl]methyl]imidazo[4,5-b]pyridin-2-amine	IC50	0.2 nM	US-9067914: Tropomyosin-related kinase (TRK) inhibitors
3-[[3-methoxy-4-[[6-(trifluoromethyl)-3-pyridinyl]methoxy]phenyl]methyl]-6-(1-methylpyrazol-4-yl)imidazo[4,5-b]pyridin-2-amine	IC50	0.2 nM	US-9067914: Tropomyosin-related kinase (TRK) inhibitors
3-[[4-[(6-cyclopropyl-3-pyridinyl)methoxy]-3-methoxyphenyl]methyl]-6-(1-methylpyrazol-4-yl)imidazo[4,5-b]pyridin-2-amine	IC50	0.2 nM	US-9067914: Tropomyosin-related kinase (TRK) inhibitors
3-[[3-methoxy-4-[(2-methyl-1,3-thiazol-4-yl)methoxy]phenyl]methyl]-6-(1-methylpyrazol-4-yl)imidazo[4,5-b]pyridin-2-amine	IC50	0.2 nM	US-9067914: Tropomyosin-related kinase (TRK) inhibitors
3-[[3-methoxy-4-[[4-(trifluoromethoxy)phenyl]methoxy]phenyl]methyl]-6-(1-methylpyrazol-4-yl)imidazo[4,5-b]pyridin-2-amine	IC50	0.2 nM	US-9067914: Tropomyosin-related kinase (TRK) inhibitors
3-[[3-methoxy-4-[(6-propan-2-yl-3-pyridinyl)methoxy]phenyl]methyl]-6-(1-methylpyrazol-4-yl)imidazo[4,5-b]pyridin-2-amine	IC50	0.2 nM	US-9067914: Tropomyosin-related kinase (TRK) inhibitors
3-[[3-methoxy-4-[[2-(trifluoromethyl)-1,3-thiazol-4-yl]methoxy]phenyl]methyl]-6-(1-methylpyrazol-4-yl)imidazo[4,5-b]pyridin-2-amine	IC50	0.2 nM	US-9067914: Tropomyosin-related kinase (TRK) inhibitors
4-[4-[methyl(oxan-4-ylmethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]benzenesulfonamide	IC50	0.2 nM	US-20250353857: COMPOUND ACTIVE AS INHIBITOR OF COLONY STIMULATION FACTOR-1 RECEPTOR (CSF-1 R)

ChEMBL bioactivities

3794 potent at pChembl≥5 of 3834 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
9.70	IC50	0.2	nM	CHEMBL5415149
9.52	IC50	0.3	nM	CHEMBL438533
9.52	IC50	0.3	nM	CHEMBL489684
9.52	IC50	0.3	nM	CHEMBL5404686
9.52	IC50	0.3	nM	CHEMBL5434318
9.52	IC50	0.3	nM	CHEMBL5432649
9.52	IC50	0.3	nM	CHEMBL5439126
9.52	IC50	0.3	nM	CHEMBL5595214
9.52	IC50	0.3	nM	CHEMBL5556952
9.52	IC50	0.3	nM	CHEMBL5563269
9.52	IC50	0.3	nM	CHEMBL5569998
9.52	IC50	0.3	nM	CHEMBL5569733
9.52	IC50	0.3	nM	CHEMBL5572754
9.52	IC50	0.3	nM	CHEMBL1171604
9.50	Ki	0.3162	nM	CHEMBL273187
9.50	Ki	0.3162	nM	CHEMBL404366
9.40	IC50	0.4	nM	CHEMBL402337
9.40	IC50	0.4	nM	CHEMBL492809
9.40	IC50	0.4	nM	CHEMBL489881
9.40	IC50	0.4	nM	CHEMBL521580
9.40	IC50	0.4	nM	CHEMBL5417607
9.40	IC50	0.4	nM	CHEMBL5425883
9.40	IC50	0.4	nM	CHEMBL5405206
9.40	IC50	0.4	nM	CHEMBL5430348
9.40	IC50	0.4	nM	CHEMBL5411617
9.40	IC50	0.4	nM	CHEMBL5574791
9.40	IC50	0.4	nM	CHEMBL5579705
9.40	IC50	0.4	nM	CHEMBL5572057
9.40	IC50	0.4	nM	CHEMBL5572766
9.40	IC50	0.4	nM	CHEMBL5574054
9.40	IC50	0.4	nM	CHEMBL3349454
9.36	IC50	0.44	nM	CHEMBL3086320
9.30	IC50	0.5	nM	CHEMBL4084391
9.30	IC50	0.5	nM	CHEMBL451575
9.30	IC50	0.5	nM	CHEMBL509044
9.30	IC50	0.5	nM	CHEMBL489298
9.30	IC50	0.5	nM	CHEMBL5407395
9.30	Kd	0.5	nM	CHEMBL5415149
9.30	IC50	0.5	nM	CHEMBL5406924
9.30	IC50	0.5	nM	CHEMBL5428404
9.30	IC50	0.5	nM	CHEMBL5583977
9.30	IC50	0.5	nM	CHEMBL5573448
9.30	IC50	0.5	nM	CHEMBL1170784
9.30	IC50	0.5	nM	CHEMBL1170786
9.29	IC50	0.51	nM	CHEMBL3086320
9.29	IC50	0.51	nM	CHEMBL4282165
9.28	IC50	0.53	nM	CHEMBL4847875
9.26	IC50	0.55	nM	CHEMBL6015818
9.24	Kd	0.58	nM	DASATINIB
9.24	IC50	0.57	nM	DASATINIB

PubChem BioAssay actives

1787 with measured affinity, of 3116 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.

Compound	Assay	Type	Value	Unit
2-[4-[6-[methyl(oxan-4-ylmethyl)amino]-7H-purin-8-yl]phenyl]ethanol	1970713: Inhibition of CSF1R (unknown origin) using FRET-peptide as substrate incubated in presence of ATP by FRET based Z-LYTE assay	ic50	0.0002	uM
5-cyano-N-[2-(4-methylpiperidin-1-yl)-4-piperidin-4-ylphenyl]furan-2-carboxamide	331271: Inhibition of FMS	ic50	0.0003	uM
6-(4-aminophenyl)-N-methyl-N-(oxan-4-ylmethyl)-7H-pyrrolo[2,3-d]pyrimidin-4-amine	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
4-[4-[methyl-[(3-methylphenyl)methyl]amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]benzoic acid	1989543: Inhibition of CSF1R (unknown origin) in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
N-(cyclohexylmethyl)-6-(3,6-dihydro-2H-pyran-4-yl)-N-methyl-7H-pyrrolo[2,3-d]pyrimidin-4-amine	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
[4-[6-[methyl(oxan-4-ylmethyl)amino]-7H-purin-8-yl]phenyl]methanol	1970713: Inhibition of CSF1R (unknown origin) using FRET-peptide as substrate incubated in presence of ATP by FRET based Z-LYTE assay	ic50	0.0003	uM
4-[4-[methyl(oxan-4-ylmethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]benzenesulfonamide	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
[4-[4-[benzyl(trideuteriomethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]methanol	1989539: Inhibition of CSF1R (530 to 910 residues) (unknown origin)	ic50	0.0003	uM
2-[4-[4-[methyl(oxan-4-ylmethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]ethanol	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
3-[4-[4-[methyl(oxan-4-ylmethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]oxetan-3-ol	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
2-[[[6-[4-(hydroxymethyl)phenyl]-7H-pyrrolo[2,3-d]pyrimidin-4-yl]-methylamino]methyl]phenol	1989539: Inhibition of CSF1R (530 to 910 residues) (unknown origin)	ic50	0.0003	uM
4-[4-[methyl(oxan-4-ylmethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenol	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
N-methyl-6-[4-(morpholin-4-ylmethyl)phenyl]-N-(oxan-4-ylmethyl)-7H-pyrrolo[2,3-d]pyrimidin-4-amine	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
[4-[4-[(2-fluorophenyl)methyl-methylamino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]methanol	1989539: Inhibition of CSF1R (530 to 910 residues) (unknown origin)	ic50	0.0003	uM
3-[4-[4-[methyl-[(3-methylphenyl)methyl]amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]propanoic acid	1989543: Inhibition of CSF1R (unknown origin) in presence of ATP by Z-LYTE based FRET assay	ic50	0.0003	uM
5-cyano-N-[2-(4,4-dimethylcyclohexen-1-yl)-4-(1,3-dimethyl-2-oxo-1,3-diazinan-5-yl)phenyl]-1H-imidazole-2-carboxamide	489881: Inhibition of human FMS by fluorescence polarization	ic50	0.0003	uM
8-(4-ethylphenyl)-N-methoxy-2-[4-(1-methylpiperidin-4-yl)anilino]-5-oxopyrido[2,3-d]pyrimidine-6-carboxamide	1798844: FMS Enzyme Inhibition Assay from Article 10.1021/jm801406h: “Pyrido[2,3-d]pyrimidin-5-ones: A Novel Class of Antiinflammatory Macrophage Colony-Stimulating Factor-1 Receptor Inhibitors (dagger).”	ic50	0.0003	uM
2-[4-(6,7-dimethoxyquinolin-4-yl)oxy-2-methoxyphenyl]-N-(5-ethyl-1H-pyrazol-3-yl)acetamide	660687: Inhibition of CSF-1R	ic50	0.0003	uM
5-cyano-N-[2-(4-methylcyclohexen-1-yl)-4-piperidin-4-ylphenyl]-1H-imidazole-2-carboxamide	331271: Inhibition of FMS	ic50	0.0004	uM
4-[4-[methyl(oxan-4-ylmethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]benzoic acid	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
6-(cyclohexen-1-yl)-N-methyl-N-(oxan-4-ylmethyl)-7H-pyrrolo[2,3-d]pyrimidin-4-amine	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
6-[4-[2-[2-(2-methoxyethoxy)ethoxy]ethoxy]phenyl]-N-methyl-N-[(3-methylphenyl)methyl]-7H-pyrrolo[2,3-d]pyrimidin-4-amine	1989543: Inhibition of CSF1R (unknown origin) in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
2-fluoro-4-[4-[methyl-[(3-methylphenyl)methyl]amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]benzoic acid	1989543: Inhibition of CSF1R (unknown origin) in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
N-[[4-[4-[benzyl(methyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]-2-fluorophenyl]methyl]-N’,N’-dimethylethane-1,2-diamine	1989539: Inhibition of CSF1R (530 to 910 residues) (unknown origin)	ic50	0.0004	uM
6-(3,6-dihydro-2H-pyran-4-yl)-N-methyl-N-(oxan-4-ylmethyl)-7H-pyrrolo[2,3-d]pyrimidin-4-amine	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
6-(3-aminophenyl)-N-methyl-N-(oxan-4-ylmethyl)-7H-pyrrolo[2,3-d]pyrimidin-4-amine	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
5-[4-[4-[methyl-[(3-methylphenyl)methyl]amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]pentanoic acid	1989543: Inhibition of CSF1R (unknown origin) in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
4-[4-[methyl-[(3-methylphenyl)methyl]amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]benzenesulfonamide	1989543: Inhibition of CSF1R (unknown origin) in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
6-(4-methoxyphenyl)-N-methyl-N-(oxan-4-ylmethyl)-7H-pyrrolo[2,3-d]pyrimidin-4-amine	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0004	uM
5-cyano-N-[4-(2-cyanoimino-1,3-dimethyl-1,3-diazinan-5-yl)-2-(4,4-dimethylcyclohexen-1-yl)phenyl]-1H-imidazole-2-carboxamide	489881: Inhibition of human FMS by fluorescence polarization	ic50	0.0004	uM
8-(2,3-dihydro-1H-inden-5-yl)-N-methoxy-2-[4-(1-methylpiperidin-4-yl)anilino]-5-oxopyrido[2,3-d]pyrimidine-6-carboxamide	1798844: FMS Enzyme Inhibition Assay from Article 10.1021/jm801406h: “Pyrido[2,3-d]pyrimidin-5-ones: A Novel Class of Antiinflammatory Macrophage Colony-Stimulating Factor-1 Receptor Inhibitors (dagger).”	ic50	0.0004	uM
8-(3-ethylphenyl)-N-methoxy-2-[4-(1-methylpiperidin-4-yl)anilino]-5-oxopyrido[2,3-d]pyrimidine-6-carboxamide	1798844: FMS Enzyme Inhibition Assay from Article 10.1021/jm801406h: “Pyrido[2,3-d]pyrimidin-5-ones: A Novel Class of Antiinflammatory Macrophage Colony-Stimulating Factor-1 Receptor Inhibitors (dagger).”	ic50	0.0004	uM
8-(4,4-dimethylcyclohexyl)-N-methoxy-2-[4-(1-methylpiperidin-4-yl)anilino]-5-oxopyrido[2,3-d]pyrimidine-6-carboxamide	1798844: FMS Enzyme Inhibition Assay from Article 10.1021/jm801406h: “Pyrido[2,3-d]pyrimidin-5-ones: A Novel Class of Antiinflammatory Macrophage Colony-Stimulating Factor-1 Receptor Inhibitors (dagger).”	ic50	0.0004	uM
5-(1-methylpyrazol-4-yl)-N-[2-methyl-5-[[3-(trifluoromethyl)benzoyl]amino]phenyl]pyridine-3-carboxamide	1449761: Inhibition of human CSF1R using poly[Glu:Tyr] (4:1) as substrate incubated for 2 hrs in presence of [33gammaP]ATP by P81 ion exchange chromatographic method	ic50	0.0005	uM
4-[(4-methylpiperazin-1-yl)methyl]-N-[4-methyl-3-[[3-(1H-pyrazol-4-yl)benzoyl]amino]phenyl]-3-(trifluoromethyl)benzamide	1449761: Inhibition of human CSF1R using poly[Glu:Tyr] (4:1) as substrate incubated for 2 hrs in presence of [33gammaP]ATP by P81 ion exchange chromatographic method	ic50	0.0005	uM
1-[4-[7-(dimethylamino)quinazolin-4-yl]oxyphenyl]-3-[[6-(trifluoromethyl)-3-pyridinyl]methyl]urea	1785256: Inhibition of wild-type human partial length CSF1R (I564 to S939 residues) expressed in bacterial expression system by Kinomescan method	ic50	0.0005	uM
[4-[4-[methyl-[(3-methylphenyl)methyl]amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]methanol	1989539: Inhibition of CSF1R (530 to 910 residues) (unknown origin)	ic50	0.0005	uM
[4-[4-[methyl(oxan-4-ylmethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]methanol	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0005	uM
[4-[4-[methyl-[(6-methyl-2-pyridinyl)methyl]amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]methanol	1989539: Inhibition of CSF1R (530 to 910 residues) (unknown origin)	ic50	0.0005	uM
[4-[6-[cyclopentylmethyl(methyl)amino]-7H-purin-8-yl]phenyl]methanol	1970713: Inhibition of CSF1R (unknown origin) using FRET-peptide as substrate incubated in presence of ATP by FRET based Z-LYTE assay	ic50	0.0005	uM
N-[3-[4-[methyl(oxan-4-ylmethyl)amino]-7H-pyrrolo[2,3-d]pyrimidin-6-yl]phenyl]prop-2-enamide	2088184: Inhibition of CSF1R (unknown origin) using EEEIYGEFE as substrate in presence of ATP by Z-LYTE based FRET assay	ic50	0.0005	uM
5-cyano-N-[2-(cyclohexen-1-yl)-4-(1,3-dimethyl-2-oxo-1,3-diazinan-5-yl)phenyl]-1H-imidazole-2-carboxamide	489881: Inhibition of human FMS by fluorescence polarization	ic50	0.0005	uM
8-(2,3-dihydro-1H-inden-5-yl)-N-ethoxy-2-[4-[2-(4-methylpiperazin-1-yl)ethyl]anilino]-5-oxopyrido[2,3-d]pyrimidine-6-carboxamide	1798844: FMS Enzyme Inhibition Assay from Article 10.1021/jm801406h: “Pyrido[2,3-d]pyrimidin-5-ones: A Novel Class of Antiinflammatory Macrophage Colony-Stimulating Factor-1 Receptor Inhibitors (dagger).”	ic50	0.0005	uM
8-(2,3-dihydro-1H-inden-5-yl)-2-[4-[2-(4-methylpiperazin-1-yl)ethyl]anilino]-5-oxo-N-propan-2-yloxypyrido[2,3-d]pyrimidine-6-carboxamide	1798844: FMS Enzyme Inhibition Assay from Article 10.1021/jm801406h: “Pyrido[2,3-d]pyrimidin-5-ones: A Novel Class of Antiinflammatory Macrophage Colony-Stimulating Factor-1 Receptor Inhibitors (dagger).”	ic50	0.0005	uM
8-(2,3-dihydro-1H-inden-5-yl)-N-methoxy-5-oxo-2-(4-piperidin-4-ylanilino)pyrido[2,3-d]pyrimidine-6-carboxamide	1798844: FMS Enzyme Inhibition Assay from Article 10.1021/jm801406h: “Pyrido[2,3-d]pyrimidin-5-ones: A Novel Class of Antiinflammatory Macrophage Colony-Stimulating Factor-1 Receptor Inhibitors (dagger).”	ic50	0.0005	uM
5-cyano-N-[2-(cyclohexen-1-yl)-4-(1-methyl-2,6-dioxopiperidin-4-yl)phenyl]-1H-imidazole-2-carboxamide	489881: Inhibition of human FMS by fluorescence polarization	ic50	0.0005	uM
N-(2-chloro-6-methylphenyl)-2-[[6-[4-(2-hydroxyethyl)piperazin-1-yl]-2-methylpyrimidin-4-yl]amino]-1,3-thiazole-5-carboxamide;hydrate	1739051: Binding affinity to human CSF1R	kd	0.0006	uM
N-[4-[1-(3-amino-3-methylbutanoyl)piperidin-4-yl]-2-(cyclohexen-1-yl)phenyl]-5-cyano-1H-imidazole-2-carboxamide	696437: Inhibition of FMS mediated phosphorylation using SYEGNSYTFIDPTQ as substrate after 80 mins by fluorescence polarization	ic50	0.0006	uM
5-cyano-N-[4-[4-(2-hydroxyethyl)piperazin-1-yl]-2-(4-methylpiperidin-1-yl)phenyl]furan-2-carboxamide	314392: Inhibition of human cytoplasmic FMS expressed in Sf9-baculovirus system after 80 mins by fluorescence polarization	ic50	0.0006	uM
trans-(1R,2R)-2-[[6-[(2-amino-3-chloro-4-pyridinyl)methoxy]-4-methoxy-1,3-benzothiazol-2-yl]amino]cyclohexan-1-ol	1723257: Binding affinity to human wild type CSF1R (I564 to S939 residues) expressed in bacterial expression system by kinome scan assay	kd	0.0006	uM

CTD chemical–gene interactions

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

Chemical	Actions (top 5)	PubMed papers
Tretinoin	affects cotreatment, increases phosphorylation, increases expression, increases reaction, decreases phosphorylation (+1 more)	4
ponatinib	decreases activity	2
Benzo(a)pyrene	affects methylation	2
Calcitriol	increases expression, decreases phosphorylation, increases reaction, increases response to substance	2
Diethylhexyl Phthalate	affects cotreatment, decreases expression, increases expression	2
Nickel	decreases expression, increases expression	2
Smoke	decreases expression, decreases reaction	2
Tobacco Smoke Pollution	decreases secretion, increases expression	2
pexidartinib	decreases activity	1
Asian ginseng	affects cotreatment, decreases expression	1
quinone	affects cotreatment, increases abundance, increases reaction	1
propionaldehyde	increases expression	1
bisphenol A	affects cotreatment, decreases methylation	1
hydroxyhydroquinone	affects cotreatment, increases abundance, increases reaction	1
hexamethylene bisacetamide	increases expression	1
sodium bichromate	decreases expression	1
sodium arsenite	increases expression	1
butyraldehyde	increases expression	1
hydroquinone	affects cotreatment, increases abundance, increases reaction	1
4-nonylphenol	affects cotreatment, decreases expression	1
pentanal	increases expression	1
4-tert-octylphenol	affects cotreatment, decreases expression	1
abrine	increases expression	1
ON 01910	increases expression	1
ormosil	affects binding, increases expression	1
(+)-JQ1 compound	decreases expression	1
Resveratrol	affects cotreatment, increases expression	1
Temozolomide	decreases expression	1
Decitabine	decreases reaction, decreases expression	1
Sunitinib	decreases activity	1

ChEMBL screening assays

897 unique, capped per target: 879 binding, 17 functional, 1 admet

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL1001229	Binding	Inhibition of C-FMS	The identification of pyrazolo[1,5-a]pyridines as potent p38 kinase inhibitors. — Bioorg Med Chem Lett
CHEMBL1964104	Functional	PUBCHEM_BIOASSAY: Navigating the Kinome. (Class of assay: other) Panel member name: CSF1R	PubChem BioAssay data set
CHEMBL4832367	ADMET	Inhibition of CSF1R (unknown origin)	Discovery and biological evaluation of phthalazines as novel non-kinase TGFβ pathway inhibitors. — Eur J Med Chem

Cellosaurus cell lines

20 cell lines: 9 induced pluripotent stem cell, 6 cancer cell line, 3 factor-dependent cell line, 1 spontaneously immortalized cell line, 1 transformed cell line

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

Cellosaurus	Name	Category	Sex
CVCL_2116	MKPL-1	Cancer cell line	Male
CVCL_6820	47.10	Factor-dependent cell line	Male
CVCL_B0UQ	JTUi007-A	Induced pluripotent stem cell	Female
CVCL_B1PD	Abcam HeLa CSF1R KO	Cancer cell line	Female
CVCL_B8E5	Abcam HCT 116 CSF1R KO	Cancer cell line	Male
CVCL_B8UD	Abcam MCF-7 CSF1R KO	Cancer cell line	Female
CVCL_B9GD	Abcam A-549 CSF1R KO	Cancer cell line	Male
CVCL_C3RG	HIHCNi007-A-1	Induced pluripotent stem cell	Male
CVCL_C3RH	HIHCNi007-A-2	Induced pluripotent stem cell	Male
CVCL_D7E2	U2OS-GFP-STAT1/CSF-1R	Cancer cell line	Female

Clinical trials (associated diseases)

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

Trial	Phase	Status	Title
NCT01662414	PHASE4	COMPLETED	Effect of Undenatured Cysteine-Rich Whey Protein Isolate (HMS 90®) in Patients With Parkinson’s Disease
NCT04871464	PHASE4	UNKNOWN	Role and Mechanism of Probiotics in Improving Motor Symptoms in Mild to Moderate Parkinson’s Disease
NCT05357612	PHASE4	RECRUITING	Characterization of the Serotonin 2A Receptor Selective PET Tracer [18F]MH.MZ in Patients With Neurodegenerative Diseases
NCT00722436	PHASE4	TERMINATED	Tranexamic Acid for Craniofacial Surgery
NCT02188576	PHASE4	COMPLETED	The Efficacy and Population Pharmacokinetics of Tranexamic Acid for Craniosynostosis Surgery
NCT00009191	PHASE4	COMPLETED	The Depression in Alzheimer’s Disease Study (DIADS)
NCT00009217	PHASE4	COMPLETED	Treatment of Behavioral Symptoms in Alzheimer’s Disease
NCT00018278	PHASE4	COMPLETED	Electrophysiologic Measures of Treatment Response in Alzheimer Disease
NCT00035204	PHASE4	COMPLETED	A Study of the Effects on Sleep, Attention, and Gastrointestinal Tolerance of Galantamine and Donepezil in Patients With Alzheimer’s Disease
NCT00042172	PHASE4	COMPLETED	Treatment for Early Memory Loss
NCT00046358	PHASE4	COMPLETED	The Effect of Short-Term Statins and NSAIDs on Levels of Beta-Amyloid, a Protein Associated With Alzheimer’s Disease
NCT00104442	PHASE4	COMPLETED	Study of the Effects of Current Drug Treatments on Levels of Certain Brain Chemicals in Alzheimer’s Disease
NCT00120874	PHASE4	COMPLETED	Memantine and Comprehensive, Individualized Management of Alzheimer’s Disease and Caregiver Training
NCT00142324	PHASE4	UNKNOWN	CALM-AD
NCT00165724	PHASE4	COMPLETED	Alzheimer’s Disease Long-term Follow-up Study (ALF Study)
NCT00165750	PHASE4	TERMINATED	Correlation Between Regional Brain Volume and Response to Donepezil Treatment in AD Patients
NCT00202124	PHASE4	COMPLETED	Double Blind Study of Trp01 in Patients With Alzheimer’s Disease
NCT00208819	PHASE4	COMPLETED	A Comparison of Two Standard Therapies in the Management of Dementia With Agitation
NCT00216515	PHASE4	COMPLETED	The Efficacy of Galantamine on the Attention and the Frontal Function of the Patients With Dementia of Alzheimer Type
NCT00230568	PHASE4	COMPLETED	EARTH 413: A Study of Aricept in Hispanic Patients With Mild to Moderate Alzheimer’s Disease (AD)
NCT00234637	PHASE4	COMPLETED	Rivastigmine Monotherapy and Combination Therapy With Memantine in Patients With Moderately Severe Alzheimer’s Disease Who Failed to Benefit From Previous Cholinesterase Inhibitor Treatment
NCT00245206	PHASE4	COMPLETED	Side Effects of Newer Antipsychotics in Older Adults
NCT00254033	PHASE4	COMPLETED	Apathy Associated With Alzheimer’s Disease
NCT00260624	PHASE4	COMPLETED	Escitalopram Treatment of Patients With Agitated Dementia
NCT00303277	PHASE4	COMPLETED	Do HMG CoA Reductase Inhibitors Affect Abeta Levels?
NCT00305903	PHASE4	COMPLETED	Safety and Tolerability of Rivastigmine With Add-on Memantine in Patients With Probable Alzheimer’s Disease
NCT00306124	PHASE4	UNKNOWN	Dopaminergic Enhancement of Learning and Memory in Healthy Adults and Patients With Dementia/Mild Cognitive Impairment
NCT00334906	PHASE4	COMPLETED	Study of Memantine in Assessment of Selected Measures of Volumetric Magnetic Resonance Imaging (MRI) and Cognition in Moderate AD (Alzheimer’s Disease)
NCT00369603	PHASE4	TERMINATED	Functional Brain Imaging of Medication Treatment Response in Mild Alzheimer’s Disease Patients
NCT00375557	PHASE4	WITHDRAWN	Safety and Efficacy of Divalproex and Quetiapine in Elderly Alzheimer’s Dementia Patients
NCT00381381	PHASE4	COMPLETED	The Clinical Response of Choline Acetyltransferase and Apolipoprotein Epsilon Gene Polymorphisms to Donepezil in Alzheimer’s Disease
NCT00385684	PHASE4	COMPLETED	Low-Dose Opiate Therapy for Discomfort in Dementia (L-DOT)
NCT00401167	PHASE4	COMPLETED	Memantine for Agitation and Aggression in Severe Alzheimer’s Disease
NCT05508789	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Donanemab (LY3002813) in Participants With Early Symptomatic Alzheimer’s Disease (TRAILBLAZER-ALZ 5)
NCT05738486	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Different Donanemab (LY3002813) Dosing Regimens in Adults With Early Alzheimer’s Disease (TRAILBLAZER-ALZ 6)
NCT06111014	PHASE3	TERMINATED	Continuation Study for Latozinemab
NCT06672237	PHASE3	RECRUITING	A Phase 3 Study of NTLA-2001 in ATTRv-PN
NCT01010932	PHASE3	COMPLETED	Carotid and Vertebral Magnetic Resonance Angiography (MRA) Study Comparing Dotarem and Time Of Flight (TOF)
NCT01012674	PHASE3	COMPLETED	Carotid and Vertebral Magnetic Resonance Angiography (MRA) Study Comparing Dotarem and Time Of Flight (TOF)
NCT02455440	PHASE3	COMPLETED	Esmolol Infusion in Patients Undergoing Craniotomy