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): brain abnormalities, neurodegeneration, and dysosteosclerosis (Definitive, ClinGen) — +3 more curated relationships
• GWAS associations: 17
• Clinical variants (ClinVar): 1,059 total — 41 pathogenic, 39 likely-pathogenic (top 3% of genes corpus-wide)
• GTEx eGene: eQTL in 19 tissues, sQTL in 1 (q < 0.05)
• Phenotypes (HPO): 79
• Drug target: yes — 18 curated mechanism-of-action drugs; 79 molecules with ChEMBL bioactivity (top 1% of genes corpus-wide)
• Approved drugs (ChEMBL mechanism of action): 5 — Axatilimab, Pazopanib Hydrochloride, Pexidartinib, +2 more
• Cancer driver (intOGen): loss-of-function (tumor-suppressor-like) across 2 cancer types (inferred from mutation patterns)
• Constraint (gnomAD): pLI 0.98 (loss-of-function intolerant), LOEUF 0.48 (moderately constrained)
• MANE Select transcript: NM_001288705
• Interaction hub: 3,914 STRING partners (top 1% of genes corpus-wide)

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	M-CSF-R, C-FMS, CSFR, CD115
Ensembl gene	ENSG00000182578
Ensembl biotype	protein_coding
OMIM	164770
Entrez	1436

Gene structure

Transcript identifiers

Ensembl transcripts: 20 — 14 protein_coding, 3 retained_intron, 2 nonsense_mediated_decay, 1 protein_coding_CDS_not_defined

MANE Select: ENST00000675795

Transcript support level (Ensembl TSL; 1 = fully mRNA-supported): TSL 1: 3, TSL 2: 2, TSL 3: 1, TSL 4: 1, TSL 5: 2

ENST00000675795, ENST00000286301, ENST00000502660, ENST00000504875, ENST00000509861, ENST00000511344, ENST00000513609, ENST00000515068, ENST00000515239, ENST00000543093, ENST00000911110, ENST00000911111, ENST00000911112, ENST00000911113, ENST00000911114, ENST00000911115, ENST00000911116, ENST00000960687, ENST00000960688, ENST00001132457

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.

Highest expression (Bgee): granulocyte, monocyte, leukocyte, mononuclear cell, spleen.

Genetic regulation across tissues (GTEx eQTL / sQTL)

CSF1R is an eGene in GTEx v10 — its expression is genetically regulated (eQTL) in 19 tissues and its splicing (sQTL) in 1 (q < 0.05). Strongest associations first; slope = normalized effect of the lead variant’s alternative allele.

20 significant tissue × QTL associations:

Tissue	QTL	q-value	Slope	Lead variant
Stomach	eqtl	9.1e-17	+0.39	5:150084099:C:G
Skin Not Sun Exposed Suprapubic	eqtl	5.3e-06	+0.34	5:150063707:G:C
Nerve Tibial	eqtl	1.0e-04	+0.22	5:150063707:G:C
Breast Mammary Tissue	eqtl	5.7e-04	+0.24	5:150063707:G:C
Skin Sun Exposed Lower leg	eqtl	1.3e-03	+0.27	5:150063707:G:C
Cells Cultured fibroblasts	eqtl	1.4e-03	-0.16	5:150067043:C:A
Testis	eqtl	1.5e-03	+0.48	5:150066601:G:A
Whole Blood	eqtl	1.6e-03	-0.08	5:150064431:C:T
Adipose Subcutaneous	eqtl	5.1e-03	-0.07	5:150064431:C:T
Adipose Visceral Omentum	sqtl	7.3e-03	-1.43	5:150444094:G:A
Small Intestine Terminal Ileum	eqtl	7.8e-03	-0.30	5:150336426:C:T
Brain Caudate basal ganglia	eqtl	1.5e-02	-0.37	5:150522906:C:T
Adipose Visceral Omentum	eqtl	1.8e-02	+0.41	5:149952411:T:A
Brain Hypothalamus	eqtl	2.3e-02	-1.26	5:151044793:G:A
Muscle Skeletal	eqtl	2.4e-02	+0.19	5:150059895:T:G
Lung	eqtl	2.4e-02	+0.22	5:150068497:G:A
Thyroid	eqtl	2.5e-02	-0.09	5:150064431:C:T
Minor Salivary Gland	eqtl	2.7e-02	+0.19	5:150122188:A:C
Artery Tibial	eqtl	3.6e-02	+0.27	5:149850548:C:T
Cells EBV-transformed lymphocytes	eqtl	4.2e-02	+0.21	5:150092493:A:G

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

showing 100 of 284 tissues 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
medulla oblongata	UBERON:0001896	88.57	gold quality
rectum	UBERON:0001052	88.52	gold quality
descending thoracic aorta	UBERON:0002345	88.51	gold quality
omental fat pad	UBERON:0010414	88.42	gold quality
peritoneum	UBERON:0002358	88.38	gold quality
upper lobe of left lung	UBERON:0008952	88.36	gold quality
left adrenal gland	UBERON:0001234	88.34	gold quality
right adrenal gland	UBERON:0001233	88.20	gold quality
coronary artery	UBERON:0001621	88.02	gold quality
left adrenal gland cortex	UBERON:0035825	88.00	gold quality
subcutaneous adipose tissue	UBERON:0002190	87.96	gold quality
upper lobe of lung	UBERON:0008948	87.89	gold quality
adipose tissue of abdominal region	UBERON:0007808	87.86	gold quality
small intestine Peyer’s patch	UBERON:0003454	87.72	gold quality
ileal mucosa	UBERON:0000331	87.63	gold quality
cranial nerve II	UBERON:0000941	87.62	gold quality
ileum	UBERON:0002116	87.54	silver quality
left coronary artery	UBERON:0001626	87.49	gold quality
adrenal cortex	UBERON:0001235	87.48	gold quality
adrenal gland	UBERON:0002369	87.28	gold quality
layer of synovial tissue	UBERON:0007616	87.09	gold quality
small intestine	UBERON:0002108	87.02	gold quality
midbrain	UBERON:0001891	86.98	gold quality
thoracic aorta	UBERON:0001515	86.96	gold quality
substantia nigra	UBERON:0002038	86.65	gold quality
ascending aorta	UBERON:0001496	86.59	gold quality
skin of hip	UBERON:0001554	86.21	gold quality
adipose tissue	UBERON:0001013	86.09	gold quality
dorsal plus ventral thalamus	UBERON:0001897	86.00	gold quality
corpus callosum	UBERON:0002336	86.00	gold quality
medial globus pallidus	UBERON:0002477	85.96	gold quality
right lung	UBERON:0002167	85.92	gold quality
hypothalamus	UBERON:0001898	85.86	gold quality
trigeminal ganglion	UBERON:0001675	85.77	gold quality
connective tissue	UBERON:0002384	85.43	gold quality
mucosa of stomach	UBERON:0001199	85.40	gold quality
mammary duct	UBERON:0001765	85.14	gold quality
apex of heart	UBERON:0002098	85.08	gold quality
lung	UBERON:0002048	84.91	gold quality
right uterine tube	UBERON:0001302	84.63	gold quality
periodontal ligament	UBERON:0008266	84.46	gold quality
body of stomach	UBERON:0001161	84.36	gold quality
urinary bladder	UBERON:0001255	84.25	gold quality
aorta	UBERON:0000947	84.11	gold quality
globus pallidus	UBERON:0001875	84.07	gold quality
left uterine tube	UBERON:0001303	84.03	gold quality
transverse colon	UBERON:0001157	83.86	gold quality
stomach	UBERON:0000945	83.85	gold quality
amygdala	UBERON:0001876	83.83	gold quality
intestine	UBERON:0000160	83.56	gold quality
muscle layer of sigmoid colon	UBERON:0035805	83.42	gold quality
cardiac muscle of right atrium	UBERON:0003379	83.31	silver quality
dorsal motor nucleus of vagus nerve	UBERON:0002870	83.30	gold quality
caudate nucleus	UBERON:0001873	83.17	gold quality
trabecular bone tissue	UBERON:0002483	82.92	gold quality
minor salivary gland	UBERON:0001830	82.83	gold quality
epithelium of mammary gland	UBERON:0003244	82.73	gold quality
large intestine	UBERON:0000059	82.69	gold quality
colon	UBERON:0001155	82.55	gold quality
eye	UBERON:0000970	82.49	gold quality
esophagogastric junction muscularis propria	UBERON:0035841	82.46	gold quality
duodenum	UBERON:0002114	82.43	gold quality
prefrontal cortex	UBERON:0000451	82.42	gold quality
lower esophagus muscularis layer	UBERON:0035833	82.39	gold quality
lower esophagus	UBERON:0013473	82.37	gold quality
substantia nigra pars reticulata	UBERON:0001966	82.19	silver quality
fundus of stomach	UBERON:0001160	82.18	gold quality
lower lobe of lung	UBERON:0008949	82.14	gold quality
Ammon’s horn	UBERON:0001954	82.11	gold quality
tibial artery	UBERON:0007610	82.07	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

HPA expression

RNA tissue specificity: Tissue enhanced

showing 60 of 80 HPA tissues / cells by nTPM:

Tissue / cell	Axis	nTPM	Protein (IHC)
Hypothalamus	tissue	109.7
Spleen	tissue	106.8	medium
Substantia nigra	tissue	99.6
Placenta	tissue	97.1	medium
Periaqueductal gray	tissue	96.0
Spinal cord	tissue	89.8
Medulla oblongata	tissue	87.0
Caudate putamen	tissue	86.3
Septum	tissue	83.6
Pons	tissue	80.3
Corpus callosum	tissue	78.7
Appendix	tissue	70.9	medium
Midbrain	tissue	70.4
Frontal cortex	tissue	68.3
Thalamus	tissue	67.9
Putamen	tissue	62.2
Ventral forebrain	tissue	59.9
Amygdala	tissue	55.9
Superior colliculi	tissue	55.1
Hippocampal formation	tissue	52.7
Locus coeruleus	tissue	51.4
Lymph node	tissue	51.0	medium
Basal ganglia	tissue	50.8	low
Hippocampus	tissue	49.4	medium
Choroid plexus	tissue	49.0
Adipose tissue	tissue	47.9	low
Cerebral cortex	tissue	41.3	medium
Insular cortex	tissue	41.3
Motor cortex	tissue	40.4
Temporal cortex	tissue	40.2
Urinary bladder	tissue	39.8	medium
Entorhinal cortex	tissue	39.5
Smooth muscle	tissue	39.1	low
Gallbladder	tissue	38.0	medium
Prefrontal cortex	tissue	37.6
Retrosplenial cortex	tissue	35.9
Small intestine	tissue	35.1	medium
Lung	tissue	34.5	medium
Retrosplenial and cingulate cortex	tissue	33.0
Somatosensory cortex	tissue	31.8
Nucleus accumbens	tissue	29.9
Adrenal gland	tissue	29.3	medium
Occipital cortex	tissue	29.0
Olfactory bulb	tissue	27.3
Liver	tissue	25.7	low
Duodenum	tissue	22.9	medium
Colon	tissue	20.5	medium
Cervix	tissue	20.3	medium
Tonsil	tissue	20.3	medium
Rectum	tissue	20.2	medium
Stomach 1	tissue	19.9	medium
Esophagus	tissue	19.7	medium
Seminal vesicle	tissue	19.6	medium
Breast	tissue	18.1	low
Fallopian tube	tissue	16.2	medium
Heart muscle	tissue	15.5	medium
Thymus	tissue	13.3
Ovary	tissue	12.9	low
Skin 1	tissue	12.7	low
Endometrium 1	tissue	11.3	medium

Brain sub-regions (HPA Brain Atlas): detected in 192; highest nTPM — Lateral funiculus (white matter) (153.1), Nucleus raphe pallidus (98.1), Nucleus rhomboideus (94.4), Medial olivary nucleus (91.9), Dorsal spinal cord (89.8).

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)

Targeting CSF1R by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific).

showing 30 of 41 miRNAs:

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

Functional genomics

gnomAD constraint: pLI 0.975, LOEUF 0.485, missense Z 2.43 — population loss-of-function intolerance (pLI ≥ 0.9 = LoF-intolerant; lower LOEUF = more constrained). gnomAD

Regional missense constraint (gnomAD)

ENST00000675795 splits into 5 regions with different missense tolerance; 0 are missense-constrained. Missense variants in a constrained region (low observed/expected) are more likely to be pathogenic. gnomAD

Region (GRCh38)	Missense obs / exp	o/e	Constrained
5:150,054,072–150,054,351	85 / 78.8	1.08	no
5:150,054,352–150,057,304	74 / 187.4	0.39	no
5:150,057,305–150,059,801	124 / 117.7	1.05	no
5:150,059,802–150,061,758	76 / 133.6	0.57	no
5:150,061,759–150,086,427	693 / 735.4	0.94	no

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), LMTK2 (ENSG00000164715), RET (ENSG00000165731), AXL (ENSG00000167601), ROR2 (ENSG00000169071), ALK (ENSG00000171094), INSR (ENSG00000171105), ERBB4 (ENSG00000178568), EPHB3 (ENSG00000182580), EPHA10 (ENSG00000183317), ROR1 (ENSG00000185483), EPHB4 (ENSG00000196411), NTRK1 (ENSG00000198400), DDR1 (ENSG00000204580)

Mouse-model phenotypes

Observed phenotypes of the mouse ortholog (MGI:1339758) from MGI (Alliance of Genome Resources) — knockout/mutant phenotypes, ranked by annotation count. 77 distinct phenotype terms.

Mouse phenotype (MP)	Records
abnormal skeleton morphology	3
increased bone mineral density	2
fragile skeleton	1
failure of bone resorption	1
osteoporosis	1
osteopetrosis	1
failure of tooth eruption	1
abnormal trabecular bone morphology	1
decreased compact bone thickness	1
abnormal leukocyte cell number	1
decreased monocyte cell number	1
increased granulocyte number	1
domed cranium	1
short limbs	1
kinked tail	1
abnormal spleen morphology	1
abnormal corpus callosum morphology	1
abnormal lateral ventricle morphology	1
demyelination	1
decreased body weight	1
decreased body size	1
increased anxiety-related response	1
ataxia	1
impaired coordination	1
abnormal skeleton physiology	1

+52 more (showing top 25).

Additional cross-species homologs

Sequence homologs in species beyond Ensembl Compara’s model organisms, by Diamond local protein alignment (% sequence identity; best hit per species). Lower-identity rows are more distant homologs. The matched accessions’ gene symbols aren’t in biobtree yet, so each row shows the UniProt accession.

Organism	UniProt	Identity	Method
Bos taurus	P43481	99.4%	Diamond
Capra hircus	Q28317	99.4%	Diamond
Felis catus	Q28889	99.2%	Diamond
Canis lupus familiaris	O97799	98.4%	Diamond
Sus scrofa	Q2HWD6	98.1%	Diamond
Xenopus tropicalis	Q67E00	95.4%	Diamond
Callithrix jacchus	Q76II0	94.6%	Diamond
Gallus gallus	P18461	92.7%	Diamond
Notophthalmus viridescens	Q91147	87.9%	Diamond
Pleurodeles waltl	Q91285	87.8%	Diamond
Coturnix coturnix	Q90330	81.0%	Diamond
Xenopus laevis	P22182	80.6%	Diamond
Takifugu rubripes	Q8AXC7	73.2%	Diamond
Coturnix japonica	P52583	73.1%	Diamond
Ciona intestinalis	Q4H3K6	60.1%	Diamond
Halocynthia roretzi	Q95YM9	59.9%	Diamond
Drosophila melanogaster	Q09147	55.0%	Diamond
Dugesia japonica	Q8MY86	51.0%	Diamond
Hydra vulgaris	Q86PM4	50.2%	Diamond
Caenorhabditis elegans	Q21038	49.5%	Diamond
Strongylocentrotus purpuratus	Q26614	46.4%	Diamond
Bombyx mori	D2IYS2	44.0%	Diamond
Feline sarcoma virus (STRAIN HARDY-ZUCKERMAN 4)	P04048	99.2%	Diamond
Feline sarcoma virus (STRAIN MCDONOUGH)	P00545	99.1%	Diamond

Genomic neighbors (locus context)

Genes flanking this one on the chromosome (NCBI/Entrez gene neighborhood). Positional context only — some neighbors share regulatory elements (bidirectional promoters, antisense/overlapping transcripts), but chromosomal adjacency is not itself a functional relationship (see Interactions for that).

Symbol	Type	Entrez
PDGFRB	protein-coding	5159
SLC6A7	protein-coding	6534
CAMK2A	protein-coding	815
CDX1	protein-coding	1044
HMGXB3	protein-coding	22993
TIGD6	protein-coding	81789
SLC26A2	protein-coding	1836

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)

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

UniProt features (183 total): strand 55, sequence variant 37, helix 30, 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 by resolution:

PDB	Title	Method	Resolution (Å)
8JOT	Crystal structure of CSF-1R kinase domain with sulfatinib	X-RAY DIFFRACTION	1.69
6T2W	Crystal structure of the CSF1R kinase domain with a dihydropurinone inhibitor (compound 4)	X-RAY DIFFRACTION	1.7
2I1M	cFMS tyrosine kinase (tie2 KID) in complex with an arylamide inhibitor	X-RAY DIFFRACTION	1.8
6IG8	Crystal structure of CSF-1R kinase domain with a small molecular inhibitor, JTE-952	X-RAY DIFFRACTION	1.8
2I0Y	cFMS tyrosine kinase (FGF KID) in complex with an arylamide inhibitor	X-RAY DIFFRACTION	1.9
8CGC	Structure of CSF1R in complex with a pyrollopyrimidine (compound 23)	X-RAY DIFFRACTION	1.93
3DPK	cFMS tyrosine kinase in complex with a pyridopyrimidinone inhibitor	X-RAY DIFFRACTION	1.95
3BEA	cFMS tyrosine kinase (tie2 KID) in complex with a pyrimidinopyridone inhibitor	X-RAY DIFFRACTION	2.02
3KRJ	cFMS tyrosine kinase in complex with 4-Cyano-1H-imidazole-2-carboxylic acid …	X-RAY DIFFRACTION	2.1
6N33	Crystal structure of fms kinase domain with a small molecular inhibitor, PLX5622	X-RAY DIFFRACTION	2.25
8W1L	Structure of CSF1R kinase domain in complex with Cpd 32	X-RAY DIFFRACTION	2.26
3KRL	cFMS Tyrosine kinase in complex with 5-Cyano-furan-2-carboxylic acid …	X-RAY DIFFRACTION	2.4
3LCD	Inhibitor Bound to A DFG-In structure of the Kinase Domain of CSF-1R	X-RAY DIFFRACTION	2.5
4LIQ	Structure of the extracellular domain of human CSF-1 receptor in complex with the Fab …	X-RAY DIFFRACTION	2.6
6WXJ	CSF1R signaling is a regulator of pathogenesis in progressive MS	X-RAY DIFFRACTION	2.62
2OGV	Crystal Structure of the Autoinhibited Human c-Fms Kinase Domain	X-RAY DIFFRACTION	2.7
7TNH	Crystal structure of CSF1R kinase domain in complex with DP-6233	X-RAY DIFFRACTION	2.7
4R7I	Crystal structure of FMS kinase domain with a small molecular inhibitor, GLEEVEC	X-RAY DIFFRACTION	2.75
2I0V	c-FMS tyrosine kinase in complex with a quinolone inhibitor	X-RAY DIFFRACTION	2.8
7MFC	Crystal structure of CSF1R in complex with vimseltinib	X-RAY DIFFRACTION	2.8
4R7H	Crystal structure of FMS KINASE domain with a small molecular inhibitor, PLX3397	X-RAY DIFFRACTION	2.8
4WRL	Structure of the human CSF-1:CSF-1R complex	X-RAY DIFFRACTION	2.8
4HW7	Crystal structure of FMS kinase domain with a small molecular inhibitor, PLX647-OME	X-RAY DIFFRACTION	2.9
4DKD	Crystal Structure of Human Interleukin-34 Bound to Human CSF-1R	X-RAY DIFFRACTION	3
3LCO	Inhibitor Bound to A DFG-Out structure of the Kinase Domain of CSF-1R	X-RAY DIFFRACTION	3.4
4WRM	Structure of the human CSF-1:CSF-1R complex	X-RAY DIFFRACTION	6.85

Predicted structure (AlphaFold)

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

Antibody-complex structures (SAbDab)

2 antibodies co-crystallized with this protein: 4LIQ, EMACTUZUMAB

Human Protein Atlas

Protein class: Enzymes, ENZYME proteins, Transferases, Kinases, Tyr protein kinases, CD markers, Transporters, Accessory Factors Involved in Transport, Predicted membrane proteins, Predicted intracellular proteins, Plasma proteins, RAS pathway related proteins, Cancer-related genes, Candidate cancer biomarkers, Disease related genes, FDA approved drug targets, Small molecule drugs, Human disease related genes

Protein evidence: Evidence at protein level

Subcellular location (HPA, imaging): plasma membrane; vesicles

Antibody reliability (HPA): IH approved · IF supported

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)

Position	Role
778	proton acceptor

Ligand- & substrate-binding residues (2)

Position	Role
588–596	ATP
616	ATP

Post-translational modifications (9)

Type	Positions
Phosphotyrosine	546, 561, 699, 708, 723, 809, 923, 969
Phosphoserine	713

Disulfide bonds (4)

Type	Positions
Disulfide bond	42–84, 127–177, 224–278, 419–485

Glycosylation sites (11)

Type	Positions
N-linked (glcnac…) asparagine	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

2 curated (TAS) of 9; 0 disease pathways. Curated-first; evidence = TAS (manually curated) vs IEA (electronic inference).

9 pathways:

ID	Pathway	Evidence
R-HSA-449836	Other interleukin signaling	curated
R-HSA-8853884	Transcriptional Regulation by VENTX	curated
R-HSA-1280215	Cytokine Signaling in Immune system	—
R-HSA-74160	Gene expression (Transcription)	—
R-HSA-212436	Generic Transcription Pathway	—
R-HSA-168256	Immune System	—
R-HSA-73857	RNA Polymerase II Transcription	—
R-HSA-9680350	Signaling by CSF1 (M-CSF) in myeloid cells	electronic
R-HSA-449147	Signaling by Interleukins	—

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), positive regulation of macrophage proliferation (GO:0120041), regulation of macrophage migration (GO:1905521), positive regulation of cell motility (GO:2000147), immune system process (GO:0002376), myeloid leukocyte differentiation (GO:0002573), regulation of immune system process (GO:0002682), protein phosphorylation (GO:0006468), response to stress (GO:0006950), anatomical structure morphogenesis (GO:0009653), regulation of cell morphogenesis (GO:0022604), regulation of cell population proliferation (GO:0042127), animal organ development (GO:0048513), regulation of developmental process (GO:0050793)

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 (5): 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

6 top-level categories:

Category	Pathways
Cytokine Signaling in Immune system	2
Signaling by Interleukins	1
Generic Transcription Pathway	1
Immune System	1
RNA Polymerase II Transcription	1
Gene expression (Transcription)	1

GO top-level categories

60 GO namespaces:

Category	Terms
myeloid leukocyte differentiation	3
protein phosphorylation	2
cellular process	2
cell population proliferation	2
regulation of cell population proliferation	2
mononuclear cell differentiation	2
protein binding	2
cellular anatomical structure	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
cell surface receptor signaling pathway	1
cellular response to cytokine stimulus	1
olfactory lobe development	1
animal gross anatomical part developmental process	1
forebrain generation of neurons	1
central nervous system neuron differentiation	1
cell development	1
protein kinase activity	1
transmembrane receptor protein tyrosine kinase activity	1
macrophage colony-stimulating factor signaling pathway	1
adenyl ribonucleotide binding	1
purine ribonucleoside triphosphate binding	1
anion binding	1
phosphatase binding	1
identical protein binding	1
protein dimerization activity	1
nucleoside phosphate binding	1
heterocyclic compound binding	1
kinase activity	1
phosphotransferase activity, alcohol group as acceptor	1
catalytic activity, acting on a protein	1
protein tyrosine kinase activity	1
transmembrane receptor protein kinase activity	1
binding	1
transferase activity, transferring phosphorus-containing groups	1
catalytic activity	1
cation binding	1
membrane	1
cell periphery	1

Experimentally-supported GO annotations

GO terms backed by direct experimental evidence (ECO experimental codes) — 18 of 72 annotations. The rest are phylogenetic / computational / electronic (IEA).

18 experimentally-supported GO terms:

GO term	NS	Evidence
cell population proliferation	BP	mutant phenotype
cell-cell junction maintenance	BP	mutant phenotype
cellular response to macrophage colony-stimulating factor stimulus	BP	mutant phenotype
cytokine-mediated signaling pathway	BP	mutant phenotype
hemopoiesis	BP	mutant phenotype
peptidyl-tyrosine phosphorylation	BP	direct assay
positive regulation of cell motility	BP	mutant phenotype
positive regulation of cell population proliferation	BP	mutant phenotype
positive regulation of chemokine production	BP	mutant phenotype
positive regulation of macrophage chemotaxis	BP	genetic interaction
positive regulation of macrophage proliferation	BP	genetic interaction
positive regulation of protein phosphorylation	BP	mutant phenotype
positive regulation of protein tyrosine kinase activity	BP	mutant phenotype
protein autophosphorylation	BP	direct assay
regulation of cell shape	BP	mutant phenotype
plasma membrane	CC	direct assay
cytokine binding	MF	direct assay
macrophage colony-stimulating factor receptor activity	MF	mutant phenotype

Protein interactions and networks

STRING

showing 15 of 3,914 interactions by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
CSF1R	CSF1	P09603	967
CSF1R	IL34	Q6ZMJ4	908
CSF1R	CSF2	P04141	865
CSF1R	SPI1	P17947	849
CSF1R	CD68	P34810	814
CSF1R	TREM2	Q9NZC2	789
CSF1R	ITGAM	P11215	780
CSF1R	TMEM119	Q4V9L6	770
CSF1R	CX3CR1	P49238	767
CSF1R	PTPRC	P08575	750
CSF1R	CCR2	P41597	748
CSF1R	IL3	P08700	742
CSF1R	P2RY12	Q9H244	741
CSF1R	SIRPA	P78324	720
CSF1R	ITGAX	P20702	718

IntAct

Human interaction partners of CSF1R in IntAct, one row per partner (best MIscore; records = supporting IntAct interaction records).

18 partners by confidence:

Partner	Interaction type	MIscore	Records
CSF1	direct interaction	0.920	14
IL34	direct interaction	0.790	9
EPHB2	association	0.500	2
EPHA7	physical association	0.500	1
PKM	phosphorylation reaction	0.440	1
PIK3R1	proximity	0.410	1
HSP90AB1	physical association	0.400	1
CTDSP1	physical association	0.370	1
CTDSP2	physical association	0.370	1
FYN	association	0.350	1
FBXW7	proximity	0.270	3
SMAD4	proximity	0.270	2
SPOP	proximity	0.270	2
AKT1	proximity	0.270	1
GRAP2	proximity	0.270	1
HGS	proximity	0.270	1
PTEN	proximity	0.270	1
TP53	proximity	0.270	1

BioGRID (91 interactions; top human partners by supporting records): CBL (Two-hybrid; 10 records), GRB2 (Two-hybrid; 4 records), SOCS1 (Two-hybrid; 4 records), FBXW11 (Affinity Capture-Western; 3 records), FYN (Reconstituted Complex; 3 records), PIK3R1 (Co-localization; 3 records), CALM3 (Affinity Capture-MS; 2 records), EPHA7 (Affinity Capture-Western; 2 records), EPHB2 (Affinity Capture-Western; 2 records), GRAP2 (Co-localization; 2 records), HSPA1B (Affinity Capture-MS; 2 records), KIT (Affinity Capture-Western; 2 records), PIK3CA (Reconstituted Complex; 2 records), SOS1 (Affinity Capture-Western; 2 records), STAP2 (Affinity Capture-Western; 2 records)

SIGNOR signaling

showing 17 of 27 signaling 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”

Protein complexes (CORUM) (5)

Experimentally-characterized complexes this protein is a subunit of (★ = the complex contains a known drug target).

5 complexes by size:

Complex	Subunits	Members
★ CSF1R-INPPL1 complex	2	CSF1R, INPPL1
★ CSF1R-EGR1 complex	2	CSF1R, EGR1
★ CSF1-CSF1R complex	2	CSF1, CSF1R
★ CSF1R-ELK1 complex	2	CSF1R, ELK1
★ CSF1R-YY1 complex	2	CSF1R, YY1

Ligand–receptor pairs (CellPhoneDB) (2)

Curated ligand–receptor partners (cell-cell communication), distinct from the protein–protein interactions above.

2 ligand–receptor pairs:

Partner	This gene’s role	Signaling class
IL34	receptor	Signaling by Interleukin
CSF1	receptor	Signaling by Colony-Stimulating factor

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 18 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	101.8×	3e-07

GO biological processes:

GO term	Partners	Fold	FDR
protein stabilization	5	18.6×	8e-04
positive regulation of gene expression	7	15.1×	8e-05

Disease & clinical

Cancer significance

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

intOGen’s role is a computational inference from cohort mutation patterns and may diverge from the gene’s curated/literature role; read it alongside the evidence above.

Genetic variants

Variant evidence for CSF1R: 1,059 ClinVar records (80 pathogenic / likely pathogenic); 17 GWAS associations. Per-variant detail is on Sugi Variant.

ClinVar

1,059 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	41
Likely pathogenic	39
Uncertain significance	417
Likely benign	338
Benign	90

Top pathogenic / likely-pathogenic (15)

Showing the 15 best-reviewed of 80 pathogenic / likely-pathogenic ClinVar records, ranked by ClinVar review status (expert panel → multiple submitters → single submitter), then by number of submitting labs.

Variant ID	HGVS	Classification	Review	Submitters
162111	NM_001288705.3(CSF1R):c.2330G>A (p.Arg777Gln)	Pathogenic	criteria provided, multiple submitters, no conflicts	6
520697	NM_001288705.3(CSF1R):c.1441C>T (p.Gln481Ter)	Pathogenic	criteria provided, multiple submitters, no conflicts	5
162118	NM_001288705.3(CSF1R):c.2342C>T (p.Ala781Val)	Pathogenic	criteria provided, multiple submitters, no conflicts	4
1029946	NM_001288705.3(CSF1R):c.1620T>A (p.Tyr540Ter)	Pathogenic	criteria provided, multiple submitters, no conflicts	2
1322164	NM_001288705.3(CSF1R):c.2026C>T (p.Arg676Ter)	Pathogenic	criteria provided, multiple submitters, no conflicts	2
162120	NM_001288705.3(CSF1R):c.2629C>T (p.Gln877Ter)	Likely pathogenic	criteria provided, multiple submitters, no conflicts	3
3068555	NM_001288705.3(CSF1R):c.2339C>T (p.Ala780Val)	Likely pathogenic	criteria provided, multiple submitters, no conflicts	3
982040	NM_001288705.3(CSF1R):c.1772G>A (p.Gly591Glu)	Likely pathogenic	criteria provided, multiple submitters, no conflicts	2
635123	NM_001288705.3(CSF1R):c.1969+115_1969+116del	Pathogenic	criteria provided, single submitter	3
29810	NM_001288705.3(CSF1R):c.2624T>C (p.Met875Thr)	Pathogenic	criteria provided, single submitter	2
162109	NM_001288705.3(CSF1R):c.1699del (p.Thr567fs)	Pathogenic	criteria provided, single submitter	2
2575888	NM_001288705.3(CSF1R):c.2471C>T (p.Pro824Leu)	Pathogenic	criteria provided, single submitter	2
38373	NM_001288705.3(CSF1R):c.1766G>A (p.Gly589Glu)	Pathogenic	criteria provided, single submitter	1
38375	NM_001288705.3(CSF1R):c.2308G>C (p.Ala770Pro)	Pathogenic	criteria provided, single submitter	1
978469	NM_001288705.3(CSF1R):c.1969+1G>A	Pathogenic	criteria provided, single submitter	1

Per-variant reference — Sugi Variant

Explore CSF1R’s pathogenic and likely-pathogenic variants individually — each with its ClinVar classification and conditions, applied ACMG criteria (ClinGen expert panel, where curated), the calibrated in-silico predictors (AlphaMissense, REVEL, SaProt, conservation), gnomAD frequency, and the predictor-disagreement QC signal — on Sugi Variant.

SpliceAI

Top 10 splice-impact sites by Δscore (adjacent positions with the same effect merged into one range; 40 predictions of 2,038):

Position(s)	Effect	Δscore	Sites
5:150054225–150054226	acceptor_loss	1.0000	5
5:150054231	acceptor_gain	1.0000
5:150054320–150054321	donor_loss	1.0000	5
5:150054330	donor_gain	1.0000
5:150054431–150054432	acceptor_loss	1.0000	6
5:150055337–150055338	acceptor_loss	1.0000	3
5:150056024–150056025	donor_loss	1.0000	5
5:150056138–150056139	acceptor_loss	1.0000	5
5:150056342–150056343	acceptor_loss	1.0000	5
5:150057285–150057286	donor_loss	1.0000	4

HPA cancer prognostics

RNA cancer specificity: Low cancer specificity

Human Protein Atlas survival analysis — favorable = higher expression predicts better survival.

1 prognostic cancer types:

Cancer	Prognostic	p-value
Testicular Germ Cell Tumor (TCGA)	unfavorable	0.000512

Disease associations

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

GenCC curated gene-disease

4 curated gene-disease records:

Disease	Classification	Inheritance	Records
hereditary diffuse leukoencephalopathy with axonal spheroids and pigmented glia	Definitive	Autosomal dominant	3
brain abnormalities, neurodegeneration, and dysosteosclerosis	Definitive	Autosomal recessive	2
leukoencephalopathy, diffuse hereditary, with spheroids 1	Strong	Autosomal dominant
early-onset calcifying leukoencephalopathy-skeletal dysplasia	Supportive	Autosomal recessive

ClinGen Gene-Disease Validity

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

2 validity classifications:

Disease	Classification	Inheritance
brain abnormalities, neurodegeneration, and dysosteosclerosis	Definitive	AR
leukoencephalopathy, diffuse hereditary, with spheroids 1	Definitive	AD

Genomics England panels (PanelApp)

Clinical diagnostic gene panels including this gene (12 panels, 12 green/diagnostic-grade). Confidence: green = diagnostic, amber = moderate.

12 panels:

Panel	Rating
Adult-onset neurological disorders	green
DDG2P	green
Dystonia, chorea or related movement disorder, adult onset	green
Early onset dementia (encompassing fronto-temporal dementia and prion disease)	green
Fetal anomalies	green
Inherited white matter disorders	green
Leukodystrophy, adult onset	green
Leukodystrophy, childhood onset	green
Neurodegenerative disorders, adult onset	green
Paediatric disorders	green
Parkinson Disease and Complex Parkinsonism	green
White matter disorders and cerebral calcification - childhood onset	green

Mondo (8): 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), neurodegenerative disease (MONDO:0005559), Alzheimer disease (MONDO:0004975), (MONDO:0009096), early-onset calcifying leukoencephalopathy-skeletal dysplasia (MONDO:0034143)

Orphanet (3): Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (Orphanet:313808), Early-onset autosomal dominant Alzheimer disease (Orphanet:1020), NON RARE IN EUROPE: Alzheimer disease (Orphanet:238616)

HPO phenotypes

showing 60 of 79 phenotypes (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
HP:0001591	Bell-shaped thorax
HP:0002015	Dysphagia
HP:0002063	Rigidity
HP:0002067	Bradykinesia
HP:0002119	Ventriculomegaly
HP:0002171	Gliosis
HP:0002172	Postural instability
HP:0002180	Neurodegeneration
HP:0002186	Apraxia
HP:0002280	Enlarged cisterna magna
HP:0002283	Global brain atrophy
HP:0002300	Mutism
HP:0002352	Leukoencephalopathy
HP:0002354	Memory impairment
HP:0002362	Shuffling gait
HP:0002465	Poor speech
HP:0002500	Abnormal cerebral white matter morphology
HP:0002510	Spastic tetraplegia
HP:0002514	Cerebral calcification
HP:0002529	Neuronal loss in central nervous system
HP:0002808	Kyphosis
HP:0002901	Hypocalcemia
HP:0003016	Metaphyseal widening
HP:0003034	Diaphyseal sclerosis
HP:0003474	Somatic sensory dysfunction
HP:0003577	Congenital onset
HP:0003581	Adult onset
HP:0003596	Middle age onset
HP:0003621	Juvenile onset
HP:0003676	Progressive

GWAS associations

17 associations:

Study	Trait	p-value
GCST90002394_346	Monocyte percentage of white cells	2e-84
GCST90002393_661	Monocyte count	5e-67
GCST90002393_663	Monocyte count	2e-63
GCST004609_6	Monocyte percentage of white cells	8e-25
GCST004625_116	Monocyte count	4e-23
GCST004608_16	Granulocyte percentage of myeloid white cells	8e-21
GCST004609_5	Monocyte percentage of white cells	3e-20
GCST004608_17	Granulocyte percentage of myeloid white cells	2e-19
GCST90002394_344	Monocyte percentage of white cells	2e-19
GCST90002399_246	Neutrophil percentage of white cells	1e-17
GCST004625_115	Monocyte count	1e-12
GCST90002393_662	Monocyte count	6e-12
GCST90002389_188	Lymphocyte percentage of white cells	2e-10
GCST90002394_345	Monocyte percentage of white cells	9e-10
GCST90011899_171	Aspartate aminotransferase levels	1e-9
GCST012146_11	Hemoglobin levels	2e-6
GCST006585_1777	Blood protein levels	8e-6

EFO canonical traits (from GWAS)

7 canonical traits:

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

3 disease descriptors:

Descriptor	Name	Tree numbers
D000544	Alzheimer Disease	C10.228.140.380.100; C10.574.945.249; F03.615.400.100
D019636	Neurodegenerative Diseases	C10.574
C580150	Hereditary Diffuse Leukoencephalopathy with Spheroids *(supp.)*

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (1): CHEMBL1844 (SINGLE PROTEIN)

Drugs by curated mechanism of action (ChEMBL)

Drugs whose curated ChEMBL mechanism of action targets the CSF1R protein itself — single-protein targets only (drugs acting on a protein family or complex that merely includes it are excluded). Includes antibodies and oligonucleotides that bioactivity screening does not capture.

18 curated-mechanism drugs:

Drug	Modality	Max phase
AXATILIMAB	Antibody	4
PAZOPANIB HYDROCHLORIDE	Small molecule	4
PEXIDARTINIB	Small molecule	4
QUIZARTINIB	Small molecule	4
SUNITINIB	Small molecule	4
EMACTUZUMAB	Antibody	3
LINIFANIB	Small molecule	3
SERALUTINIB	Small molecule	3
VATALANIB	Small molecule	3
ARRY-382	Small molecule	2
CABIRALIZUMAB	Antibody	2
EDICOTINIB	Small molecule	2
ENMD-2076	Small molecule	2
AMG-820	Unknown	1
CT-1578	Small molecule	1
IMC-CS4	Unknown	1
PLX-5622	Small molecule	1
PLX-7486	Unknown	1

Molecules with ChEMBL bioactivity

Phase ≥1, by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 410,106 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

showing 60 of 79 molecules by phase:

Molecule	Name	Phase	Patents
CHEMBL477772	PAZOPANIB	4	16,241
CHEMBL3813873	PEXIDARTINIB	4	3,751
CHEMBL576982	QUIZARTINIB	4	4,647
CHEMBL535	SUNITINIB	4	80,232
CHEMBL1567	SUNITINIB MALATE	4	166
CHEMBL1289926	AXITINIB	4	16,510
CHEMBL288441	BOSUTINIB	4	12,869
CHEMBL3545311	BRIGATINIB	4	5,988
CHEMBL601719	CRIZOTINIB	4	15,133
CHEMBL5416410	DASATINIB	4	674
CHEMBL1421	DASATINIB ANHYDROUS	4	55,947
CHEMBL1983268	ENTRECTINIB	4	3,737
CHEMBL1287853	FEDRATINIB	4	3,770
CHEMBL3301607	FILGOTINIB	4	3,101
CHEMBL1873475	IBRUTINIB	4	8,363
CHEMBL941	IMATINIB	4	113,105
CHEMBL608533	MIDOSTAURIN	4	7,641
CHEMBL180022	NERATINIB	4	9,891
CHEMBL255863	NILOTINIB	4	39,362
CHEMBL502835	NINTEDANIB	4	8,978
CHEMBL2035187	PACRITINIB	4
CHEMBL1171837	PONATINIB	4
CHEMBL1336	SORAFENIB	4
CHEMBL24828	VANDETANIB	4
CHEMBL223360	LINIFANIB	3
CHEMBL101253	VATALANIB	3
CHEMBL428690	ALVOCIDIB	3
CHEMBL377300	BRIVANIB	3
CHEMBL491473	CEDIRANIB	3
CHEMBL1879463	DACTOLISIB	3
CHEMBL522892	DOVITINIB	3
CHEMBL603469	LESTAURTINIB	3
CHEMBL1908391	MASITINIB	3
CHEMBL572881	MOTESANIB	3
CHEMBL5314535	PIMICOTINIB	3
CHEMBL276711	SEMAXANIB	3
CHEMBL5095202	VIMSELTINIB	3
CHEMBL3644252	ARRY-382	2
CHEMBL3674570	EDICOTINIB	2
CHEMBL482968	ENMD-2076	2
CHEMBL3977543	BELVARAFENIB	2
CHEMBL575448	BMS-754807	2
CHEMBL3586573	CC-115	2
CHEMBL1967878	CENISERTIB	2
CHEMBL2010872	CEP-11981	2
CHEMBL2029988	CEP-32496	2
CHEMBL4116008	CERDULATINIB	2
CHEMBL215152	DEFOSBARASERTIB	2
CHEMBL4297187	DERAZANTINIB	2
CHEMBL103667	DORAMAPIMOD	2
CHEMBL5095061	ELZOVANTINIB	2
CHEMBL5882296	ENRUPATINIB	2
CHEMBL1230609	FORETINIB	2
CHEMBL3673452	GZ-389988	2
CHEMBL1980297	ILORASERTIB	2
CHEMBL5095102	INIXACICLIB	2
CHEMBL3115681	NARAZACICLIB	2
CHEMBL119385	NEFLAMAPIMOD	2
CHEMBL3586404	ONATASERTIB	2
CHEMBL607707	PELITINIB	2

PharmGKB: 1 entry (CPIC guideline: no)

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)

2575 measured of 3241 human assays (3249 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
3-[2-(Imidazo[1,2-b]pyridazin-3-yl)ethynyl]-4-methyl-N-{4-[(4-methylpiperazin-1-yl)methyl]-3-(trifluoromethyl)phenyl}benzamide	EC50	0.05 nM
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-11406644: 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
(11S)-16-amino-2-ethyl-6-fluoro-11-methyl-14-oxo-10-oxa-2,13,17,18,21-pentazatetracyclo[13.5.2.04,9.018,22]docosa-1(21),4(9),5,7,15(22),16,19-heptaene-5-carbonitrile	IC50	0.14 nM	US-20240132517: MACROCYCLIC COMPOUND, PHARMACEUTICAL COMPOSITION, AND USE 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-11406644: 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-11406644: 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-11406644: 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-11406644: 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-11406644: 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-11406644: Tropomyosin-related kinase (TRK) inhibitors

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

1810 with measured affinity, of 4159 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	decreases expression, affects cotreatment	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 expression, decreases reaction	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)

Via MONDO — disease-level, not drug-specific.

showing 60 of 288 trials:

NCT	Phase	Status	Title
NCT01662414	Phase 4	COMPLETED	Effect of Undenatured Cysteine-Rich Whey Protein Isolate (HMS 90®) in Patients With Parkinson’s Disease
NCT04871464	Phase 4	UNKNOWN	Role and Mechanism of Probiotics in Improving Motor Symptoms in Mild to Moderate Parkinson’s Disease
NCT05357612	Phase 4	RECRUITING	Characterization of the Serotonin 2A Receptor Selective PET Tracer [18F]MH.MZ in Patients With Neurodegenerative Diseases
NCT00722436	Phase 4	TERMINATED	Tranexamic Acid for Craniofacial Surgery
NCT02188576	Phase 4	COMPLETED	The Efficacy and Population Pharmacokinetics of Tranexamic Acid for Craniosynostosis Surgery
NCT00009191	Phase 4	COMPLETED	The Depression in Alzheimer’s Disease Study (DIADS)
NCT00009217	Phase 4	COMPLETED	Treatment of Behavioral Symptoms in Alzheimer’s Disease
NCT00018278	Phase 4	COMPLETED	Electrophysiologic Measures of Treatment Response in Alzheimer Disease
NCT00035204	Phase 4	COMPLETED	A Study of the Effects on Sleep, Attention, and Gastrointestinal Tolerance of Galantamine and Donepezil in Patients With Alzheimer’s Disease
NCT00042172	Phase 4	COMPLETED	Treatment for Early Memory Loss
NCT00046358	Phase 4	COMPLETED	The Effect of Short-Term Statins and NSAIDs on Levels of Beta-Amyloid, a Protein Associated With Alzheimer’s Disease
NCT00104442	Phase 4	COMPLETED	Study of the Effects of Current Drug Treatments on Levels of Certain Brain Chemicals in Alzheimer’s Disease
NCT00120874	Phase 4	COMPLETED	Memantine and Comprehensive, Individualized Management of Alzheimer’s Disease and Caregiver Training
NCT00142324	Phase 4	UNKNOWN	CALM-AD
NCT00165724	Phase 4	COMPLETED	Alzheimer’s Disease Long-term Follow-up Study (ALF Study)
NCT00165750	Phase 4	TERMINATED	Correlation Between Regional Brain Volume and Response to Donepezil Treatment in AD Patients
NCT00202124	Phase 4	COMPLETED	Double Blind Study of Trp01 in Patients With Alzheimer’s Disease
NCT00208819	Phase 4	COMPLETED	A Comparison of Two Standard Therapies in the Management of Dementia With Agitation
NCT00216515	Phase 4	COMPLETED	The Efficacy of Galantamine on the Attention and the Frontal Function of the Patients With Dementia of Alzheimer Type
NCT00230568	Phase 4	COMPLETED	EARTH 413: A Study of Aricept in Hispanic Patients With Mild to Moderate Alzheimer’s Disease (AD)
NCT00234637	Phase 4	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	Phase 4	COMPLETED	Side Effects of Newer Antipsychotics in Older Adults
NCT00254033	Phase 4	COMPLETED	Apathy Associated With Alzheimer’s Disease
NCT00260624	Phase 4	COMPLETED	Escitalopram Treatment of Patients With Agitated Dementia
NCT00303277	Phase 4	COMPLETED	Do HMG CoA Reductase Inhibitors Affect Abeta Levels?
NCT00305903	Phase 4	COMPLETED	Safety and Tolerability of Rivastigmine With Add-on Memantine in Patients With Probable Alzheimer’s Disease
NCT00306124	Phase 4	UNKNOWN	Dopaminergic Enhancement of Learning and Memory in Healthy Adults and Patients With Dementia/Mild Cognitive Impairment
NCT05508789	Phase 3	ACTIVE_NOT_RECRUITING	A Study of Donanemab (LY3002813) in Participants With Early Symptomatic Alzheimer’s Disease (TRAILBLAZER-ALZ 5)
NCT05738486	Phase 3	ACTIVE_NOT_RECRUITING	A Study of Different Donanemab (LY3002813) Dosing Regimens in Adults With Early Alzheimer’s Disease (TRAILBLAZER-ALZ 6)
NCT06111014	Phase 3	TERMINATED	Continuation Study for Latozinemab
NCT06672237	Phase 3	RECRUITING	A Phase 3 Study of NTLA-2001 in ATTRv-PN
NCT01010932	Phase 3	COMPLETED	Carotid and Vertebral Magnetic Resonance Angiography (MRA) Study Comparing Dotarem and Time Of Flight (TOF)
NCT01012674	Phase 3	COMPLETED	Carotid and Vertebral Magnetic Resonance Angiography (MRA) Study Comparing Dotarem and Time Of Flight (TOF)
NCT02455440	Phase 3	COMPLETED	Esmolol Infusion in Patients Undergoing Craniotomy
NCT05677659	Phase 2	TERMINATED	A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
NCT00686699	Phase 2	TERMINATED	Study of Preladenant for the Treatment of Antipsychotic Induced Movement Disorders in Participants With Schizophrenia (Study P04628)
NCT01385592	Phase 2	COMPLETED	Evaluation of the Efficacy and Safety of AFQ056 in Parkinson’s Patients With L-dopa Induced Dyskinesias
NCT01491529	Phase 2	COMPLETED	Evaluation of the Efficacy and Safety of Modified Release AFQ056 in Parkinson’s Patients With L-dopa Induced Dyskinesias
NCT01491932	Phase 2	COMPLETED	Open-label, Long-term Safety Extension Study of AFQ056 in Parkinson’s Patients With L-dopa Induced Dyskinesias
NCT04727658	Phase 2	TERMINATED	Linac FRACtionated Radiosurgical THALamotomie in Tremors (FRACTHAL)
NCT00001365	Phase 2	COMPLETED	Dextromethorphan for the Treatment of Parkinson’s Disease and Similar Conditions of the Nervous System
NCT00406029	Phase 2	COMPLETED	Dyskinesia in Parkinson’s Disease (Study P04501)
NCT00537017	Phase 2	COMPLETED	Follow Up Safety Study of SCH 420814 in Subjects With Parkinson’s Disease (P05175)
NCT00907283	Phase 2	UNKNOWN	Ferrochelating Treatment in Patients Affected by Neurodegeneration With Brain Iron Accumulation (NBIA)
NCT01518374	Phase 2	COMPLETED	Clinical Evaluation of Florbetapir F 18 (18F-AV-45)
NCT02656498	Phase 2	COMPLETED	[18F]THK-5351 Positron Emission Computed Tomography Study of Normal, Mild Cognitive Impairment, Alzheimer’s Disease and Other Neurodegenerative Disease
NCT03127514	Phase 2	COMPLETED	AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT03538522	Phase 2	COMPLETED	A Double-Blind, Placebo-Controlled Safety and Efficacy Study of NA-831
NCT04838301	Phase 2	ACTIVE_NOT_RECRUITING	Allopregnanolone Regenerative Therapeutic for Mild Alzheimer’s Disease
NCT04937452	Phase 2	COMPLETED	Dopaminergic Therapy for Frontotemporal Dementia Patients
NCT05318976	Phase 2	COMPLETED	A Study of XPro1595 in Patients With Early Alzheimer’s Disease With Biomarkers of Inflammation
NCT05321498	Phase 2	WITHDRAWN	Study to Assess the Efficacy of XPro1595 in Patients With Mild Cognitive Impairment With Biomarkers of Inflammation
NCT05479981	Phase 2	COMPLETED	Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
NCT05522387	Phase 2	TERMINATED	An Open-Label Extension of XPro1595 in Patients With Alzheimer’s Disease
NCT02229968	Phase 2	ACTIVE_NOT_RECRUITING	Efficacy of Amicar for Children Having Craniofacial Surgery
NCT03065192	Phase 1	COMPLETED	Safety and Efficacy Study of VY-AADC01 for Advanced Parkinson’s Disease
NCT07718659	Phase 1	NOT_YET_RECRUITING	ZR001 Chemogenetics Gene Therapy Study in Patients With Parkinson’s Disease
NCT00316797	Phase 1	COMPLETED	Biodistribution and Safety of a Radiopharmaceutical in Healthy Subjects
NCT01758510	Phase 1	COMPLETED	Safety Study of HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
NCT02267434	Phase 1	COMPLETED	Study Assessing Tolerability and Safety of AFFITOPE® PD03A in Patients With Early Parkinson’s Disease

Predicted chemistry — Sugi Predict

The predicted patent-compound landscape for CSF1R across ~30M SureChEMBL compounds — explore on Sugi Predict.