{"entity":{"id":"csf1r","kind":"target","name":"CSF1R","aka":[],"tldr":"The receptor that macrophages depend on; blocking it shrinks tenosynovial giant cell tumour (a CSF1-driven tumour) and depletes tumour-supporting macrophages, though the latter has not yet helped patients with common cancers.","summary":"CSF1R signalling sustains macrophages and microglia. Tenosynovial giant cell tumour is driven by CSF1 translocation in a minority of cells that recruit a CSF1R-positive mass: pexidartinib (2019) and vimseltinib (2025) are approved; emactuzumab and cabiralizumab (antibodies) showed activity. In solid tumours, CSF1R blockade to deplete tumour-associated macrophages and combine with PD-1 inhibitors (cabiralizumab-nivolumab in pancreatic cancer) was disappointing. Axatilimab (anti-CSF1R) was approved in 2024 for chronic GVHD, targeting macrophage-driven fibrosis.","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Colony_stimulating_factor_1_receptor","links":[{"label":"ENLIVEN (Lancet 2019)","url":"https://doi.org/10.1016/S0140-6736(19)30764-0"}],"tags":["gap-fill"],"related":["kit","pdgfra"],"cancers":["sarcoma","pancreatic","glioblastoma"],"sections":[],"technologies":["kinase-inhibitors","monoclonal-antibody"],"targets":[],"drugs":["pexidartinib","vimseltinib","emactuzumab","chiauranib"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-tap-lancet"],"journals":[],"dependsOn":[],"notes":["In pancreatic cancer and glioblastoma the target is on tumour-associated macrophages and microglia rather than tumour cells, so no tumour-cell prevalence is recorded."],"symbol":"CSF1R","role":[],"sources":[],"specificity":"immune-microenvironment","distribution":"few-types","specificityNote":"Immune or microenvironment target: its medicines act on immune, stromal or bone cells rather than on the tumour cell (drug mechanisms in the corpus). HPA CSF1R: RNA tissue enhanced (lymphoid tissue 107 nTPM, placenta 97 nTPM); blood lineage lineage enriched (monocytes 650 nTPM); no normal tissue stained high; highest cancer staining ovarian cancer (1 of 9 high). Distribution: 3 cancer families in the corpus carry a prevalence row, label threshold or catalogue link for it (Sarcomas (soft tissue, bone, GIST), Pancreatic ductal adenocarcinoma, Brain and spinal cord tumours (all types)); Open Targets associates it with 5 specific cancer types at or above 0.5 (gastrointestinal stromal tumor, renal cell carcinoma, pigmented villonodular synovitis, soft tissue sarcoma, tenosynovial giant cell tumor). (Rule 1 of scripts/fetch-target-specificity.ts.)","specificitySources":[{"label":"Human Protein Atlas CSF1R tissue","url":"https://www.proteinatlas.org/ENSG00000182578-CSF1R/tissue","note":"RNA tissue and blood lineage specificity, normal tissue antibody staining (version 25.1, CC BY-SA 3.0)"},{"label":"UniProt P07333","url":"https://www.uniprot.org/uniprotkb/P07333/entry","note":"involvement in disease"},{"label":"Open Targets ENSG00000182578 associations","url":"https://platform.opentargets.org/target/ENSG00000182578/associations","note":"cancer associations at or above 0.5 (CC0)"}],"hgnc":"HGNC:2433","ensembl":"ENSG00000182578","uniprot":"P07333","entrez":"1436","firstDescribed":1985,"firstDescribedBasis":"sequence","firstDescribedNote":"Earliest sequence paper UniProt cites for the protein: Nienhuis A.W. et al, Cell, 1985, \"Expression of the human c-fms proto-oncogene in hematopoietic cells and its deletion in the 5q- syndrome\".","firstDescribedSource":"https://pubmed.ncbi.nlm.nih.gov/4028159/","biology":"Type III receptor tyrosine kinase for CSF1 and IL-34; controls differentiation, survival and function of monocytes, macrophages, osteoclasts and microglia.","whereFound":["Tenosynovial giant cell tumour (CSF1 translocation in neoplastic cells; CSF1R+ reactive mass)","Tumour-associated macrophages in most solid tumours","Glioblastoma microglia","Chronic GVHD macrophages"],"targetClass":"kinase","prevalence":[{"cancerId":"sarcoma","pct":100,"measure":"CSF1 overexpression by ISH/IHC in tenosynovial giant cell tumour / PVNS (57 of 57); CSF1 translocation in 61%","source":"https://doi.org/10.1097/PAS.0b013e31802b86f8","note":"TGCT only, where the CSF1R-positive macrophage mass is the tumour bulk (West 2006, doi:10.1073/pnas.0507321103)"}]},"route":"/targets/csf1r/","neighbours":{"target":[{"id":"kit","kind":"target","name":"KIT","route":"/targets/kit/"},{"id":"pdgfra","kind":"target","name":"PDGFRA","route":"/targets/pdgfra/"}],"cancer":[{"id":"glioblastoma","kind":"cancer","name":"Glioma & glioblastoma","route":"/cancers/glioblastoma/"},{"id":"pancreatic","kind":"cancer","name":"Pancreatic ductal adenocarcinoma","route":"/cancers/pancreatic/"},{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"},{"id":"tenosynovial-giant-cell-tumour","kind":"cancer","name":"Tenosynovial giant cell tumour (TGCT)","route":"/cancers/tenosynovial-giant-cell-tumour/"}],"technology":[{"id":"monoclonal-antibody","kind":"technology","name":"Monoclonal antibodies","route":"/technologies/monoclonal-antibody/"},{"id":"kinase-inhibitors","kind":"technology","name":"Small-molecule kinase inhibitors","route":"/technologies/kinase-inhibitors/"}],"drug":[{"id":"chiauranib","kind":"drug","name":"Chiauranib","route":"/drugs/chiauranib/"},{"id":"emactuzumab","kind":"drug","name":"Emactuzumab","route":"/drugs/emactuzumab/"},{"id":"pexidartinib","kind":"drug","name":"Pexidartinib","route":"/drugs/pexidartinib/"},{"id":"surufatinib","kind":"drug","name":"Surufatinib","route":"/drugs/surufatinib/"},{"id":"vimseltinib","kind":"drug","name":"Vimseltinib","route":"/drugs/vimseltinib/"}],"paper":[{"id":"paper-tap-lancet","kind":"paper","name":"Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial","route":"/key-papers/paper-tap-lancet/"},{"id":"paper-quail-joyce-microenvironment-metastasis-natmed-2013","kind":"paper","name":"Quail and Joyce 2013: microenvironmental regulation of tumour progression and metastasis","route":"/key-papers/paper-quail-joyce-microenvironment-metastasis-natmed-2013/"}],"pathway":[{"id":"immune-desert-exclusion","kind":"pathway","name":"Cold tumours: immune deserts and exclusion","route":"/pathways/immune-desert-exclusion/"},{"id":"complement-in-cancer","kind":"pathway","name":"Complement in cancer","route":"/pathways/complement-in-cancer/"},{"id":"intravasation-ctc-survival","kind":"pathway","name":"Intravasation & circulating tumour cells","route":"/pathways/intravasation-ctc-survival/"},{"id":"myeloid-suppression-axis","kind":"pathway","name":"Myeloid suppression: TAMs, MDSCs & don't-eat-me signals","route":"/pathways/myeloid-suppression-axis/"},{"id":"nutrient-competition-tme","kind":"pathway","name":"Nutrient competition & metabolic immunosuppression","route":"/pathways/nutrient-competition-tme/"},{"id":"angiogenic-switch","kind":"pathway","name":"The angiogenic switch & tumour vessels","route":"/pathways/angiogenic-switch/"},{"id":"pre-metastatic-niche","kind":"pathway","name":"The pre-metastatic niche","route":"/pathways/pre-metastatic-niche/"}],"company":[{"id":"abbisko","kind":"company","name":"Abbisko Therapeutics","route":"/companies/abbisko/"},{"id":"qurient","kind":"company","name":"Qurient","route":"/companies/qurient/"}],"trial":[{"id":"enliven","kind":"trial","name":"ENLIVEN","route":"/trials/enliven/"}]}}