CSF1R colony stimulating factor 1 receptor
ENSG00000182578 · protein coding · Open Targets profile
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 (PubMed:20504948, PubMed:30982609). 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 (PubMed:30982608). {ECO:0000269|PubMed:12882960, ECO:0000269|PubMed:15117969, ECO:0000269|PubMed:16170366, ECO:0000269|PubMed:16337366, ECO:0000269|PubMed:16648572, ECO:0000269|PubMed:17121910, ECO:0000269|PubMed:18467591, ECO:0000269|PubMed:18814279, ECO:0000269|PubMed:19193011, ECO:0000269|PubMed:19934330, ECO:0000269|PubMed:20489731, ECO:0000269|PubMed:20504948, ECO:0000269|PubMed:20829061, ECO:0000269|PubMed:30982608, ECO:0000269|PubMed:30982609, ECO:0000269|PubMed:7683918}.
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Approved Drug, Structure with Ligand, High-Quality Ligand, High-Quality Pocket, Druggable Family
- Antibody
- Advanced Clinical, GO CC high conf, UniProt loc med conf, UniProt SigP or TMHMM, Human Protein Atlas loc
- PROTAC
- UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.36375001072883606 (upper bound 0.4735099971294403). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 206 registry trials, updated in the last three years, of the most advanced drugs against CSF1R (axatilimab, pazopanib, pexidartinib, quizartinib, sunitinib, vatalanib, seralutinib, linifanib, emactuzumab, edicotinib, ARRY-382, cabiralizumab), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Eric JonaschM.D. Anderson Cancer Center · 3 of 3 trials Phase 2+
- Musa YilmazM.D. Anderson Cancer Center · 3 of 3 trials Phase 2+Ph1/Ph2NCT04128748 · Liposomal Cytarabine and Daunorubicin (CPX-351) and Quizartinib for the Treatment of Acute Myeloid Leukemia and High Risk Myelodysplastic Syndromebrief
- Richard ArandaGossamer Bio USA, Inc. · 3 of 3 trials Phase 2+
- Amado ZuritaM.D. Anderson Cancer Center · 2 of 2 trials Phase 2+
- Arun RajanNational Cancer Institute (NCI) · 2 of 2 trials Phase 2+
- Juan BerguaHospital San Pedro Alcántara · 2 of 2 trials Phase 2+
- Pau MontesinosHospital Universitario La Fe · 2 of 2 trials Phase 2+
- Aaron R WeissChildren's Oncology Group · 1 of 1 trials Phase 2+
- Aiwu Ruth HeGeorgetown University · 1 of 1 trials Phase 2+
- Alain RavaudUniversity Hospital, Bordeaux, France · 1 of 1 trials Phase 2+
- Alba RubioHospital Infantil Universitario Niño Jesús · 1 of 1 trials Phase 2+
- Albert AssadIncyte Corporation · 1 of 1 trials Phase 2+
- Alejandro Pérez FidalgoHospital Clínico Universitario de Valencia · 1 of 1 trials Phase 2+
- Alex MenziesMelanoma Institute Australia · 1 of 1 trials Phase 2+
- Alexander MusteaUniversity Hospital, Bonn · 1 of 1 trials Phase 2+
Registry trials of axatilimab, pazopanib, pexidartinib, quizartinib, sunitinib, vatalanib, seralutinib, linifanib, emactuzumab, edicotinib, ARRY-382, cabiralizumab updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 146 of 202 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 4 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
200 trials of drugs against CSF1R were stopped early; 28 give no reason
Terminated, withdrawn and suspended interventional trials of axatilimab, pazopanib, pexidartinib, quizartinib, sunitinib, vatalanib, seralutinib, linifanib, emactuzumab, edicotinib, ARRY-382, cabiralizumab, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 200.
- terminated · 2026-11-05 · Gossamer Bio USA, Inc. · GB002 (seralutinib)“Enrollment has declined and remaining participants have been on study more than three years. Continued study visits/data collection are no longer considered necessary. This is not expected to affect study interpretation or the overall benefit-risk.”
- suspended · 2026-10-05 · M.D. Anderson Cancer Center · Axatilimab (SNDX-6352)“PI Request”
- withdrawn · 2026-09-03 · Mayo Clinic · Axatilimab“Sponsor declined to fund study due to inability to agree on IP.”
- terminated · 2026-08-25 · Association of Urologic Oncology (AUO) · SunitinibNo reason given
- terminated · 2026-07-09 · Washington University School of Medicine · Cabiralizumab“BMS terminated the study”
- terminated · 2026-04-14 · Institut Claudius Regaud · Pazopanib“Slow recruitment rate”
- suspended · 2026-03-19 · GB002, Inc. · Seralutinib“Sponsor decision”
- terminated · 2025-10-16 · NYU Langone Health · Nivolumab 240 mg IV every 2 weeks + Cabiralizumab 4 mg/kg IV every 2 weeks“Study was terminated early by the drug sponsor due to slow accrual and a change in the company's drug development priorities”
- terminated · 2025-04-30 · University of Washington · Pazopanib hydrochloride“Study was terminated at completion of the Phase 1 dose escalation due to loss of financial support.”
- terminated · 2025-03-21 · Gulam Manji · matched by the registry's drug synonyms“Poor enrollment”
- terminated · 2025-03-07 · Aptose Biosciences Inc. · matched by the registry's drug synonyms“Change in corporate strategy”
- terminated · 2025-02-14 · AIO-Studien-gGmbH · matched by the registry's drug synonyms“insufficient accrual rate”
- terminated · 2025-02-12 · Georgetown University · sunitinib and capecitabine“Unanticipated side effects and futility”
- terminated · 2024-09-19 · M.D. Anderson Cancer Center · Quizartinib“This study was terminated early due to other competing trials, therefore did not go on to the Phase II portion of the study.”
- terminated · 2024-08-09 · Ludwig-Maximilians - University of Munich · Pazopanib, Placebo (for Pazopanib)“poor recruitment”
- terminated · 2024-06-27 · M.D. Anderson Cancer Center · matched by the registry's drug synonyms“Slow accrual.”
- terminated · 2024-06-25 · Brown University · Sunitinib“Funding withdrawn”
- terminated · 2024-06-05 · Theseus Pharmaceuticals · matched by the registry's drug synonyms“The Sponsor terminated the study due to early dose-limiting toxicities.”
- terminated · 2024-03-13 · M.D. Anderson Cancer Center · Emactuzumab“The study was terminated early by the Sponsor”
- terminated · 2024-03-04 · OHSU Knight Cancer Institute · Sunitinib“Low accrual”
- terminated · 2024-02-09 · Jiangsu HengRui Medicine Co., Ltd. · Sunitinib Capsules“R\&D strategy adjustment”
- terminated · 2024-01-05 · National Eye Institute (NEI) · Sunitinib Malate“Inability to recruit and adequate number of participants”
- terminated · 2023-12-05 · SCRI Development Innovations, LLC · Quizartinib“Closed due to slow enrollment”
- terminated · 2023-04-11 · Nektar Therapeutics · sunitinib“Sponsor decision”
- terminated · 2022-10-12 · Ludwig Institute for Cancer Research · Sunitinib malate“Closed prematurely due to toxicity”
Paediatric incentives and obligations for CSF1R's drugs
For axatilimab, pazopanib, pexidartinib, quizartinib, sunitinib, vatalanib, seralutinib, linifanib, emactuzumab, edicotinib, ARRY-382, cabiralizumab: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: yes · CSF1R is on the list. Membership alone neither requires nor waives a pediatric study.Rule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: yes · EMEA-003385-PIP01-22 (axatilimab, Treatment of chronic Graft versus Host Disease (cGvHD)): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 15 Apr 2025; EMEA-002972-PIP02-23 (seralutinib, Treatment of pulmonary arterial hypertension): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 13 Sept 2024; EMEA-001821-PIP01-15-M08 (quizartinib, Treatment of acute myeloid leukaemia): PM: decision on the application for modification of an agreed PIP, 14 Jun 2024; EMEA-003172-PIP01-21 (emactuzumab, Treatment of tenosynovial giant cell tumour): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 2 Dec 2022; +3 moreRule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: yes · EU/3/19/2158 positive 29 May 2019: Treatment of pulmonary arterial hypertension; EU/3/22/2591 positive 16 Mar 2022: Treatment of tenosynovial giant cell tumour, localised and diffuse type; EU/3/15/1457 withdrawn 19 Mar 2015: Treatment of tenosynovial giant cell tumour, localised and diffuse type; EU/3/16/1799 withdrawn 12 Dec 2016: Treatment of tenosynovial giant cell tumour, localised and diffuse type; +1 moreRule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
- Authorised in the EU: yes · Sunitinib Accord: authorised since 11 Feb 2021; Vanflyta: authorised since 6 Nov 2023; Votrient: authorised since 14 Jun 2010; Sutent: authorised since 19 Jul 2006; +2 moreRule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of axatilimab, pazopanib, pexidartinib, quizartinib, sunitinib, vatalanib, seralutinib, linifanib, emactuzumab, edicotinib, arry-382, cabiralizumabRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
- Registered studies open to children: yes · 63 interventional studies of axatilimab, pazopanib, pexidartinib, quizartinib, sunitinib, vatalanib, seralutinib, linifanib, emactuzumab, edicotinib, arry-382, cabiralizumab include patients under 18; 24 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 21 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.