OnCo
targetsTarget

CSF1R

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.

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.

CSF1R: what it is and how drugs act on it · animated generic schematic, not to scale
  • Target · the protein and the cell it sits on
  • Drug · antibody, small molecule, cell or radioligand
  • Effect · signal, damage or kill

In plain words · 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.

  1. 1 · What it is

    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.

  2. 2 · What goes wrong in cancer

    Type III receptor tyrosine kinase for CSF1 and IL-34; controls differentiation, survival and function of monocytes, macrophages, osteoclasts and microglia.

  3. 3 · How drugs use it

    1 product aims at CSF1R: small molecules. Kinases are switched on by binding ATP inside the cell, so most drugs are small molecules shaped to plug that ATP pocket.

Biology

Type III receptor tyrosine kinase for CSF1 and IL-34; controls differentiation, survival and function of monocytes, macrophages, osteoclasts and microglia.

Where it is found
  • Tenosynovial giant cell tumour (CSF1 translocation in neoplastic cells; CSF1R+ reactive mass)
  • Tumour-associated macrophages in most solid tumours
  • Glioblastoma microglia
  • Chronic GVHD macrophages
Class
kinase · CSF1R

Products

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Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

Query for this target: (TITLE:"CSF1R" OR ABSTRACT:"CSF1R") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about CSF1R, not a curated reading list.

Connected

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