Target intelligence / Profile preview

Granulocyte-macrophage colony-stimulating factor (CSF2) (GM-CSF)

Target
GM-CSF
Molecular classification
Cytokine, Growth factor, Hematopoietic growth factor
01

Overview

Granulocyte-macrophage colony-stimulating factor (CSF2), also known as GM-CSF, is a monomeric glycoprotein that functions as a potent cytokine and hematopoietic growth factor [1, 2]. It is produced by various cell types, including activated T cells, B cells, macrophages, endothelial cells, and fibroblasts, in response to immune stimuli and inflammatory cytokines like IL-1 or TNF-alpha [2, 4]. CSF2 plays a vital role in the survival, proliferation, and differentiation of myeloid progenitor cells into neutrophils, monocytes, and macrophages, and it is essential for the functional activity of mature myeloid cells [1, 4]. In the lungs, CSF2 is critical for the clearance of surfactant by alveolar macrophages; its deficiency or neutralization by autoantibodies leads to pulmonary alveolar proteinosis [1, 2]. Clinically, recombinant forms of CSF2, such as Sargramostim, are used to accelerate myeloid recovery in patients undergoing bone marrow transplantation or chemotherapy [3, 4]. Conversely, because CSF2 is a key mediator of tissue inflammation and the 'cytokine storm,' monoclonal antibodies targeting the CSF2 ligand are being investigated for the treatment of autoimmune diseases like rheumatoid arthritis and severe inflammatory complications of viral infections [5]. Sources: [1] UniProt (P04141); [2] NCBI Gene (ID: 1437); [3] DrugBank (DB00020); [4] StatPearls (Granulocyte Macrophage Colony Stimulating Factor); [5] PubMed (PMID: 32833830).

Other names
Colony-stimulating factor 2CSF-2MolgramostimSargramostimPluripoietin-alpha
02

Mechanism of action

Agonism of the CSF2 receptor to stimulate myeloid cell production and activation; or neutralization of the CSF2 ligand to inhibit pro-inflammatory signaling.

03

Biological functions

HematopoiesisMyeloid cell differentiationImmune responseInflammationCell proliferationAlveolar macrophage maturation
04

Disease associations

NeutropeniaRheumatoid arthritisPulmonary alveolar proteinosisCancerCytokine release syndromeInfection
05

Safety considerations

Capillary leak syndromeBone painFeverInjection site reactionsRespiratory distressHypersensitivity reactionsSplenomegaly
06

Interacting drugs

Sargramostim

6 more in the full profile.

07

Biomarkers

GM-CSF autoantibodiesAbsolute neutrophil count (ANC)C-reactive protein (CRP)Alveolar-arterial oxygen gradient

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