Drug intelligence / Profile preview

MSC-GM-CSF

Development stage
Preclinical
Lead developer
Mass General Brigham
Modality
Stem Cell Therapies → Cell Therapies, Cytokines & Interferons → Recombinant Proteins and Enzymes
Administration
Intravenous, Intratumoral
01

Overview

MSC-GM-CSF is an engineered cell therapy construct consisting of mesenchymal stromal/stem cells (MSCs) modified to overexpress granulocyte-macrophage colony-stimulating factor (GM-CSF). This approach utilizes the innate tumor-homing capabilities of MSCs to deliver the immunostimulatory cytokine GM-CSF directly into the tumor microenvironment. GM-CSF acts as a potent cytokine that promotes the differentiation of myeloid cells into dendritic cells and shifts macrophage polarization toward a proinflammatory M1 phenotype, thereby enhancing antitumor immune responses. Preclinical research has primarily focused on its application in hepatocellular carcinoma (HCC) and advanced melanoma, including brain metastases. It is frequently studied in combination with other therapeutic modalities, such as oncolytic herpes simplex virus (oHSV) or chemotherapy, to overcome the immunosuppressive nature of the tumor microenvironment.

Other names
GM-CSF-expressing mesenchymal stem cellsGM-CSF-modified MSCsMSC releasing GM-CSF
02

Targets

CSF2R (Granulocyte-macrophage colony-stimulating factor receptor)

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