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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.
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