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Palifermin is a recombinant human keratinocyte growth factor (KGF) produced using recombinant DNA technology in Escherichia coli. It is used to prevent and reduce the severity of oral mucositis (severe mouth and throat sores) in patients with hematologic malignancies undergoing high-dose chemotherapy and radiation therapy followed by stem cell support. Palifermin acts primarily as an agonist of the fibroblast growth factor receptor 2b (FGFR2b), which is expressed on epithelial cells. By binding to this receptor, it stimulates proliferation, differentiation, migration, and cytoprotection of epithelial cells in various tissues including the oral mucosa. This results in increased thickness of the mucosal epithelium, upregulation of protective enzymes, antiapoptotic effects, reduced angiogenesis, and modulation of cytokine profiles. The drug was developed for use specifically in blood or bone marrow cancers requiring myelotoxic therapy with hematopoietic stem cell support; its efficacy has not been established for non-hematologic malignancies.
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