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Folate-appended methyl-beta-cyclodextrin (FA-M-beta-CyD) is a targeted anticancer research compound developed by investigators at Kumamoto University. It consists of a methyl-beta-cyclodextrin (M-beta-CyD) core conjugated with folic acid to provide selectivity for cells overexpressing the folate receptor (FR), particularly folate receptor-alpha (FR-alpha). The compound functions by binding to FR-alpha on the surface of tumor cells, leading to its internalization. Once inside the cell, it induces autophagy-mediated cell death and mitophagy. Preclinical studies in mouse models have demonstrated that FA-M-beta-CyD significantly inhibits tumor growth in FR-positive malignancies, including colorectal, ovarian, and lung cancers, with efficacy comparable or superior to conventional chemotherapeutic agents like doxorubicin.
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