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Malformin C is a marine-derived fungal cyclic peptide, originally isolated from *Aspergillus niger*, that exhibits potent anti-tumor activity against glioblastoma. It specifically targets glioblastoma stem-like cells (GSCs), which are typically resistant to standard chemo- and radiotherapy. The drug's mechanism of action involves the induction of proteotoxic stress and the unfolded protein response (UPR) by binding to various proteins and causing aggregation. Additionally, malformin C accumulates in lysosomes, leading to a blockade of autophagic flux. This dual action drives GSCs toward cell death. Preclinical studies in xenograft models have demonstrated significant reduction in tumor growth, highlighting its potential as a therapeutic agent for aggressive brain tumors.
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