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Mefloquine + temozolomide is an investigational combination therapy consisting of the antimalarial agent mefloquine and the alkylating chemotherapy agent temozolomide. This combination has been studied primarily in patients with newly diagnosed glioblastoma, often as part of multi-drug regimens that may also include memantine and metformin. Temozolomide acts as a DNA-alkylating agent, leading to tumor cell death by inducing DNA damage through methylation of guanine residues, which triggers futile repair cycles and apoptosis. Mefloquine is thought to enhance the anticancer effects of temozolomide by sensitizing tumor cells, potentially through disruption of lysosomal function or autophagy pathways. Clinical studies have demonstrated that this combination can be administered safely at defined doses in glioblastoma patients following chemoradiation[1][2][6]. The primary indication under investigation is glioblastoma.
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