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ML00253764 is a brain-penetrant, nonpeptidic small molecule antagonist of the melanocortin 4 receptor (MC4R). Originally developed by Millennium Pharmaceuticals, it was identified for its potential to prevent tumor-induced weight loss (cachexia) and regulate energy homeostasis. More recently, ML00253764 has been investigated for its direct anticancer properties, demonstrating antiproliferative and proapoptotic activities in glioblastoma and breast cancer cell lines, particularly when combined with metronomic chemotherapy such as temozolomide.
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