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AMC-01 is a small molecule piperazine oxalate derivative developed by researchers at the Asan Medical Center. It acts as a potent modulator of the Unfolded Protein Response (UPR) by inducing the phosphorylation of eukaryotic translation initiation factor 2-alpha (eIF2-alpha) at the Serine 51 residue. This mechanism leads to the inactivation of global protein translation, which triggers apoptosis in cancer cells, particularly those under endoplasmic reticulum (ER) stress. Preclinical studies have demonstrated its anti-tumor efficacy in models of breast cancer and liver cancer, where it exploits the metabolic stress pathways common in solid tumors.
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