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AOP208 is a first-in-class, orally available small molecule antagonist of the serotonin 1B receptor (5-HT1B), specifically designed to target cancer stem cells. By blocking a particular conformation of the serotonin 1B receptor on the membrane of cancer stem cells, AOP208 disrupts metabolic pathways that are believed to be crucial for tumor initiation and maintenance. This novel mechanism aims to address the root cause of various cancers by eradicating cancer stem cells rather than just reducing tumor bulk. The drug was initially developed by Leukos Biotech based on research at the Josep Carreras Leukemia Research Institute and is now being co-developed with AOP Orphan Pharmaceuticals (AOP Health). It is currently in phase I clinical trials for solid tumors and lymphomas, with preclinical development ongoing for acute myeloid leukemia and myelodysplastic syndromes[2][4][5][6][7][9].
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