MPT0G521

MPT0G521 is a brain-penetrant small molecule dual inhibitor of lysine-specific demethylase 1 (KDM1A/LSD1) and histone deacetylase (HDAC), specifically targeting HDAC2. Developed by researchers in Taiwan, including those from Taipei Medical University, Shuang Ho Hospital, and Keelung Chang Gung Medical Foundation, the compound is designed to address therapy-refractory glioblastoma (GBM). By co-targeting KDM1A and HDAC2, MPT0G521 aims to reverse epigenetic adaptations that drive resistance to standard temozolomide (TMZ) and radiotherapy. Preclinical studies have demonstrated that MPT0G521 can cross the blood-brain barrier and produce dose-dependent growth inhibition, induce G2/M arrest, and trigger apoptosis in TMZ-resistant GBM cell lines and patient-derived models.