96
α-synuclein inhibitor_Neurodegenerative Disorders
University of California, San Diego
Small molecule
Neurology, CNS disease
Hit To Lead or Lead Optimization
Researchers at UC San Diego have developed a novel strategy to treat PD by using small molecules interacting with native α-Syn that could protect and stabilize specific conformations present in the ensemble, which in turn could provide protective action. A compound has been identified that displayed protective activity in preventing the transmission of α-Syn from cell to cell thus supporting the notion that small molecules can target an intrinsically disordered protein such as α-Syn. This compound does not directly affect the process of α-Syn misfolding or aggregation and thus offers a novel mode-of-action beyond previously described aggregation blockers.
