一种 PAK抑制剂,对 PAK1,PAK2 和 PAK3 的 IC50 值分别为 14,33 和 39 nM。
FRAX486 is a potent p21-activated kinase (PAK) inhibitor (IC50 values are 14, 33, 39 and 575 nM for PAK1, PAK2, PAK3 and PAK4 respectively). FRAX486 rescues seizures and behavioral abnormalities such as hyperactivity and repetitive movements, thereby supporting the hypothesis that a drug treatment that reverses the spine abnormalities can also treat neurological and behavioral symptoms. A single administration of FRAX486 is sufficient to rescue all of these phenotypes in adult Fmr1 KO mice, demonstrating the potential for rapid, postdiagnostic therapy in adults with FXS. FRAX486 ameliorate schizophrenia-associated dendritic spine deterioration in vitro and in vivo during late adolescence.