Description
FRAX486 is an inhibitor of PAK that is currently under investigation as a potential treatment for schizophrenia; it exhibits neuroprotective activity and anti-psychotic potential. In vitro, FRAX486 ameliorates synaptic deterioration induced by DISC1 knockdown. In animal models of fragile X syndrome, FRAX486 rescues seizures and other behavioral abnormalities.
References
Hayashi-Takagi A, Araki Y, Nakamura M, et al. PAKs inhibitors ameliorate schizophrenia-associated dendritic spine deterioration in vitro and in vivo during late adolescence. Proc Natl Acad Sci U S A. 2014 Apr 29;111(17):6461-6. PMID: 24706880.
Dolan BM, Duron SG, Campbell DA, et al. Rescue of fragile X syndrome phenotypes in Fmr1 KO mice by the small-molecule PAK inhibitor FRAX486. Proc Natl Acad Sci U S A. 2013 Apr 2;110(14):5671-6. PMID: 23509247.