S6K1 inhibitors for fragile X syndrome and triple negative breast cancer

21st RSC/SCI Med Chem Symposium, 2021.

David Walker

ABSTRACT: S6K1 is implicated in the etiology of fragile X syndrome (FXS), a genetic neurodevelopmental disorder, and in triple negative breast cancer (TNBC), a highly aggressive cancer. We set out to determine if small molecule S6K1 inhibitors with the ability to reach the brain could treat either of these diseases. We identified the lead compound FS115, a proprietary oral brain penetrant S6K1 inhibitor, which showed robust efficacy in models of FXS and TNBC.