Compound 7 was designed by scientists at Novartis, as a potent and selective brain-penetrant inhibitor of mechanistic target of rapamycin (mTOR) [1
]. It is an ATP-competitive inhibitor. It is already established that inhibition of mTOR in the CNS providees clinical efficacy in certain seizure disorders (such as tuberous sclerosis complex; TSC [2
]) that are associated with overactivated mTOR signalling [3-4
]. The allosteric mTOR inhibitor everolimus
is already approved as adjunctive treatment for patients with TSC-associated partial-onset seizures. Compound 7 was optimised to provide a molecule with improved brain permeability compared to everolimus.