The mTOR pathway as a therapeutic target for precision medicine

Abstract

The mTOR-pathway (mTOR: mammalian target of rapamycin) has been increasingly linked to the pathophysiology of epilepsy in recent years. As a master regulator of cell growth and survival, this signaling pathway has been associated with focal malformations of cortical development (MCD) subtypes often associated with intractable epilepsy. The tuberous sclerosis complex serves as a model disease, in which targeted therapy with mTOR inhibitors alleviate, improve, and might even reverse disease burden. Further, there is evidence of an mTOR effect on established antiepileptic strategies such as ketogenic diet and GABAergic-enhancing medication, which underlies the importance of the mTOR pathway in epileptogenesis.