Behind the Science: Behind Potential of Preventing Seizures in TSC Through Sirolimus

Initiated in October 2021, TSC Steps is a phase 1/2 study that is the first of its kind to evaluate a targeted, disease-modifying therapy for preventing or delaying seizure onset in tuberous sclerosis complex (TSC) using a mechanism based approach. Led by Darcy Krueger, MD, PhD, the study use TAVT-18, a novel formulation of sirolimus (Ramapune; Pfizer), an FDA-approved medication for TSC that aims to regulate the target of rapamycin signaling pathway. This discussion talks about how sirolimus had been previously used and the nuances with treatment decisions.