Uncovering New, Synergistic Ways to Treat Tuberous Sclerosis Complex
Previous data has shown that early seizure control in children can improve learning as compared to those with poorer control. Some antiseizure medications are effective in individuals with TSC; although, there remains a portion of the population that remains refractory to the medications available. An ongoing phase 1/2 trial, TSC Steps (NCT04595513), is assessing whether treatment with TAV-18, a novel formulation of sirolimus, a mechanistic target of rapamycin (mTOR) inhibitor, can prevent or delay seizure onset in patients with TSC.
If successful, this study could have significant impacts on the way patients with TSC are treated, and the timing in which they receive treatment. Recently, NeurologyLive® sat down with trial investigator Darcy Krueger, MD, PhD, to discuss exploring different types of approaches to TSC, including combining therapies early in a patient’s diagnosis. Krueger, director of the Tuberous Sclerosis Clinic at Cincinnati Children’s, detailed the challenges with expanding ways to treat the disease, as well as provided commentary on the significance of TSC Steps and the thoughts of preventing seizures in TSC.
(https://www.neurologylive.com/....view/uncovering-new-
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