Transforming TSC Epilepsy with a Precision Therapy

Tuberous sclerosis complex is a rare genetic condition caused by changes in the TSC1 or TSC2 gene that over-activate a key growth-control pathway, known as mTOR. This leads to seizures that often don’t respond to existing medicines and to noncancerous tumors in organs such as the brain, kidneys, lungs, and skin. In fact, more than 65 percent of people with TSC-related epilepsy still have seizures despite available treatments. Though mTOR inhibitors can help some patients, their benefits are limited because of side effects due to their lack of specificity. Aeovian Pharmaceuticals is developing an experimental therapy that precisely targets the overactive part of this pathway and avoids the part thought to cause many side effects. We spoke to Allison Hulme, CEO of Aeovian Pharmaceuticals, about tuberous sclerosis complex, the problems with existing therapies for seizures related to the condition, and the opportunity for its next-generation, selective mTOR inhibitors to treat tuberous sclerosis complex and beyond.