Tuberous Sclerosis Research

The Tuberous Sclerosis Clinic at Cincinnati Children’s is active in many clinical, translational, and basic science research studies to improve care for children and adults with tuberous sclerosis complex (TSC) at any given time.

Led by Darcy Krueger, MD, PhD, our research team includes three research coordinators, regulatory staff, and faculty from across the institution as well as the University of Cincinnati. This diverse group allows us to investigate all TSC-related issues and push us to the goal of developing patient-specific treatments.

Together, we've led the way in understanding the safety and effectiveness of mTOR inhibitors and other agents to treat subependymal giant cell astrocytoma (SEGA) tumors in the brain, angiomyolipoma (AMLs) in the kidneys, lymphangioleiomyomatosis (LAM) of the lungs and epilepsy. We continue to investigate the use of these agents for the treatment of other manifestations of TSC and their role in preventing TSC symptoms.

We are also involved in several human clinical trials evaluating new medications and therapies to treat multiple aspects of TSC, including epilepsy and behavior. Additional natural history and translational research studies seek to understand poorly understood manifestations of TSC that occur throughout the lifespan of individuals with this diagnosis. 

If you're interested in participating in a research study, contact TSCResearch@cchmc.org. You do not need to be a patient to participate in research studies.