Impacting Patients through Increased Options for Treatment

The Office for Clinical and Translational Research (OCTR) provides investigators with clinical trial development, management and regulatory services, research tools, recruitment and retention services, and personnel to support pediatric and adult clinical research studies.

In November 2010, David Neal Franz, MD, Darcy Krueger, MD, PhD, and other collaborators published a groundbreaking report in the New England Journal of Medicine regarding the use of everolimus in patients with tuberous sclerosis complex (TSC) who have subependymal giant cell astrocytomas (SEGAs). This study was instrumental in demonstrating significant tumor volume reduction and decrease in seizure frequency, resulting in the US Food and Drug Administration’s (FDA) accelerated approval of everolimus in this patient population.

More recently, in November of 2015, researchers published the final analysis from a five-year longitudinal study confirming that everolimus continues to demonstrate a sustained effect on SEGA tumor reduction over five or more years of treatment. The analysis also establishes safety and efficacy in the long-term treatment of SEGA associated with TSC. This oral medication provides an alternative therapeutic option to patients who would otherwise require surgery, significantly impacting the lives of TSC patients.

Translational and clinical team members within the OCTR worked alongside Dr. Franz and Dr. Krueger to help move this promising drug through the FDA, and into the hands of TSC patients. Throughout the course of these everolimus studies, translational specialists partnered with Dr. Franz and the TSC research team to provide regulatory oversight, support for FDA and IRB submissions, as well as monitoring services. In addition, OCTR research nurses and study coordinators supported these studies by conducting study visits with patients, and collecting data for analysis.

OCTR experts will continue to support investigators and clinicians in their ongoing efforts to find safe, and effective, treatments for TSC patients, with the goal to improve treatment options and outcomes for patients. These investigators and clinicians at Cincinnati Children’s collaborate to head up the world’s largest TSC clinic, caring for children and adults with TSC.