Cincinnati Children's Hospital Medical Research Scores Big Win: New Drug Quickly Approved, Shrinks Kids' Brain Tumors



Children's Hospital



CINCINNATI (TDB) -- Swiss pharmaceutical giant Novartis announced today in Europe the FDA granted accelerated approval for a new drug that shrinks brain tumors otherwise requiring surgery.  The drug was tested on 28 patients at Cincinnati Children's Hospital Medical Center and nearly a third had a reduction in the size of their tumors.  Doctors say the drug should fill an unmet medical need.  All of the patients were being treated for subpendymal giant cell astrocytoma, known to physicians as SEGA, an inherited disease related to a genetic disorder called tuberous sclerosis.  Tuberous sclerosis can cause severe mental retardation and seizures.  Novartis credited the breakthrough to Cincinnati Children's Hospital in an announcement from Basel today. Early approval came because the results in Cincinnati were so dramatic. Still, Novartis said more studies are taking place:

 
"The accelerated approval of Afinator [generic name everolimus} is based on an open-label, single-arm 28-patient study conducted by Cincinnati Children's Hospital Medical Center. The effectiveness of Afinator is based on an analysis of change in SEGA volume. A Phase III study is under way that compares Afinator to placebo to explore the clinical benefits of Affinator for patients with treatment associated with TS. 

"Prior to this FDA approval, the only treatment option for growing SEGAs, which primarily affect children and adolescents, was brain surgery. Tuberous sclerosis is a genetic disorder affecting approximately 25,000 to 40,000 people in the U.S. that may cause tumors to form in vital organs. SEGAs, benign brain tumors, occur in up to 20% of patients with TS."

Cincinnati Children's opened its clinic for tuberous sclerosis in the early 1990s and it now has a team of researchers battling the genetic disorder. Dr. David Neal Franz is the clinic director and you can learn more about him by clicking here.  Franz did not start out to become a physician.  He went to Earlham College in Richmond, Ind., and majored in history and literature.  That small liberal arts school set Dr. Franz on the path, though, that should help make life a little bit easier for many people on this planet.