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Drug Shrinks Brain Tumors in Children with Tuberous Sclerosis Complex

Posted Nov 15 2012 5:48pm

A drug originally developed to prevent the rejection of transplanted organs has now been shown to dramatically reduce a particular kind of brain tumor in patients with tuberous sclerosis complex (TSC) — a genetic disease that causes tumors to grow on vital organs.

The study, published online in The Lancet, is the latest to show the effectiveness of everolimus in slowing the cell growth that is overactive in patients with TSC.

“Every patient in this study experienced a decrease in size of their tumors, and no patient required surgery for their tumors after treatment with everolimus,” says Dr. Franz, director of the TSC Clinic at Cincinnati Children’s and the study’s main author. “Thirty-five percent of patients in this study on everolimus had at least a 50 percent reduction in tumor volume after an average of 42 weeks on medication.”


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