Everolimus shrinks brain tumors in children with tuberous sclerosis complex

14 November 2012

Everolimus, a drug originally developed to prevent the rejection of transplanted organs and is also marketed for the treatment of various cancers, 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 on-line in The Lancet, is the latest to show the effectiveness of everolimus in slowing the cell growth that is overactive in patients with TSC. Everolimus is marketed under the brand names Afinitor, Certican and Votubia by Swiss drug major Novartis (NOVN: VX), which provided drug and financial support for the study.

"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 David Franz, co-director of the TSC Clinic at Cincinnati Children's and the study's main author, adding: "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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