Drug shrinks brain tumors in children with tuberous sclerosis
complex 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, co-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." The phase III study was conducted
among 117 patients with TSC who were randomly assigned to either
everolimu