April 26, 2012 -- The Tuberous Sclerosis Alliance (TS Alliance) today announced the U.S. Food and Drug Administration (FDA) has approved a drug to treat growing kidney tumors called angiomyolipomas (AMLs). AMLs are common in individuals with a rare genetic disease called tuberous sclerosis complex (TSC). This drug, manufactured by Novartis Oncology, is called Afinitor and helps shrink the size of AMLs, which occur in 70 to 80 percent of adults and older children with TSC. Afinitor was previously approved by the FDA to treat subependymal giant cell astrocytomas, a type of brain tumor, in people with TSC.
“Renal angiomyolipomas can be challenging for individuals with TSC, which is why we are thrilled to see a new treatment option for these individuals,” said Steven L. Roberds, PhD, TS Alliance Chief Scientific Officer. “Prior to Afinitor, surgery or embolization were the only treatment options for individuals with growing AMLs.”
“At the TS Alliance, it’s crucial for us to raise awareness about the tuberous sclerosis complex, as many individuals go undiagnosed or even misdiagnosed,” explained Kari Luther Rosbeck, President and Chief Executive Officer for the TS Alliance. “Therefore, it’s important that anyone with TSC get the care needed by having multiple treatment options available.”
“TSC research moved very rapidly from the identification of the genes for TSC in the 1990s to clinical trials in recent years. The continued development of new treatment options for all of the symptoms of TSC is our highest research priority,” Dr. Roberds continued. “Today’s FDA approval of Afinitor to treat growing AMLs is a major step forward in our continued search for new treatments to improve the lives of people with TSC.”
“Because identifying new treatments is so important, the TS Alliance instituted a new TSC Drug Screening Program in 2010, which will help drive and accelerate basic and translational research,” Rosbeck said. According to Rosbeck, the program provides funding to test drugs that are already FDA-approved for other indications, or new compounds, in order to fast track the translation of basic research to new treatments for individuals with TSC.
“By bringing together biotech and pharmaceutical companies with researchers interested in TSC, our hope is that we can accelerate the testing of additional new treatments for TSC,” added Dr. Roberds.
For more information, you can read the FDA press release and the Novartis press release.
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