Combination of Everolimus with Sorafenib for Solid Renal Tumors in Tsc2^+/- Mice Is Superior to Everolimus Alone.

ABSTRACT: Tuberous sclerosis (TSC) is an inherited tumor syndrome caused by mutations in TSC1 or TSC2 that lead to aberrant activation of mTOR and development of tumors in multiple organs including the kidneys. The mTOR inhibitors rapamycin and everolimus (rapalogs) have demonstrated clinical efficacy in treating TSC-associated tumors including renal angiomyolipomas. However, tumor responses are usually only partial, and regrowth occurs after drug withdrawal. TSC-associated tumors are highly vascular, and TSC patients with renal angiomyolipomas have elevated levels of circulating vascular endothelial growth factor (VEGF) A and VEGFD. Sorafenib inhibits multiple kinases including VEGF receptors and has been used to treat metastatic epithelioid angiomyolipoma in one case, but formal trials have not be

SUBMITTER: Yang J 

PROVIDER: S-EPMC5238457 | biostudies-literature | 2017 Feb

REPOSITORIES: biostudies-literature