Abstract 2288: Targeting mTOR pathway in rare and recalcitrant tumors

Abstract

Abstract INTRODUCTION: Genomic alterations affecting the PI3K/AKT/mTOR pathway are commonly seen among various cancer types [Kandoth 2013]. The objective of this study is to determine the clinical utility of using comprehensive genomic profiling (CGP) in the course of clinical care to identify genomic alterations affecting the PI3K/AKT/mTOR pathway in patients with either rare or refractory cancers. METHODS: CGP was performed by Foundation Medicine, Inc. Genomic alterations were reviewed by members of an institutional molecular tumor board (MTB). Consensus recommendations on genomically targeted, clinically relevant FDA-approved, on- and off-label therapies and clinical trials were sent to the treating physician for their consideration as treatment alternatives. Ninety-seven tumors with alterations in the PI3K/AKT/mTOR pathway were analyzed. RESULTS: Thirty-three patients with cancers that were either rare or refractory to standard therapy received drug targeting the PI3K/AKT/mTOR pathway. Age range was 18-78 years old. Thirteen patients were non-Hispanic White, three non-Hispanic Black, two Hispanic and one Asian. Tumors tested included 6 gynecologic, 5 renal, 2 sarcomas and one each of pancreatic, melanoma, T cell lymphoma, bladder, adrenal, and skin-not melanoma. Treatment outcomes were available for 19/33 patients (one patient died soon after presentation at the MTB and could not be treated). Seven (37%) patients derived clinical benefit from a drug targeting the PI3K/AKT/mTOR pathway; the duration of clinical benefit ranged from 90-245 days. The clinical benefit was not tumor type specific and included two endometrial tumors, 2 renal tumors, one sarcoma, one adrenal paraganglioma and one micropapillary urothelial carcinoma. Eight patients have not been started on PI3K/AKT/mTOR targeted therapy yet. CONCLUSION: These data suggest that tumors with alterations in the PI3K/AKT/mTOR pathway may respond to targeted therapy regardless of tumor of origin. Citation Format: Veronica Rojas, Shridar Ganesan, Kim M. Hirshfield, Robert S. Dipaola, Lorna Rodriguez-Rodriguez. Targeting mTOR pathway in rare and recalcitrant tumors. [abstract]. In: Proceedings of the 107th Annual Meeting of the American Association for Cancer Research; 2016 Apr 16-20; New Orleans, LA. Philadelphia (PA): AACR; Cancer Res 2016;76(14 Suppl):Abstract nr 2288.