Characterizing patterns of mTORC1 activation across sarcomas using single-sample gene set enrichment analysis ssGSEA and a national biomarker database


Daniel S. Lefler, Andrew Elliott, Erik Blomain, Robert G. Maki, Jaime Modiano, Andreas Seeber, Richard Riedel, Aaron Sarver, Andrea Espejo-Freire, Moh’d Khushman, Sosipatros Boikos, Galina Lagos, Vaia Florou, Margaret von Mehren, Matthew Oberley, Gina D’Amato, Jonathan Trent, Atrayee Basu Mallick


  • The mTOR pathway is a central signaling circuit in many tumors and contributes to cell growthThe phase III SUCCEED trial (2012) showed statistically significant—but clinically minimal—difference in PFS with the mTOR inhibitor (mTORi) ridaforolimus after chemo response, but didn’t stratify by molecular findings
  • The AMPECT trial (2021) showed activity of nab-sirolimus in PEComa, especially in TSC2-mut tumors
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