Small-molecule ligands bind to a distinct pocket in Ras and inhibit SOS-mediated nucleotide exchange activity

T Maurer, LS Garrenton, A Oh, K Pitts… - Proceedings of the …, 2012 - National Acad Sciences
T Maurer, LS Garrenton, A Oh, K Pitts, DJ Anderson, NJ Skelton, BP Fauber, B Pan, S Malek…
Proceedings of the National Academy of Sciences, 2012National Acad Sciences
The Ras gene is frequently mutated in cancer, and mutant Ras drives tumorigenesis.
Although Ras is a central oncogene, small molecules that bind to Ras in a well-defined
manner and exert inhibitory effects have not been uncovered to date. Through an NMR-
based fragment screen, we identified a group of small molecules that all bind to a common
site on Ras. High-resolution cocrystal structures delineated a unique ligand-binding pocket
on the Ras protein that is adjacent to the switch I/II regions and can be expanded upon …
The Ras gene is frequently mutated in cancer, and mutant Ras drives tumorigenesis. Although Ras is a central oncogene, small molecules that bind to Ras in a well-defined manner and exert inhibitory effects have not been uncovered to date. Through an NMR-based fragment screen, we identified a group of small molecules that all bind to a common site on Ras. High-resolution cocrystal structures delineated a unique ligand-binding pocket on the Ras protein that is adjacent to the switch I/II regions and can be expanded upon compound binding. Structure analysis predicts that compound-binding interferes with the Ras/SOS interactions. Indeed, selected compounds inhibit SOS-mediated nucleotide exchange and prevent Ras activation by blocking the formation of intermediates of the exchange reaction. The discovery of a small-molecule binding pocket on Ras with functional significance provides a new direction in the search of therapeutically effective inhibitors of the Ras oncoprotein.
National Acad Sciences