Selective targeting of mutant adenomatous polyposis coli (APC) in colorectal cancer

L Zhang, PC Theodoropoulos, U Eskiocak… - Science translational …, 2016 - science.org
L Zhang, PC Theodoropoulos, U Eskiocak, W Wang, YA Moon, B Posner, NS Williams
Science translational medicine, 2016science.org
Mutations in the adenomatous polyposis coli (APC) gene are common in colorectal cancer
(CRC), and more than 90% of those mutations generate stable truncated gene products. We
describe a chemical screen using normal human colonic epithelial cells (HCECs) and a
series of oncogenically progressed HCECs containing a truncated APC protein. With this
screen, we identified a small molecule, TASIN-1 (truncated APC selective inhibitor–1), that
specifically kills cells with APC truncations but spares normal and cancer cells with wild-type …
Mutations in the adenomatous polyposis coli (APC) gene are common in colorectal cancer (CRC), and more than 90% of those mutations generate stable truncated gene products. We describe a chemical screen using normal human colonic epithelial cells (HCECs) and a series of oncogenically progressed HCECs containing a truncated APC protein. With this screen, we identified a small molecule, TASIN-1 (truncated APC selective inhibitor–1), that specifically kills cells with APC truncations but spares normal and cancer cells with wild-type APC. TASIN-1 exerts its cytotoxic effects through inhibition of cholesterol biosynthesis. In vivo administration of TASIN-1 inhibits tumor growth of CRC cells with truncated APC but not APC wild-type CRC cells in xenograft models and in a genetically engineered CRC mouse model with minimal toxicity. TASIN-1 represents a potential therapeutic strategy for prevention and intervention in CRC with mutant APC.
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