Scientists use organoid model to identify potential new pancreatic cancer treatment

A drug screening system that models cancers using lab-grown tissues called organoids has helped uncover a promising target for future pancreatic cancer treatments, according to a new study from researchers at Weill Cornell Medicine.

In the study, published Dec. 26 in Cell Stem Cell, the scientists tested more than 6,000 compounds on their pancreatic tumor organoids, which contain a common pancreatic cancer-driving mutation. They identified one compound—an existing heart drug called perhexiline maleate—that powerfully suppresses the organoids’ growth.

The researchers discovered that the cancer-driving mutation in the organoids forces the abnormally high production of cholesterol, which the drug largely reverses.

“Our findings identify hyperactive cholesterol synthesis as a vulnerability that may be targetable in most pancreatic cancers,” said study co-senior author Dr. Todd Evans, vice chair for research in surgery, the Peter I. Pressman MD Professor in Surgery, and a member of the Hartman Institute for Therapeutic Organ Regeneration at Weill Cornell Medicine.