Freedman Lab Tests a Promising mTOR Inhibitor in PKD Organoids

Freedman Lab Tests a Promising mTOR Inhibitor in PKD Organoids

Polycystic kidney disease (PKD) is an inherited, and currently incurable, disorder that affects more than 600,000 people in the United States. The disease causes fluid-filled cysts which can impair kidney function and lead to kidney failure, an outcome requiring dialysis or transplant.

However, even patients who are fortunate enough to receive a new kidney may be vulnerable to other health challenges associated with PKD. Harmful cysts can grow on the liver and pancreas and the disease can contribute to high blood pressure and vascular complications.

Because PKD is a whole-body disease, scientists are looking for whole-body treatments. One known target is the mTOR pathway, which regulates many aspects of cell biology and controls important processes related to metabolism and immunity. Overactivity of mTOR can lead to disorders of cell proliferation, like cancer.

“It’s really a growth pathway,” says Dr. Benjamin Freedman, an associate professor of Medicine/Nephrology and a member of the Institute for Stem Cell and Regenerative Medicine (ISCRM). “When you see mTOR activity in a cell, it means the cells are growing and dividing. That’s why it’s a target for cancer treatment. And that’s why we think it could be a target for PKD treatment.”

Dr. Freedman is the senior author of the paper. The first author is Ramila Gulieva, a Research Scientist in the Freedman Lab.