Scientists in the laboratory of Dr. Thomas Weimbs have demonstrated that a new drug is effective in treating a very common genetic disease, polycystic kidney disease (PKD). Over 600,000 people in the U.S., and 12 million worldwide, are affected by PKD, a disease that is characterized by the proliferation of thousands of cysts that eventually debilitate the kidneys, causing kidney failure in half of all patients by the time they reach age 50. The is currently no available treatment for this disease. In collaboration with the Indiana-based biopharmaceutical company Endocyte, Dr. Weimbs’ laboratory developed and tested a new compound called folate-conjugated rapamycin that targets to the kidneys and slows renal cyst growth in a PKD mouse model. Dr. Weimbs is also interested in stem cell-based treatments for kidney disease.