Our paper is highly cited as it is the first study to demonstrate a potential therapeutic role for rapamycin (and mTOR inhibition) in polycystic kidney disease (PKD). Polycystic means multiple cysts. These cysts grow and multiply over time, also causing the mass of the kidney to increase. Ultimately, the diseased kidney shuts down, causing end-stage renal disease for which dialysis and transplantation are the only forms of treatment.

PKD is the commonest hereditary life-threatening disease in the USA. It is more common than cystic fibrosis, muscular dystrophy, hemophilia, Down syndrome, and sickle cell anemia—combined.

It probably involves more than 600,000 Americans and an estimated 12.5 million people worldwide—regardless of sex, age, race, or ethnic origin. As it is such a common disease and may ultimately lead to end-stage kidney disease and the need for dialysis and kidney transplantation, there is a large movement to find a cure for PKD. Rapamycin is a relatively safe drug and is already FDA-approved for use in transplantation immunosuppression and certain cancers.