The drug, also called sirolimus, is currently used as an immunosuppressant to help prevent rejection of a transplanted kidney. The new research shows studies previously performed on mice are more likely to translate to humans than has been thought.
More than 12 million worldwide are affected by the inherited kidney disease known as ADPKD, short for autosomal-dominant polycystic kidney disease. The disease, for which there is no treatment, is characterized by the proliferation of cysts that eventually debilitate the kidney, causing kidney failure in half of all patients by the time they reach age 50.
"While we had previously shown rapamycin is highly effective in mouse models of polycystic kidney disease, the problem had been that these mice had different genes affected than human patients," said Associate Professor Thomas Weimbsm who led the latest research. "Therefore, the question always remained whether rapamycin would be effective in patients, too.
"Our new study now is the first to show that rapamycin is also highly effective in a new mouse model in which the same gene is affected as in most human patients."
The study that included researchers from Johns Hopkins University is detailed in the online issue of The Journal of the American Society of Nephrology.
Copyright 2010 United Press International
Post new comment