Jan 26, 2010 2:35 PM by Carina Corral
U.C. Santa Barbara researchers have found that the drug Rapamycin is a potential treatment for kidney disease.
According to a press release, more than 600,000 people in the U.S., and 12 million worldwide, are affected by the inherited kidney disease known as ADPKD, short for autosomal-dominant polycystic kidney disease.
In the U.S., the number of individuals affected by ADPKD is greater than the number affected by cystic fibrosis, muscular dystrophy, hemophilia, Down's syndrome, and sickle cell anemia combined.
The disease 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.
Since there is no cure, most patients require kidney transplants or lifelong dialysis.
Rapamycin is currently used to help prevent rejection of a new, transplanted kidney.
"While we had previously shown that 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 Thomas Weimbs, director of the laboratory where the discovery was made. "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."
Currently, there are several clinical trials ongoing internationally to test the safety and efficacy of Rapamycin.
First results from these studies are expected to appear this year.
"But it will be critical to balance any benefits against the expected side effects to judge whether these drugs should be recommended for the treatment of polycystic kidney disease," said Jonathan Shillingford, UCSB project scientist in the Department of Molecular, Cellular and Developmental Biology.
The results are published in the current online issue of the Journal of the American Society of Nephrology.
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