FRIDAY, April 11 (HealthDay News) -- Compounds called CFTR
inhibitors show promise in slowing the progression of polycystic
kidney disease (PKD), according to preliminary research conducted
at the University of California, San Francisco, School of
Medicine.
PKD is the most common kind of genetic kidney disease. Patients
develop cysts on the kidneys, which progressively increase in size
and number. As a result, the kidneys become enlarged, eventually
leading to kidney failure. There is no cure for PKD.
It's believed the accumulation of fluid in the cysts is related
to chloride secretion, which is affected by the CFTR (cystic
fibrosis transmembrane conductance regulator) gene, according to
background information in a news release about the research.
"The CFTR inhibitors could be the basis of a lifelong treatment
to slow renal cyst growth and decline in renal function, prolonging
dialysis-free patient survival," researcher Dr. Alan S. Verkman
said in a prepared statement.
In laboratory tests, he identified a few CFTR inhibitors that
reduced the number and growth of cysts by more than 80 percent. He
then tested these inhibitors in mice genetically engineered to
develop a condition similar to PKD.
Mice treated with these CFTR inhibitors for up to seven days had
significantly slower cyst expansion and kidney enlargement, and
better preservation of kidney function. There was no evidence that
the CFTR inhibitors harmed kidney function.
The findings indicate that CFTR plays a role in the growth of
kidney cysts and suggest that CFTR inhibitors may prove useful in
treating PKD. However, much more research needs to be done, Verkman
said.
"The mouse model of PKD is not the real human disease for many
reasons, such as the more rapid progression of disease in mice.
Clinical trials will be needed to determine the efficacy of these
compounds in human PKD," he said.
The research was expected to be published in the July issue of
the
Journal of the American Society of Nephrology.
More information
The U.S. National Institute of Diabetes and Digestive and Kidney
Diseases has more about
PKD.
