Experiments point to new treatments for PKD

Apr 02, 2008

A family of small molecules called CFTR inhibitors show promising effects in slowing the progression of polycystic kidney disease (PKD), the most common genetic disease of the kidneys, according to preliminary research reported in the July 2008 issue of the Journal of the American Society Nephrology.

Although much more study is needed, CFTR inhibitors could provide a useful new approach for the treatment of PKD. "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," comments Dr. Alan S. Verkman of University of California, San Francisco, School of Medicine.

Patients with PKD develop cysts on the kidneys, which progressively increase in size and number. The kidneys become enlarged, eventually leading to kidney failure. Previous research has suggested that the buildup of fluid in the cysts is related to chloride secretion, which is affected by the CFTR (cystic fibrosis transmembrane conductance regulator) gene. The researchers used automated "high-throughput" screening techniques to identify CFTR inhibitors that might affect cyst growth.

These screening studies identified two classes of small-molecule CFTR inhibitors that slowed the growth of renal cysts. The best inhibitor of each class was identified and shown to reduce the number and growth of cysts by more than 80 percent.

The inhibitors were then tested in mice that had been genetically altered to produce a condition similar to PKD. Animals treated with 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 of harmful effects on kidney function.

PKD is an incurable condition for which new treatments are urgently needed. If effective medications to reduce the rate of fluid buildup in cysts could be developed, they might provide an entirely new approach to treatment to slowing the progression of the disease.

The results show that CFTR plays a role in the growth of renal cysts, and suggest that CFTR inhibitors have potential as treatments to reduce cyst growth in PKD. However, much more research will be needed to see if drugs based on the CFTR blockers will be useful in human PKD.

"The mouse model of PKD is not the real human disease for many reasons, such as the more rapid progression of disease in mice," says Dr. Verkman. "Clinical trials will be needed to determine the efficacy of these compounds in human PKD."

Source: American Society of Nephrology

Explore further: The human parasite Leishmania is a probiotic for the fly that carries it

add to favorites email to friend print save as pdf

Related Stories

Kingston, Jamaica hybrid project to harness sun and wind

8 hours ago

A hybrid energy project in Kingston, Jamaica, aims to satisfy the need for money-saving renewable energy. U.S.-based WindStream Technologies recently announced the wind solar hybrid installation commissioned ...

Archaeologists excavate NY Colonial battleground

9 hours ago

Archaeologists are excavating an 18th-century battleground in upstate New York that was the site of a desperate stand by Colonial American troops, the flashpoint of an infamous massacre and the location of the era's largest ...

Google eyes Chrome on Windows laptop battery drain

16 hours ago

Google Chrome on Microsoft Windows has been said to have a problem for some time but this week comes news that Google will give it the attention others think the problem quite deserves. Namely, Google is to ...

Security contest techies say they hacked Tesla Model S

18 hours ago

The good news: Tomorrow's cars are computers on wheels. The bad news: Tomorrow's cars are computers on wheels. Ma Jie, writing in Bloomberg News, reported this week that the Tesla Model S sedan was the target ...

Water problems lead to riots, deaths in South Africa

20 hours ago

Three babies who died from drinking tap water contaminated by sewage have become a tragic symbol of South Africa's struggle to cope with a flood of people into cities designed under apartheid to cater to ...

Recommended for you

Meningitis diagnosis prompted W.Va. clinic probe

22 hours ago

A health official says an investigation that found syringes were being reused at a West Virginia pain management clinic was triggered by patient who developed bacterial meningitis.

California firm issues nationwide fruit recall

Jul 22, 2014

A Central California company has issued a voluntary nationwide recall of specific lots of its fresh peaches, plums, nectarines and pluots over concerns of possible listeria contamination.

User comments : 0