Featured Research

from universities, journals, and other organizations

Experiments Point To New Treatments For PKD

Date:
April 6, 2008
Source:
American Society of Nephrology
Summary:
A family of small molecules called CFTR inhibitors show promising effects in slowing the progression of polycystic kidney disease, the most common genetic disease of the kidneys. Patients with PKD develop cysts on the kidneys, which progressively increase in size and number.

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.

Related Articles


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."

The study entitled, "Small Molecule CFTR Inhibitors Slow Cyst Growth in PKD" April 2, 2008 and in print in the July issue of the Journal of the American Society of Nephrology (JASN). This research was supported through grants from the National Institutes of Health (NIH), Cystic Fibrosis Foundation, and Polycystic Kidney Disease Foundation.


Story Source:

The above story is based on materials provided by American Society of Nephrology. Note: Materials may be edited for content and length.


Cite This Page:

American Society of Nephrology. "Experiments Point To New Treatments For PKD." ScienceDaily. ScienceDaily, 6 April 2008. <www.sciencedaily.com/releases/2008/04/080402120508.htm>.
American Society of Nephrology. (2008, April 6). Experiments Point To New Treatments For PKD. ScienceDaily. Retrieved March 31, 2015 from www.sciencedaily.com/releases/2008/04/080402120508.htm
American Society of Nephrology. "Experiments Point To New Treatments For PKD." ScienceDaily. www.sciencedaily.com/releases/2008/04/080402120508.htm (accessed March 31, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Tuesday, March 31, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Solitair Device Aims to Takes Guesswork out of Sun Safety

Solitair Device Aims to Takes Guesswork out of Sun Safety

Reuters - Innovations Video Online (Mar. 31, 2015) — The Solitair device aims to take the confusion out of how much sunlight we should expose our skin to. Small enough to be worn as a tie or hair clip, it monitors the user&apos;s sun exposure by taking into account their skin pigment, location and schedule. Matthew Stock reports. Video provided by Reuters
Powered by NewsLook.com
Soda, Salt and Sugar: The Next Generation of Taxes

Soda, Salt and Sugar: The Next Generation of Taxes

Washington Post (Mar. 30, 2015) — Denisa Livingston, a health advocate for the Dinι Community Advocacy Alliance, and the Post&apos;s Abby Phillip discuss efforts around the country to make unhealthy food choices hurt your wallet as much as your waistline. Video provided by Washington Post
Powered by NewsLook.com
UnitedHealth Buys Catamaran

UnitedHealth Buys Catamaran

Reuters - Business Video Online (Mar. 30, 2015) — The $12.8 billion merger will combine the U.S.&apos; third and fourth largest pharmacy benefit managers. Analysts say smaller PBMs could also merge. Fred Katayama reports. Video provided by Reuters
Powered by NewsLook.com
S. Leone in New Anti-Ebola Lockdown

S. Leone in New Anti-Ebola Lockdown

AFP (Mar. 28, 2015) — Sierra Leone imposed a three-day nationwide lockdown Friday for the second time in six months in a bid to prevent a resurgence of the deadly Ebola virus. Duration: 01:17 Video provided by AFP
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:

Strange & Offbeat Stories

 

Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins