Featured Research

from universities, journals, and other organizations

Repurposed drug may be first targeted treatment for serious kidney disease

Date:
November 8, 2013
Source:
Massachusetts General Hospital
Summary:
A team of researchers is reporting that treatment with abatacept appeared to halt the course of focal segmental glomerulosclerosis in five patients, preventing four from losing transplanted kidneys and achieving disease remission in the fifth.

A drug approved for the treatment of rheumatoid arthritis may also turn out to be the first targeted therapy for one of the most common forms of kidney disease, a condition that almost inevitably leads to kidney failure. A team led by Massachusetts General Hospital (MGH) researchers is reporting that treatment with abatacept (Orencia) appeared to halt the course of focal segmental glomerulosclerosis (FSGS) in five patients, preventing four from losing transplanted kidneys and achieving disease remission in the fifth. The report is being issued online in the New England Journal of Medicine to coincide with a presentation at the American Society for Nephrology annual meeting.

"We identified abatacept as the first personalized, targeted treatment for kidney disease and specifically for FSGS, a devastating and largely untreatable disease," says Peter Mundel, MD, of the Division of Nephrology in the MGH Department of Medicine, senior author of the NEJM paper. "We also identified a biomarker that helps us discern which patients are most likely to benefit from therapy with abatacept."

FSGS is characterized by the formation of scar tissue in the glomeruli, the kidney's essential filtering units. Some forms of FSGS are inherited and some have no known cause, but the vast majority of cases develop in individuals with hypertension, obesity or diabetes. Although the underlying mechanism is unclear, FSGS disrupts the function of podocytes, cells within the glomeruli that are crucial to kidney function. While treatment with steroids and some immunosuppressive drugs helps some patients, the drugs' side effects make long-term use problematic.

Previous research by Mundel's team found that the expression on podocytes of an immune molecule called B7-1 signaled the breakdown of the kidney's filtering function, leading to protein leakage into the urine (proteinuria) and ultimate kidney failure. Currently approved for the treatment of rheumatoid arthritis and being studied for other conditions, abatacept inhibits the activity of B7-1, a molecule that is not expressed in healthy podocytes. After in vitro tests indicated that abatacept blocked the primary pathogenic effect of B7-1 expression in podocytes, the team tested treatment with the drug in five FSGS patients -- four with recurrent disease affecting a transplanted kidney and one with treatment-resistant disease who was at high risk for kidney failure.

In all five patients, abatacept treatment induced remission of FSGS-caused proteinuria. Two of those with recurrent disease have remained in remission with a single dose of abatacept for three and four years. The other two required a second dose when proteinuria reappeared a few weeks later and have been in remission for 10 and 12 months respectively. The patient with high-risk, treatment-resistant disease, who is being treated at MGH, went into remission for the first time in more than a year, continues in remission a year later and has resumed a normal lifestyle. While she continues to receive monthly doses of abatacept, she no longer needs the high-dose steroids and immunosuppressive drugs she had been dependent on, some of which actually increase the risk for kidney failure.

An associate professor of Medicine at Harvard Medical School, Mundel explains that, while a large-scale clinical trial is needed, he and his colleagues are hopeful that abatacept will prove an effective treatment for kidney disease characterized by B7-1 expression on podocytes. "We have a decade of good experience with the use of abatacept for rheumatoid arthritis, so we have every reason to believe that it will be an excellent long-term option for the treatment of all B7-1-positive diseases, including FSGS and perhaps diabetic kidney disease."


Story Source:

The above story is based on materials provided by Massachusetts General Hospital. Note: Materials may be edited for content and length.


Cite This Page:

Massachusetts General Hospital. "Repurposed drug may be first targeted treatment for serious kidney disease." ScienceDaily. ScienceDaily, 8 November 2013. <www.sciencedaily.com/releases/2013/11/131108153524.htm>.
Massachusetts General Hospital. (2013, November 8). Repurposed drug may be first targeted treatment for serious kidney disease. ScienceDaily. Retrieved October 1, 2014 from www.sciencedaily.com/releases/2013/11/131108153524.htm
Massachusetts General Hospital. "Repurposed drug may be first targeted treatment for serious kidney disease." ScienceDaily. www.sciencedaily.com/releases/2013/11/131108153524.htm (accessed October 1, 2014).

Share This



More Health & Medicine News

Wednesday, October 1, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Ebola Cases Keep Coming for Monrovia's Island Hospital

Ebola Cases Keep Coming for Monrovia's Island Hospital

AFP (Oct. 1, 2014) A look inside Monrovia's Island Hospital, a key treatment centre in the fight against Ebola in Liberia's capital city. Duration: 00:34 Video provided by AFP
Powered by NewsLook.com
Ebola Puts Stress on Liberian Health Workers

Ebola Puts Stress on Liberian Health Workers

AP (Oct. 1, 2014) The Ebola outbreak is putting stress on first responders in Liberia. Ambulance drivers say they are struggling with chronic shortages of safety equipment and patients who don't want to go to the hospital. (Oct. 1) Video provided by AP
Powered by NewsLook.com
Doctors Reassure Public Ebola Patient Won't Cause Outbreak

Doctors Reassure Public Ebola Patient Won't Cause Outbreak

Newsy (Sep. 30, 2014) After the announcement that the first U.S. patient had been diagnosed with Ebola, doctors were quick to say a U.S. outbreak is highly unlikely. Video provided by Newsy
Powered by NewsLook.com
TX Hospital Confirms Patient Admitted With Ebola

TX Hospital Confirms Patient Admitted With Ebola

AP (Sep. 30, 2014) Medical officials from Texas Health Presbyterian Hospital confirm they are treating a patient with the Ebola virus, the first case found in the US. (Sept. 30 Video provided by AP
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