Featured Research

from universities, journals, and other organizations

Drug Treatment For Marfan Syndrome Looks Promising

Date:
June 27, 2008
Source:
Johns Hopkins Medical Institutions
Summary:
A small study in 18 patients assessing the effectiveness of the drug losartan for treating Marfan syndrome in children has yielded encouraging results. Reporting in the New England Journal of Medicine, researchers showed that losartan -- a compound used for years to treat high blood pressure -- slowed the enlargement of the aorta, the most life-threatening defect associated with Marfan syndrome.

A small study in 18 pattients assessing the effectiveness of the drug losartan for treating Marfan syndrome in children has yielded encouraging results. Reporting in the June 26 issue of The New England Journal of Medicine, Johns Hopkins researchers showed that losartan-a compound used for years to treat high blood pressure-slowed the enlargement of the aorta, the most life-threatening defect associated with Marfan syndrome.

"This experience increases my belief that losartan holds great promise for treating Marfan syndrome," says Harry Dietz, M.D., a professor in the McKusick-Nathans Institute of Genetic Medicine and director of the William S. Smilow Center for Marfan Syndrome Research at Hopkins. "This would be the first therapy generated by basic research that revealed the molecular mechanism of this genetic disease."

In mice engineered to contain the same genetic defect that causes Marfan syndrome, Dietz's team previously discovered that most features of the syndrome arise from excessive activity of the protein TGF-beta, a protein vital to cell growth and specialization.

Treating the mice with losartan, a drug also known to decrease TGF-beta activity, slowed, and in some cases stopped, potentially lethal enlargement of the aorta, the body's largest. Such enlargement is a key feature of Marfan syndrome.

On the basis of these findings, the Pediatric Heart Network of the National Heart, Lung and Blood Institute at the National Institutes of Health has approved and launched a large, multicenter clinical trial of losartan for Marfan syndrome, which Dietz oversees with Ronald Lacro, M.D., director of the cardiovascular genetics clinic at Children's Hospital Boston.

Before the start of that clinical trial, physicians at Johns Hopkins felt compelled to first try losartan in a small group of children, with severe Marfan syndrome, whose aortas, which carry oxygenated blood from the hear to the resto of the body, were enlarging rapidly and unresponsive to other treatment.

"These patients had severe forms of the disease and had shown progressive aortic enlargement despite treatment with existing therapies, including beta-blockers and ACE inhibitors," says Benjamin Brooke, M.D., a research fellow in genetic medicine and surgery at Hopkins.

For the 18 patients in the small, preliminary study, the average rate of aortic enlargement before starting losartan was 3.5 millimeters in diameter per year, and after losartan treatment, just shy of a half millimeter per year. "While the response to therapy varied somewhat, it was exciting to see such a dramatic change in the majority of these patients," Brooke says.

"I am very encouraged by this initial experience, but it cannot substitute for a properly controlled clinical trial," says Dietz. "I encourage patients and families with Marfan syndrome to talk to their doctors and get involved with the current clinical trial."

The research was funded by the National Institutes of Health, Howard Hughes Medical Institute, William S. Smilow Center for Marfan Syndrome Research, Dana and Albert "Cubby" Broccoli Center for Aortic Diseases, and the National Marfan Foundation.

Authors on the paper are Brooke, Dietz, Jennifer Habashi, Daniel Judge, Bart Loeys and Nishant Patel, all of Hopkins.


Story Source:

The above story is based on materials provided by Johns Hopkins Medical Institutions. Note: Materials may be edited for content and length.


Cite This Page:

Johns Hopkins Medical Institutions. "Drug Treatment For Marfan Syndrome Looks Promising." ScienceDaily. ScienceDaily, 27 June 2008. <www.sciencedaily.com/releases/2008/06/080625193909.htm>.
Johns Hopkins Medical Institutions. (2008, June 27). Drug Treatment For Marfan Syndrome Looks Promising. ScienceDaily. Retrieved September 30, 2014 from www.sciencedaily.com/releases/2008/06/080625193909.htm
Johns Hopkins Medical Institutions. "Drug Treatment For Marfan Syndrome Looks Promising." ScienceDaily. www.sciencedaily.com/releases/2008/06/080625193909.htm (accessed September 30, 2014).

Share This



More Health & Medicine News

Tuesday, September 30, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

How 'Yes Means Yes' Defines Sexual Assault

How 'Yes Means Yes' Defines Sexual Assault

Newsy (Sep. 29, 2014) Aimed at reducing sexual assaults on college campuses, California has adopted a new law changing the standard of consent for sexual activity. Video provided by Newsy
Powered by NewsLook.com
Scientists May Have Found An Early Sign Of Pancreatic Cancer

Scientists May Have Found An Early Sign Of Pancreatic Cancer

Newsy (Sep. 29, 2014) Researchers looked at 1,500 blood samples and determined people who developed pancreatic cancer had more branched chain amino acids. Video provided by Newsy
Powered by NewsLook.com
Colo. Doctors See Cluster of Enterovirus Cases

Colo. Doctors See Cluster of Enterovirus Cases

AP (Sep. 29, 2014) Doctors at the Children's Hospital of Colorado say they have treated over 4,000 children with serious respiratory illnesses since August. Nine of the patients have shown distinct neurological symptoms, including limb weakness. (Sept. 29) Video provided by AP
Powered by NewsLook.com
Dr.'s Unsure of Cause of Fast-Spreading Virus

Dr.'s Unsure of Cause of Fast-Spreading Virus

AP (Sep. 29, 2014) Doctors at the Children's Hospital of Colorado say they have treated over 4,000 children with serious respiratory illnesses since August. Nine of the patients have shown distinct neurological symptoms, including limb weakness. (Sept. 29) 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