As a result, efforts are already under way with the National Heart, Lung and Blood Institute and its affiliated network of hospitals in the Pediatric Heart Health Network and The Johns Hopkins Hospital to begin a clinical trial for people with Marfan syndrome, which is expected to start in the fall.
"The results of our study in mice greatly increase the likelihood that losartan will also serve as an effective treatment in humans and quickly, because it is already approved for use in the United States as a safe and effective treatment for hypertension," says study senior author, Harry (Hal) Dietz, M.D., a professor at the McKusick-Nathans Institute of Genetic Medicine at The Johns Hopkins University School of Medicine.
The Hopkins findings, to be published in the journal Science online April 6, are considered a breakthrough discovery, researchers say, because they are the first to identify a drug that can prevent Marfan syndrome's most life-threatening complications from developing and potentially reverse the damage already done. Marfan syndrome can lead to a fatal tear or rupture in the aorta, the body's main blood vessel that carries blood away from the heart. The disease is often diagnosed in childhood or early adulthood, when people are still young enough to consider long-term therapies.
"It is very exciting that an existing medication has proven capable of not only treating the problems of Marfan syndrome, but also disrupting the biological pathway that precipitated them," says cardiac geneticist Daniel P. Judge, M.D., an assistant professor at Hopkins and its Heart Institute and co-lead author of the study.
"Until now, surgery has been our main option for repairing an aorta at risk of rupturing," says cardiac surgeon Vincent Gott,
Contact: David March
Johns Hopkins Medical Institutions