Vessel rescue
Blood pressure drug might offer new treatment for Marfan syndrome
By Nathan Seppa
The drug losartan may prevent lethal damage to the aorta in people with a genetic disorder called Marfan syndrome, a preliminary study shows. The findings have already paved the way for a large-scale trial of the drug for this condition.
“If this observational information is backed up and confirmed by the randomized clinical trial … it would be a tremendous breakthrough in the management of Marfan’s syndrome,” says Alan Braverman, a cardiologist at the WashingtonUniversity in St. Louis, who wasn’t involved in this study.
People with Marfan syndrome — a genetic condition that can be inherited or, in roughly one-fourth of cases, can appear out of nowhere — tend to have long limbs and fingers, are often tall and may encounter a broad range of symptoms. The worst complication is ballooning of the aorta, the huge artery that carries blood out of the heart. This weakens the vessel over time and poses a risk of lethal rupture that can be prevented only with major surgery. Aorta injury is the most common cause of death from Marfan syndrome.
In the 1990s, cardiologist Harry Dietz of Johns Hopkins University in Baltimore led a team of researchers that discovered that Marfan syndrome stems from a mutation in the FBN1 gene that encodes a glycoprotein called fibrillin-1.In people with the syndrome, a lack of fibrillin-1 allows excess production of a signaling protein called transforming growth factor beta, which kicks on a host of reactions that damage the aorta walls over time, says research physician Benjamin Brooke of Johns Hopkins.