Dr. Josephine Grima, vice president for research at the National Marfan Foundation, said the drug holds great promise but should not become a mainstream treatment until answers emerge from the larger trial.
"Without a controlled trial, we will not be able to determine how effective it is," she said. In that trial, 604 patients between the ages of 6 months and 25 years will receive either losartin or a beta blocker.
For many years, doctors have tried to forestall ruptures with beta blockers - blood-pressure medications that may relieve pressure on the aorta but do nothing to correct the underlying defect. According to Dietz, it remains unclear whether those drugs lower the risk of aortic complications or prevent the need for surgery.
Losartin belongs to a class of medications called angiotensin receptor blockers or ARBs. Though it's also a blood-pressure medication, losartin works differently than beta blockers and appears to attack the underlying defect of Marfan.
In 1991, Hopkins researchers identified the genetic defect that causes the disease - an abnormality in a protein that normally puts brakes on tissue growth. More recently, Dietz's lab found that the drug had the potential in mice "to prevent aneurysms for a lifetime."
In an editorial accompanying today's article in the New England Journal of Medicine, Dr. Reed E. Pyeritz wrote that "the most appropriate course of action for patients with Marfan's syndrome and their physicians is to give serious consideration to enrolling in the trial." Pyeritz is chief of molecular genetics at the University of Pennsylvania School of Medicine.
Some patients, however, may not be eligible for the trial because they have had previous heart surgery or aortic tears. For those, he said, losartin may be "a reasonable choice."
jonathan.bor@baltsun.com
