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A drug typically used to treat patients with high blood pressure has been shown to be effective in treating Marfan syndrome in children.
Researchers at the John Hopkins University in Baltimore, US, found that losartan worked to slow the enlargement of the aorta, which is the most life-threatening defect associated with Marfan syndrome.
Marfan syndrome is a genetic disorder characterised by disproportionately long limbs, long, thin fingers, a tall stature and a predisposition to heart abnormalities – particularly in the heart valves and aorta.
In mice engineered to contain the same genetic defect that causes Marfan syndrome, scientists previously discovered that most features of the syndrome arise from excessive activity of the protein TGF-beta, which is vital to cell growth. Treating the mice with losartan slowed, and in some cases stopped, potentially lethal enlargement of the aorta.
The researchers then carried out a study of a small group of children with severe Marfan syndrome and found that losarten reduced aortic enlargement quite dramatically. The next step is a large, multicentre clinical trial of the drug.
Reporting in the New England Journal of Medicine, lead researcher Professor Harry Dietz said: “This would be the first therapy generated by basic research that revealed the molecular mechanism of this genetic disease.”
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New England Journal of Medicine
