Breakthrough seen for Fabry disease

BIRMINGHAM, Ala., July 25 (UPI) -- Combining two blood pressure-lowering drugs with enzyme therapy is the first treatment to halt kidney deterioration in Fabry disease, say U.S. researchers.

Researchers at the University of Alabama saw improved kidney function in 11 patients with Fabry disease, a rare genetic problem, after they were given an angiotensin-converting enzyme inhibitor, an angiotensin-receptor blocker and enzyme replacement therapy with alpha-galactosidase-A.

Four of the 11 patients had relatively mild kidney disease, while seven had more severe damage, and improvement in kidney function was measured by a reduction of protein in the urine.

The amount of protein in patients' urine decreased significantly in those with severe disease and stabilized in those with milder kidney involvement, the researchers said.

People with Fabry disease do not produce alpha-galactosidase-A, which the body needs to break down fats. ERT alone is not totally effective, and lipids build up in the system causing progressive kidney, brain and heart damage, the researchers said.

The study is published in the advance online version of the September issue of the Journal of the American Society of Nephrology.