Drug therapy for PKU reverses he...(ctoral cardiology research fellow at Hop...),Health

Before and after treatment, heart function was monitored by several key tests, such as echocardiogram and magnetic resonance imaging, as well as catheters placed within the heart. This was supplemented by post-treatment tissue analysis. Results from this group were compared to another group of 25 mice, also subjected to high blood pressure, who were given placebo instead of BH4 during the same timeframe.

After induced heart failure and five weeks of subsequent therapy, researchers found that BH4-treated mice showed remarkable improvements, according to Kass, when compared to placebo-treated animals. Ejection fraction measures of heart pumping function not only stabilized with BH4, but improved, from an average 87 percent before heart failure to roughly 48 percent at the start of therapy, then back to 59 percent at the end of study. Meanwhile, average pumping function in placebo-treated mice showed a perilous decline, from 87 percent to 48 percent, to 35 percent.

Heart weight, as measured by muscle mass, showed similar results. Pressure stress resulted in mice hearts growing from an average 100 milligrams to 290 milligrams before therapy, returning to an average 209 milligrams in the BH4-treated hearts, while placebo-treated hearts grew increasingly worse, to an average 330 milligrams.

Improvements with BH4 therapy were almost as dramatic in at least three other measurements of organ health, including heart wall thickness, muscle cell size and fibrosis, and lowered chemical production of dangerous free radicals.

Hearts clearly got better from administering the drug, and our results offer proof of principle that damage to the left ventricle from hypertrophy can be stopped
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