Hypertension Drug Treats MD in Mice

Iosartan (Cozaar), which lowers blood pressure, has reversed muscle wasting in mice with Duchenne muscular dystrophy, say U.S. researchers.

The drug also shows promise against Marfan syndrome, said a team from Johns Hopkins in Baltimore, Md.

The researchers, led by Ronald Cohn and Hal Dietz, were originally looking for ways to treat Marfan syndrome, a genetic condition in which weak connective tissue causes organs and blood vessels to collapse from lack of structure.

Children and adults with severe Marfan syndrome often have very small, weak muscles with excess levels of TGF-beta protein, which prevents muscle repair. The team decided to see whether Cozaar could restore normal muscle structure and function in Marfan syndrome because the drug blocks TGF-beta activity and is safe for all age groups.

They gave Cozaar genetically engineered mice with Marfan syndrome, and after six months of treatment the drug had completely restored muscle architecture and vastly improved their strength. A clinical trial of Cozaar in human beings with Marfan will begin within weeks.

The team wanted to see if Cozaar had the same effect on mice with Duchenne muscular dystrophy, the most common form of incurable muscular dystrophy in children, that also results from excess TGF-beta activity. After nine months of treatment, the mice showed completely restored ability to regenerate muscle after injury and their muscle strength matched that of normal mice.

We may have a real treatment alternative for … Duchenne muscular dystrophy that improves both length and quality of life, said Cohn.

The study appears in this week’s online issue of Nature Medicine.