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Viagra May be Heart Helper in DMD

The erectile-dysfunction drug sildenafil (Viagra) conferred heart protection and reversal of heart-muscle damage in Duchenne muscular dystrophy research mice.



Viagra, informally referred to as "the little blue pill," has been shown to benefit the hearts of mice with a disease resembling Duchenne muscular dystrophy.

* Results from a new study show that the erectile-dysfunction drug sildenafil imparts cardioprotective effects in mice with both an early- and late-stage DMD-like disease.
* Sildenafil belongs to a class of drugs called phosphodiesterase 5 (PDE5) inhibitors, which relax the smooth muscles lining blood vessels, increasing blood flow to muscles and the heart.
* The new findings, along with the results from several previous studies, suggest that PDE5 inhibitors may be effective treatments for DMD- and BMD-associated cardiomyopathy.
* PDE5 inhibitors already have been FDA-approved for treatment of erectile dysfunction and high blood pressure in humans; this may speed the testing and approval process for their use as a treatment in muscular dystrophy.

The results from these studies suggest that PDE5 inhibitors like sildenafil and tadalafil may be effective treatments for DMD- and BMD-associated cardiomyopathy, both in the early and late stages of the diseases.

More testing will be needed, however, to determine the precise way in which these drugs impart cardioprotective effects.

Although PDE5 inhibitors ultimately will not cure DMD, current studies suggest the possibility that they may be used in combination with other current or future therapies, say the study authors, noting that "targeting skeletal muscle repair without cardiac treatment exacerbates damage to the heart, emphasizing the importance for a cardiac-specific treatment."

Because PDE5 inhibitors already have been FDA-approved for use in humans, the testing and approval process for their use as a treatment in muscular dystrophy potentially may be shorter than for an experimental treatment.