Viagra may treat deadly disease in children

Everyone’s favorite little blue pill may offer more than just a boost in the bedroom.New research suggests the erectile dysfunction drug Viagra could help treat a devastating neurological disorder that primarily strikes young children — most of whom die before the age of three.The study was small, so scientists caution the findings are still preliminary.Still, the results could offer a much-needed spark of hope for families facing the rare genetic disorder with no approved therapies to slow its course.

Part of the challenge is just how rare Leigh syndrome is, affecting only about one in every 40,000 births.“The low case numbers make it difficult to research the disease and present some obstacles in our urgent search for effective therapies,” Dr.Markus Schuelke, lead clinical scientist, said in a press release.  The need for solutions is urgent.

Leigh syndrome is caused by genetic mutations that prevent mitochondria — the cell’s power plants — from producing enough energy.Over time, this starves critical tissues, particularly the brain and muscles, of the fuel they need to function.

The first signs usually appear in infancy or early childhood, though in rare cases the disease can emerge in adolescence or adulthood.Early symptoms can include vomiting, diarrhea and difficulty swallowing.

As the disorder worsens, patients may develop more serious complications such as loss of motor skills, developmental delays, seizures, breathing problems and feeding difficulties — often leading to an early death.That’s where sildenafil, the active ingredient in Viagra, may come in.

In the study, researchers added the drug to lab-grown cells taken from Leigh syndrome patients.They found it switched on genes tied to brain development and reduced some of the harmful molecular changes seen in the disease.Further experiments confirmed the results.

In miniature, three-dimensional models that mimic the human brain, sildenafil boosted nerve cell growth.The drug also ...