Viagra may very well be used to deal with ‘lethal illness’ in kids throughout main breakthrough
A sex drug could soon be used in children’s medicine to treat a childhood disease.
Not just for giving a boost in the bedroom, science boffs believe they have found a new use for the drug which could help youngsters diagnosed with a devastating neurological disorder that primarily strikes young children — and can kill before the age of three.
Preliminary studies are in the “early stages” but clever beans say their research has shown the erection pill could offer a lifeline for families diagnosed with the rare genetic disorder which at present, has no approved therapies to slow its course.
They believe it could help treat Leigh syndrome, which affects only about one in every 40,000 births.
Leigh syndrome is a severe, rare genetic neurometabolic disorder, typically appearing in the first year of life, that causes rapid degeneration of the central nervous system.
Symptoms include the loss of mental ability and movement skills, weak muscle tone, feeding difficulties, and respiratory issues. It is a fatal mitochondrial disease, usually leading to death by age 2–3.
“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, steps 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 improved energy metabolism and extended life span in mice and pigs carrying Leigh syndrome mutations.
“Based on these results, we decided to administer the drug as part of an individual therapeutic trial in six patients with Leigh syndrome,” said Dr. Alessandro Prigione, senior author of the study.
“Another decisive factor was the fact that detailed safety data was available for the long-term use of sildenafil in children, as the active ingredient is already approved for other pediatric disorders,” he continued.
While sildenafil is best known for treating erectile dysfunction, it’s also used to treat children with pulmonary arterial hypertension, a rare condition that causes high blood pressure in the arteries of the lungs.
For the study, researchers recruited six Leigh syndrome patients who began taking sildenafil between the ages of 9 months and 38 years. Within just a few months, their muscular strength and ability to move improved.
