This Viagra ingredient just did something remarkable for a deadly childhood disease
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This Viagra ingredient just did something remarkable for a deadly childhood disease
Viagra may unexpectedly become the first promising treatment for a deadly childhood disease.
Date:
March 31, 2026
Source:
Charité - Universitätsmedizin Berlin
Summary:
A surprising breakthrough suggests that a drug best known as Viagra could help treat a devastating childhood disease. Researchers found that sildenafil significantly improved symptoms in patients with Leigh syndrome—a rare and often fatal disorder that affects the brain and muscles. In a small study, patients showed stronger muscles, fewer seizures, and better recovery from dangerous metabolic crises, with some experiencing dramatic improvements in mobility and daily life.
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Scientists have discovered that sildenafil—better known as Viagra—can improve symptoms in patients with Leigh syndrome, a rare and life-threatening disorder. Early results show stronger muscles, fewer seizures, and major quality-of-life gains, sparking plans for larger trials. Credit: Shutterstock
Sildenafil, the active ingredient in Viagra, may offer new hope for people living with Leigh syndrome, a rare and severe childhood disorder. Researchers from Charité -- Universitätsmedizin Berlin, working with collaborators from Heinrich Heine University Düsseldorf (HHU), University Hospital Düsseldorf (UKD), and the Fraunhofer Institute for Translational Medicine and Pharmacology ITMP in Hamburg, reported in the journal Cell that the drug improved symptoms in a small group of patients.
Leigh syndrome is a rare metabolic condition that affects the brain and muscles, usually appearing in infancy or early childhood. In a pilot study of six patients, sildenafil treatment was linked to measurable improvements in disease progression.
A Rare and Devastating Childhood Disorder
Leigh syndrome is caused by defects in cellular energy production. Because cells cannot generate enough energy, the brain and muscles are particularly affected. Symptoms often include seizures, muscle weakness, paralysis, and delayed development. Over time, the disease worsens, and life expectancy is significantly reduced. At present, there are no approved drug treatments.
An Unexpected Use for a Well-Known Drug
Researchers identified sildenafil, a PDE-5 inhibitor best known for treating erectile dysfunction, as a potential therapy. The drug is also used in infants to treat pulmonary hypertension due to its ability to widen blood vessels.
In the study, six patients between 9 months and 38 years old received continuous sildenafil treatment. Within a few months, many showed stronger muscles, and some experienced improvements in neurological symptoms. Patients also recovered more quickly from metabolic crises, i.e., overloads of the energy metabolism, which can suddenly worsen the course of this disorder.
"For example, in the case of a child undergoing sildenafil treatment, the walking distance increased tenfold, from 500 to 5,000 meters," as Prof. Markus Schuelke explains. "In another child, the therapy completely suppressed metabolic crises that occurred almost monthly, while another patient no longer suffered from epileptic seizures."
Markus Schuelke, a physician and scientist at Charité's Department of Pediatric Neurology and one of the study's lead authors, added: "Such effects significantly improve the quality of life of Leigh syndrome patients. While we will have to confirm these initial observations in a more comprehensive study, we are very pleased to have found a promising drug candidate for the treatment of this serious hereditary disease."
Why Rare Diseases Are Difficult to Treat
Leigh syndrome affects about one in 36,000 children, making research especially challenging. "The low case numbers make it difficult to research the disease and present some obstacles in our urgent search for effective therapies," explains Markus Schuelke.
Because so few patients are available, large clinical trials are difficult to conduct. Researchers often need to collaborate across multiple centers and countries. In addition, studying the disease directly is complicated, since brain or nerve tissue cannot easily be collected from patients.
Screening Thousands of Drugs for a Solution
To find potential treatments, scientists used an innovative approach. They collected skin cells from patients and reprogrammed them into induced pluripotent stem cells, which can develop into many different cell types. These were then turned into nerve cells that mimicked the same metabolic defects seen in Leigh syndrome.
Researchers tested more than 5,500 compounds that were already approved or had strong safety data. They evaluated how each substance affected the diseased nerve cells, leading to the identification of sildenafil as a promising candidate.
Benefits Seen in Cells, Animal Models, and Patients