Imagine finding out that one out of every 500 people you know — a teacher, a neighbor, maybe even a family member — carries a condition that thickens their heart muscle and can, in rare cases, cause sudden death. That's the reality for millions of people worldwide living with hypertrophic cardiomyopathy, the most common inherited heart disease. Now, researchers at the University of Manchester in the United Kingdom are offering a glimpse of hope with a drug that might help.
The drug is called trientine, and it was recently tested in a yearlong trial involving 154 adults with the condition. The study, called TEMPEST, compared people who took trientine against people who took a placebo — a harmless pill with no active ingredient. The results, published in the European Heart Journal, showed that trientine reduced heart muscle thickening, with the strongest effects seen in patients who had the most thickening at the start of the trial.
Professor Chris Miller from the University of Manchester said the findings suggest trientine could become a new way to treat this disease. "People with more advanced disease seemed to benefit the most, but larger studies are needed to confirm this," he said. "There were few side effects, and the drug was generally well tolerated."
So how does a drug meant for one condition end up helping the heart? The answer lies in copper, a mineral the body needs to produce energy, protect cells from damage, and control scarring. When copper levels drop inside heart cells, the heart struggles to make energy and the muscle can thicken. At the same time, loose copper outside cells can cause harmful stress and trigger scar tissue to form. Trientine, which has been used for over 50 years to treat a rare inherited condition involving copper processing, helps by boosting copper availability inside cells and removing excess copper from outside them.
Earlier research in animals showed that trientine improved the heart's energy production, reduced stress on cells, and decreased muscle thickening and scarring. A small previous study in people with diabetes-related heart problems also showed similar benefits. This new trial in Manchester is the first to test the approach specifically in adults with hypertrophic cardiomyopathy.
Dr. John Farrant, a Ph.D. student and the study's first author, said the findings mark an important milestone. "These findings provide the first evidence that targeting copper could open a new chapter in treating all patients with this condition," he said.
The trial is part of a larger effort at the NIHR Manchester Biomedical Research Center to develop new ways to diagnose and treat heart disease. Professor Danny McAuley, scientific director of NIHR Programs, called the results encouraging and said they demonstrate how partnerships between research organizations can drive life-changing discoveries.
While the results are promising, researchers caution that larger studies are still needed to confirm how well trientine works and whether it genuinely improves patients' daily lives and functioning. But for millions of people living with a condition that has long been managed rather than treated, this trial represents something rare: a potential new path forward.