Hypertrophic cardiomyopathy is the most common inherited heart disease in the world, affecting about one in every 500 people.
The condition causes the heart muscle to become thicker than normal, making it harder for the heart to pump blood.
Some people never notice symptoms, while others develop shortness of breath, tiredness, chest pain, dizziness, or fainting.
A small number are also at risk of dangerous heart rhythms that can lead to sudden death.
Although doctors can treat symptoms and reduce some risks, there are still limited options that directly target the disease itself.
Researchers have therefore been searching for medicines that can slow or reverse the thickening of the heart muscle. A new study now suggests an older drug may offer a new way to help these patients.
The research was led by scientists from The University of Manchester and published in the European Heart Journal.
It tested a medicine called trientine in a clinical trial known as TEMPEST. The study included 154 adults with hypertrophic cardiomyopathy and compared trientine with a placebo over one year.
The results showed that people taking trientine had a reduction in heart muscle thickening. The improvement was greatest in patients whose heart muscle was already more severely thickened when the study began. The drug was also generally well tolerated, and only a small number of side effects were reported.
Trientine is not a new medicine. It has been safely used for more than 50 years to treat Wilson disease, a rare inherited disorder in which the body cannot handle copper properly. Because doctors already know a great deal about the medicine, it may be easier to study for new uses than creating a completely new drug.
Copper is an essential mineral that helps heart cells produce energy and stay healthy. However, copper must remain in the right place and in the right amount. Too little copper inside heart cells can reduce energy production, while too much loose copper outside the cells may increase harmful stress and encourage scar tissue to develop.
The researchers believe trientine helps restore this balance. It increases the availability of copper inside cells while removing excess copper outside them. Earlier studies in animals found that this process improved heart energy production and reduced both thickening and scarring of the heart muscle.
A previous study in people with diabetes-related heart disease also suggested that trientine could reduce heart muscle thickening. The new TEMPEST trial provides the strongest evidence so far that the drug might also help people with hypertrophic cardiomyopathy. This raises the possibility that it could eventually benefit patients with other conditions that cause heart muscle thickening as well.
Professor Chris Miller said the findings are encouraging but stressed that larger studies are still needed. Researchers must confirm that the medicine not only changes the heart muscle but also improves symptoms, daily activities, quality of life, and long-term survival.
Overall, this study provides promising early evidence rather than a final answer. The trial was carefully designed and included a placebo comparison, making the results more reliable, but it involved only 154 participants and lasted one year.
If future studies confirm these findings, trientine could become an important new treatment for one of the world’s most common inherited heart diseases.
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