A new drug offers hope for people with hypertrophic cardiomyopathy, the world’s most common inherited heart disease.
The TEMPEST trial led by University of Manchester researchers tested the drug – called trientine – in 154 adults with the condition in a year‑long, placebo‑controlled study.
The trial was funded by a partnership between the Medical Research Council (MRC) and the National Institute for Health and Care Research (NIHR). NIHR Manchester Biomedical Research Centre (BRC) supported the trial.
Hypertrophic cardiomyopathy – which affects around 1 in 500 people worldwide- causes thickening and scarring of the heart muscle.
The condition is sometimes asymptomatic, but other patients suffer from breathlessness, fatigue, chest pain and blackouts.
A small minority of people have an increased risk of developing a dangerous heart rhythm, which can lead to sudden death.
In the trial, treatment with trientine led to a reduction in heart muscle thickening. The effect was greater in patients with greater heart muscle thickening at the beginning of the trial.
It is a potentially new way of treating hypertrophic cardiomyopathy, and potentially heart muscle thickening caused by other conditions.
However, the researchers say, larger studies are required to confirm how well it works and whether it improves how people feel and function.
Professor Chris Miller from The University of Manchester said: “This trial suggests trientine may be a promising option for people with hypertrophic cardiomyopathy.
“People with more advanced disease seemed to benefit the most, but larger studies are needed to confirm this.
“There were few side effects, and the drug was generally well tolerated.”
Copper is an essential mineral involved in key processes in the heart muscle, including energy production, protection cells from damage, and controlling scarring.
When copper levels are low in cells, the heart struggles to make energy and the heart muscle can thicken. At the same time, loose copper outside cells can cause harmful stress on the cells and trigger scar tissue to form.
Trientine has been used for more than 50 years to treat a rare inherited condition that affects how the body handles copper. It improves the availability of copper within cells and binds and removes copper outside cells.
This study follows earlier research in animals showing that trientine improves the heart’s energy production, reduces harmful stress on cells and reduces heart muscle thickening and scarring.
In a previous small study in people with diabetes related heart problems, trientine led to a reduction in heart muscle thickening.
First author and PhD student, Dr John Farrant from The University of Manchester added: “These findings provide the first evidence that targeting copper could open a new chapter in treating all patients with this condition.”
The trial is part of the NIHR Manchester Biomedical Research Centre’s (BRC) Integrative Cardiovascular Medicine Theme, which aims to develop new ways to diagnose and treat heart disease.
Professor Danny McAuley, Scientific Director of NIHR Programmes, said:
“These promising results offer hope to patients, demonstrating how Trientine – a drug targeting copper metabolism – can treat this potentially fatal inherited heart condition.
“Once again this shows how MRC and NIHR’s key partnership can drive life-changing research. Together we are addressing complex clinical challenges and translating cutting-edge ideas into evidence-based treatments as part of the government’s mission for fewer lives to be lost to the biggest killers.”
- The paper Trientine for Hypertrophic Cardiomyopathy: a phase 2 trial, published in European Heart Journal is published in the European heart journal. https://doi.org/10.1093/eurheartj/ehag512
