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A drug known as a ‘gene silencer’ has shown promise in prolonging life and reducing admission to hospital for a type of heart disease, which is a common cause of heart failure in the elderly, according to a trial led by a Royal Free Hospital (RFH) doctor.
Vutrisiran, works by binding to a molecule called messenger RNA (mRNA) to reduce the production of a disease-causing protein called transthyretin.
mRNA works by carrying instructions from DNA, telling cells to make proteins. Once the drug binds to a particular type of mRNA, it cannot do its job and the cell cannot produce the harmful protein.
Researchers are hopeful the treatment – which is given as an injection – could become 'the new standard of care' for patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
ATTR-CM occurs when the faulty transthyretin protein builds up in the heart, making it more difficult to pump blood.
Professor Marianna Fontana (pictured below), of RFH and University College London, said: “ATTR is a progressive, fatal disease in which misfolded transthyretin protein accumulates as amyloid deposits in various parts of the body, often damaging the heart.
“We investigated whether vutrisiran, which targets transthyretin production, could improve clinical outcomes in patients with ATTR-CM and the results were very promising.”
The study, known as HELIOS-B, involved 655 patients from 87 sites across 26 countries, including patients at the National Amyloidosis Centre at the RFH.
The patients taking part in the trial were already suffering from symptoms from this condition and 40% were already taking tafamidis, a medication to delay disease progression, which they continued with throughout the trial.
For the study, half of the patients were given 25mg of vutrisiran once every three months for up to 36 months, while half were given a placebo.
Researchers found the risk of death and recurring 'cardiovascular events' – such as heart attacks or strokes – were markedly reduced in all patients (by 28%), irrespective of being on background medications such as tafamidis and SGLT2-i.
The trial also found that the quality of life and functional capacity of patients on vutrisiran improved compared to the placebo group.
Professor Fontana added: “Vutrisiran was highly effective and well tolerated in this contemporary population representative of patients that we see in our clinics, with consistent benefits regardless of background tafamidis therapy.
“Our findings indicate that vutrisiran has the potential to become the new standard of care.
“The data also suggests that vutrisiran may provide greater benefit to patients in earlier stages of the disease where, due to the progressive nature of ATTR-CM, early treatment can more effectively preserve functional capacity and quality of life.
“This trial is also important as it is the first to show the benefit of gene silencers in any type of cardiomyopathy.”
The findings were presented at the European Society of Cardiology Congress and published in The New England Journal of Medicine.
The results will now need to be reviewed by a regulatory authority prior to potentially becoming an available treatment option.
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