‘Gene silencer’ drug could reduce deaths from rare heart disease

Drugs known as “gene silencers” have shown promise in reducing heart attacks , strokes and deaths caused by a rare heart disease, a trial has found. Vutrisiran, sold under the brand name Amvuttra, works by binding to, and stifling, messenger RNA (mRNA) to reduce a disease-causing protein called transthyretin. 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 happens when the faulty transthyretin proteins do not form correctly and build up in the heart’s main pumping chamber, making it more difficult to pump blood. The condition can be hereditary or develop with age. Professor Marianna Fontana, of University College London (UCL) and Royal Free Hospital, 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 a novel RNA interference (RNAi) therapeutic, 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. More than three quarters (77.

6 per cent) had heart failure and 40 per cent were already taking tafamidis, a medication to delay disease progression, which they continued with throughout the trial. For the .