The National University Heart Centre Singapore (NUHCS) has become the first in Asia to recruit patients for a pioneering clinical trial aimed at treating transthyretin amyloid cardiomyopathy (ATTRCM), a rare and potentially fatal heart disease. The trial, known as MAGNITUDE, explores a novel gene editing therapy that could alter patients’ DNA to slow the production of harmful proteins causing the disease.
ATTRCM affects around 150 individuals in Singapore, including Chua Ah Hai, a 62-year-old participant in the trial. The disease is characterised by the build-up of misfolded proteins in the heart and other organs, leading to symptoms such as numbness, lethargy, and dizziness. If untreated, it can result in heart failure.
Assistant Professor Lin Weiqin, Clinical Director of the Heart Failure and Cardiomyopathy Programme at NUHCS, is spearheading the Singapore arm of this international study. The trial investigates the impact of the gene editing research medicine Nexiguran Ziclumeran (NTLA-2001) on ATTRCM. “If successful, it will be the first DNA altering treatment used in adult cardiology,” Lin stated.
The trial’s first Asian participant received the therapy in September 2024, with four more patients, including Chua, joining since. Early results have shown promising reductions in the abnormal protein with minimal side effects. NUHCS continues to recruit patients aged 18 to 90 who meet specific criteria, including a history of heart failure due to ATTRCM.
NUHCS, a leading heart centre in Singapore, is renowned for its expertise in treating complex cardiovascular diseases and is actively involved in cutting-edge research to improve patient outcomes.
“`
