RNA based treatment lowers levels of dangerous cholesterol carrying protein

Embargoed until: Publicly released:
Peer-reviewed: This work was reviewed and scrutinised by relevant independent experts.

Randomised controlled trial: Subjects are randomly assigned to a test group, which receives the treatment, or a control group, which commonly receives a placebo. In 'blind' trials, participants do not know which group they are in; in ‘double blind’ trials, the experimenters do not know either. Blinding trials helps removes bias.

People: This is a study based on research using people.

An RNA based treatment can help lower levels of lipoprotein A, a cholesterol carrying protein that increases your risk of heart attacks and strokes, according to the results of a trial conducted in Australia, the US, and United Kingdom. Reducing the levels of this protein could help prevent blockages from forming in blood vessels, but unlike cholesterol, it is not something you can reduce with diet and lifestyle choices. This world first human trial used a type of RNA, called siRNA or small interfering RNA, to stop the protein from being made in the first place.  At 150 days after a single injection, the levels of lipoprotein A in 32 patients were reduced by between 46 and 98 per cent, depending on the dose of treatment. The authors say the findings support further trials on the safety and efficacy of this siRNA.

Journal/conference: JAMA

Link to research (DOI): 10.1001/jama.2022.5050

Organisation/s: The University of Western Australia, Cleveland Clinic, USA

Funder: The trial was funded by Silence Therapeutics PLC and coordinated by Silence Therapeutics, Medpace (a contract research organization), and C5Research. Dr Ray received support from the National Institute for Health Research Biomedical Research Centre at Imperial College London.

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