Progress in gene therapy for heart problems

9 September 2013

At least 750,000 people in the UK have heart failure, due to damaged muscle that reduces heart function – for example, following a heart attack. Treatments can alleviate the symptoms to some extent, and reduce the risk of further damage, but not repair the damage already sustained.
The world’s first trial of gene therapy for heart failure, CUPID2, is beginning, including 200 UK patients in London and Glasgow; it will assess safety, improvements in quality and length of life and any reduction in emergency hospital admissions of the treatment, which will be delivered via a coronary angiogram under local anaesthetic, a common procedure.  The gene therapy agent is a modified viral vector that contains SERCA2A genes; levels of SERCA2a protein have been shown to be reduced in heart failure patients.
It is hoped that increased levels of the protein will initiate self-repair mechanisms in heart cells and improve heart function in patients who receive the therapy, although it is not expected to be a full cure. However, even small improvements in function can have a big impact on quality of life and general fitness for patients, so if successful the therapy could have a significant impact.

Meanwhile, trials in Canada are to examine a combination of gene therapy and stem cell therapy for heart attack patients, which will attempt to stimulate the regenerative capacity of the stem cells by simultaneous delivery of genes for endothelial nitric oxide synthase enzyme.  

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