First UK trials of heart failure gene therapy

Markus MacGill

pharmaphorum

The first UK clinical trials of gene therapy for heart failure have begun. The British Heart Foundation at Imperial College London and the Royal Brompton Hospital are collaborating on two trials that are looking to reverse heart failure by boosting SERCA2a protein levels in heart muscle cells.

The first trial, called CUPID2 and funded by US biotech Celladon, will assess the safety of the gene therapy and test whether it can improve both quality and length of life, and reduce emergency hospital admissions. It will involve 200 heart failure patients.

The second trial, called SERCA-LVAD and co-funded by the British Heart Foundation charity, will test the SERCA2a gene therapy in 24 heart failure patients already fitted with mechanical left ventricular assist devices. It will measure the amount of the SERCA2a gene and protein that has been introduced into heart muscle.

“Our goal is to fight back against heart failure by targeting and reversing some of the critical molecular changes arising in the heart when it fails.”

Dr Alexander Lyon, study lead investigator

The trials are the culmination of 20 years of research to find the SERCA2a gene and a potentially effective treatment.

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