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World first gene therapy trial for heart muscle launches

11 August, 2014
A patient with a mechanical heart pump, called an LVAD, has received a new gene therapy treatment for heart failure at London’s Harefield Hospital. The first of its kind in the world, the clinical trial, funded by the British Heart Foundation (BHF), will assess the treatment for patients with LVADs and provide greater understanding of how the gene therapy affects heart muscle.

Heart failure, where the heart is less able to pump blood around the body, affects hundreds of thousands of people in the UK. In many cases heart failure is caused by damage after a heart attack, where heart muscle and other tissue dies because its blood supply is cut off.


In severe cases heart failure can leave people disabled and gasping for breath, with a life expectancy of less than five years. In some of these cases patients are fitted with a LVAD (Left Ventricular Assist Device) which helps the failing heart in order to restore normal blood flow around the body.


Gene therapy aims to improve the function of weak heart muscle cells, whereas our Mending Broken Hearts Appeal funds regenerative medicine research aimed at finding ways to replace or regenerate dead heart muscle and other tissue after a heart attack.


The SERCA-LVAD trial, led by Imperial College London and funded by the BHF and Celladon Corporation, will explore whether this gene therapy could help the hearts of people with LVADs recover, offering an alternative treatment. This study complements the CUPID2 Trial, which is investigating the benefits of this treatment in 250 people with severe heart failure, who do not have LVADs.


The trial is led by Professor Sian Harding, Director of the BHF Centre of Regenerative Medicine at Imperial College London, and Dr Alex Lyon, BHF Research Fellow and Senior Lecturer, from Imperial’s Heart and Lung Institute and honorary consultant cardiologist at the Royal Brompton Hospital.


Professor Peter Weissberg, Medical Director at the BHF, which is funding the SERCA-LVAD trial, said: “Heart failure devastates the lives of hundreds of thousands of people in the UK. Despite major advances in treating heart attacks, we’re still some way off a treatment that restores function in hearts damaged by one. This cutting-edge trial offers genuine hope of an effective treatment in the near future.


“The trial has been made possible by decades of BHF funding for laboratory research and demonstrates the importance of translating promising laboratory findings into effective treatments for heart patients. Our new strategy puts greater emphasis on providing funding to ensure even more lab discoveries make it to clinical trials.


“Improved treatments for heart failure are urgently needed. That’s why we launched our Mending Broken Hearts Appeal to fund regenerative medicine research which could lead to treatments that replace areas of the heart damaged after a heart attack.”

  • Those wishing to help the BHF continue its work  may text FIGHT to 70123 and give £3, or make a donation by visiting

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