World first gene therapy trial launches thanks to your support

11 August 2014        

Dr Alex Lyon, BHF research fellow

A patient with a mechanical heart pump, called an LVAD, has received a new gene therapy treatment for heart failure as part of a clinical trial funded by your donations.

The clinical trial is the first of its kind in the world to assess the treatment in people with LVADs. It will provide greater understanding of how the gene therapy affects heart muscle.

The trial is led by Dr Alexander Lyon (pictured), a BHF Research Fellow at Imperial College London and Professor Sian Harding, Director of our Centre of Regenerative Medicine at Imperial. The clinical trial involves 24 patients from Harefield Hospital, London and Papworth Hospital, Cambridgeshire.

Which patients are on the clinical trial?

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.

Diagram on an LVAD

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. It is this group of patients who will be given the treatment as part of the trial. Currently there are around 100 to 150 people in the UK living with an LVAD.

What does the clinical trial involve?

The trial is part of a huge range of groundbreaking research funded by us to help people living with heart failure – which also includes our Mending Broken Hearts Appeal, our quest to find a treatment to repair or replace lost heart muscle after a heart attack.

Each patient will be given either the gene therapy treatment or a placebo treatment, which should have no effect, meaning the researchers can compare the two patient groups to see exactly what the treatment does. The therapy involves giving the patient a harmless virus which adds DNA directly to heart cells to correct a genetic problem in failing heart cells, that can stop the heart from beating powerfully enough to pump blood round the body properly.

The researchers will take small samples of each person's heart muscle six months after treatment to detect if the gene has been taken up by the person's heart and measure if it is working.

Our hopes for this research

Our Medical Director, Professor Peter Weissberg, said:

This cutting-edge trial offers genuine hope of an effective treatment


“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.”

Support our life-saving research

This clinical trial marks the culmination of more than 20 years of research we've funded. It's only been possible because of the generous donations of our supporters.

Help us find a cure for heart failure today – support our Mending Broken Hearts Appeal.

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