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New hope for struggling hearts as gene therapy trial begins

 Targeting abnormalities in heart muscle calcium using gene therapy to restore heart function

30 April 2013

Researchers at Royal Brompton Hospital have begun the first UK clinical trials of a gene therapy for heart failure.

Heart failure, when the heart is unable to pump blood adequately, affects more than 750,000 people in the UK, causing breathlessness and hindering day-to-day activities.

The new therapy is designed to increase the levels of SERCA2a protein in heart muscle cells by using a harmless virus to insert extra genes into the cells.

The two clinical trials announced today mark the culmination of more than 20 years of research funded by the British Heart Foundation (BHF) at Imperial and Royal Brompton Hospital, which have identified SERCA2a as an important factor affecting how well heart muscle cells can contract in people with heart failure.

The trials will be led in the UK by scientists at Imperial and cardiologists at Royal Brompton and in collaboration with its sister hospital, Harefield, along with doctors at several other UK hospitals including Papworth in Cambridge and the Golden Jubilee National Hospital in Scotland.

Dr Alexander Lyon, consultant cardiologist at Royal Brompton Hospital, who is the UK lead investigator for both studies, said: “Heart failure affects more than three quarters of a million people across the UK. Once heart failure starts, it progresses into a vicious cycle where the pumping becomes weaker and weaker, as each heart cell simply cannot respond to the increased demand.

“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.”
The trials are the next step in the research after laboratory studies found that the gene therapy can be used to effectively restore function to the failing heart, in collaboration with colleagues from the United States.
Doctors plan to study the gene therapy in two separate clinical trials. The first, called CUPID2, will begin treating people with heart failure in the next few weeks in the Cardiovascular Biomedical Research Unit at Royal Brompton Hospital.

CUPID2 will assess whether cardiac gene therapy to increase SERCA2a is safe and can improve both quality and length of life, and reduce emergency hospital admissions, for heart failure patients. The trial will involve 200 patients with heart failure from Royal Brompton Hospital and other centres across the world.

The second trial, called SERCA-LVAD, is due to start recruitment in the summer of 2013. Co-funded by the BHF, this trial will test the SERCA2a gene therapy in 24 UK heart failure patients already fitted with mechanical heart pumps, known as left ventricular assist devices (LVADs). It will give vital information about the effectiveness of the therapy by measuring the amount of the SERCA2a gene and protein that has been introduced into heart muscle.

Professor Sian Harding, professor of cardiac pharmacology and head of the BHF Centre for Regenerative Medicine at Imperial College London, who developed the treatment, said: “It’s been a painstaking, 20-year process to find the right gene and make a treatment that works, but we’re thrilled to be working with cardiologists to set up human trials that could help people living with heart failure. It’s been a long journey to get to this point and we are proud to have helped to develop the first therapy under investigation in clinical trials for people with heart disease.”

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