Page URL:

Gene therapy trials for heart failure launched

7 May 2013
Appeared in BioNews 703

Gene therapy will be used to treat heart failure for the first time in the UK, in clinical trials being launched by the British Heart Foundation.

The two trials will involve injecting copies of a healthy gene into the heart via a cardiac catheter and will be run by a team from Imperial College London. The first patients will start treatment in the next month at the Royal Brompton Hospital and over 200 people will receive the therapy worldwide.

The researchers will deliver a gene known as SERCA2a into heart cells using a virus known as an adenoassociated virus, which has been genetically altered so it is not harmful. The gene will cause the expression of a protein involved in calcium signalling that will allow the heart to pump more effectively, the researchers hope.

Heart failure is the term used to describe a variety of problems that can damage the heart, and affects more than 750,000 people in the UK. Those with heart failure often rely on medicines, but as their condition worsens they may have to use mechanical aids, such as a pacemaker, or wait for a transplant.

'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', said Dr Alexander Lyon, a consultant cardiologist at the Royal Brompton. '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 same team will also be testing the gene therapy in people who are using a type of mechanical heart pump called a Left Ventricular Assist Device.

Previous trials of SERCA2a gene therapy have shown that it is safe in humans and reduces the symptoms of heart failure. The researchers claim that the symptoms of those tested have been reduced, such as breathlessness and fatigue, and the treatment has resulted in fewer emergency admissions to hospital and fewer deaths.

The researchers are optimistic that the gene technique will improve the quality of life in some of the patients. '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', said Professor Sian Harding from Imperial College London.

However, Dr Lyon estimated that it will be at least eight years until the treatment becomes more widely available, as a longer-term study involving many patients will need to take place to confirm that the therapy is safe and effective. The treatment will not be suitable for everyone, as some people produce antibodies to the virus carrying the gene, which would be neutralised so that the therapy would have no effect.

Gene therapy: 'Heart-healing virus' trial starts
BBC News |  30 April 2013
New gene therapy could treat devastating heart failure (press release)
Imperial College London |  30 April 2013
New gene therapy trials aim to mend broken hearts
Reuters |  30 April 2013
Pioneering gene therapy trials offer hope for heart patients
Guardian |  30 April 2013
18 August 2014 - by Isobel Steer 
A gene therapy trial has been launched to help people with advanced heart failure who must constantly carry a bulky battery to power an artificial heart pump....
16 December 2013 - by Dr Rachel Montgomery 
Gene therapy trials using engineered immune cells have shown considerable progress in treating blood disorders, according to findings presented at the American Society of Hematology's annual meeting...
18 November 2013 - by Siobhan Chan 
Heart failure can be reversed using gene therapy, with the effect being amplified when two genes are used together, a study in pigs has found...
9 September 2013 - by Siobhan Chan 
The first patients from two separate ongoing studies have been treated using gene and stem cell therapies to repair damage caused by heart attacks...
27 August 2013 - by Lanay Tierney 
Researchers have concocted a 'gene cocktail' that may help to turn scar tissue into functioning cardiac cells after a heart attack...
12 November 2012 - by Dr Anna Cauldwell 
Eight heart attack patients given injections of stem cells harvested from their own hearts show significant improvement in their heart function two years after treatment...
15 May 2012 - by Helen Brooks 
Stem cell therapy may benefit patients with chronic heart disease, according to the results of a small clinical trial carried out by US scientists....
16 April 2012 - by Dr Kimberley Bryon-Dodd 
A woman with Parkinson's disease is reportedly able to write again for the first time in 15 years after receiving pioneering gene therapy at Addenbrooke's Hospital, Cambridge. Mrs Shelia Roy took part in an early stage clinical trial of ProSavin - a treatment developed by biopharmaceutical company, Oxford BioMedica....
19 March 2012 - by Heidi Colleran 
The largest trial for a gene therapy for cystic fibrosis (CF) will begin in the UK this month. Coordinated by the UK Cystic Fibrosis Gene Therapy Consortium (GTC), 130 patients will be recruited and given working copies of the defective gene that leads to their condition...
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.