Gene therapy to offer up to 1m heart patients new lease of life

Synthetic virus that can boost heartbeat will be injected into sufferers for breakthrough trial

The first attempt in Britain to treat heart failure patients with gene therapy is to begin within weeks, as part of study aimed at improving the lives of up to a million people in the UK who suffer the debilitating and potentially fatal condition.

Click HERE to view 'how to treat a failing heart' graphic

Two clinical trials are planned for a few dozen British patients who will be deliberately exposed to a virus carrying a synthetic copy of a human gene known to be involved in boosting heartbeat.

The first trial will be carried out at the Royal Brompton Hospital in London and the Golden Jubilee National Hospital in Glasgow. The patients will be part of a group of 200 from around the world who will have the virus injected via a cardiac catheter inserted through a vein in the leg. A second trial at the Harefield and Papworth hospitals will be based entirely within the UK and involve 24 patients with chronic heart failure who are already fitted with an "artificial heart" known as a left ventricular assist device, which helps to pump blood around the body.

The aim in both trials is to inject additional copies of a healthy gene, known to be responsible for a key protein involved in regulating the rhythmic contraction of the heart muscle. It is hoped that the extra genes will remain active within a patient's heart for many months or even years.

Scientists believe the approach could lead to a significant improvement in the efficiency of the diseased heart to pump blood around the body – so improving the quality of life of thousands of patients with progressive heart failure who develop serious ailments as well as severe fatigue.

Scientists warned that it will still be several years before the technique can be made widely available. They do not want to raise hopes unduly as many previous gene therapy trials on patients with a range of other illnesses have failed to live up to expectations.

However, the heart researchers said they are optimistic that the gene technique will improve the quality of life in at least some of the patients, who would otherwise suffer deteriorating health and life expectancy – a third of patients die within a year of diagnosis.

"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 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 gene-therapy trial involves the injection of a harmless virus, called adeno-associated virus, which has been genetically modified with the SERCA2a gene responsible for a protein that stimulates contraction of heart-muscle cells.

Dr Lyon said extensive safety work has already been conducted on the virus and it has not been found to cause health problems in other gene therapy patients.

Once the virus has delievered the SERCA2a into the muscle cells, it is quickly degraded, he said.

Professor Sian Harding of Imperial College London said that extensive research on the SERCA2a gene has shown that it can be safely inserted into heart-muscle cells with the effect of boosting the size and speed of contraction. "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," Professor Harding said.

Professor Peter Weissberg, medical director of the British Heart Foundation, said gene therapy shows great promise in providing a new way to treat heart failure, but warned that there is still a long way to go.

"There was a terrific fanfare around gene therapy about 10 to 15 years ago. It was going to cure everything and, like all things in science, it wasn't that simple," he said.

"This project is a great example of the slow burn of good laboratory science translating into potential clinical treatment. We don't know if it's going to work yet, we all hope that it will."

News
peopleFrankie Boyle responds to referendum result in characteristically offensive style
Arts and Entertainment
'New Tricks' star Dennis Waterman is departing from the show after he completes filming on two more episodes
tvHe is only remaining member of original cast
Arts and Entertainment
tvHighs and lows of the cast's careers since 2004
News
news
PROMOTED VIDEO
New Articles
i100
Life and Style
Couples have been having sex less in 2014, according to a new survey
life
Arts and Entertainment
musicBiographer Hunter Davies has collected nearly a hundred original manuscripts
News
ebooksAn unforgettable anthology of contemporary reportage
Latest stories from i100
Have you tried new the Independent Digital Edition apps?
Independent Dating
and  

By clicking 'Search' you
are agreeing to our
Terms of Use.

iJobs Job Widget
iJobs General

Cover Supervisor

£75 - £90 per day + negotiable: Randstad Education Group: Are you a cover supe...

Marketing Manager - Leicestershire - £35,000

£30000 - £35000 per annum: Ashdown Group: Marketing Manager (CIM, B2B, MS Offi...

Marketing Executive (B2B and B2C) - Rugby, Warwickshire

£22000 - £25000 per annum: Ashdown Group: A highly successful organisation wit...

SEN Coordinator + Teacher (SENCO)

£1 per day: Randstad Education Leeds: Job Purpose To work closely with the he...

Day In a Page

Scottish referendum: The Yes vote was the love that dared speak its name, but it was not to be

Despite the result, this is the end of the status quo

Boyd Tonkin on the fall-out from the Scottish referendum
Manolo Blahnik: The high priest of heels talks flats, Englishness, and why he loves Mary Beard

Manolo Blahnik: Flats, Englishness, and Mary Beard

The shoe designer who has been dubbed 'the patron saint of the stiletto'
The Beatles biographer reveals exclusive original manuscripts of some of the best pop songs ever written

Scrambled eggs and LSD

Behind The Beatles' lyrics - thanks to Hunter Davis's original manuscript copies
'Normcore' fashion: Blending in is the new standing out in latest catwalk non-trend

'Normcore': Blending in is the new standing out

Just when fashion was in grave danger of running out of trends, it only went and invented the non-trend. Rebecca Gonsalves investigates
Dance’s new leading ladies fight back: How female vocalists are now writing their own hits

New leading ladies of dance fight back

How female vocalists are now writing their own hits
Mystery of the Ground Zero wedding photo

A shot in the dark

Mystery of the wedding photo from Ground Zero
His life, the universe and everything

His life, the universe and everything

New biography sheds light on comic genius of Douglas Adams
Save us from small screen superheroes

Save us from small screen superheroes

Shows like Agents of S.H.I.E.L.D are little more than marketing tools
Reach for the skies

Reach for the skies

From pools to football pitches, rooftop living is looking up
These are the 12 best hotel spas in the UK

12 best hotel spas in the UK

Some hotels go all out on facilities; others stand out for the sheer quality of treatments
These Iranian-controlled Shia militias used to specialise in killing American soldiers. Now they are fighting Isis, backed up by US airstrikes

Widespread fear of Isis is producing strange bedfellows

Iranian-controlled Shia militias that used to kill American soldiers are now fighting Isis, helped by US airstrikes
Topshop goes part Athena poster, part last spring Prada

Topshop goes part Athena poster, part last spring Prada

Shoppers don't come to Topshop for the unique
How to make a Lego masterpiece

How to make a Lego masterpiece

Toy breaks out of the nursery and heads for the gallery
Meet the ‘Endies’ – city dwellers who are too poor to have fun

Meet the ‘Endies’ – city dwellers who are too poor to have fun

Urbanites are cursed with an acronym pointing to Employed but No Disposable Income or Savings
Paisley’s decision to make peace with IRA enemies might remind the Arabs of Sadat

Ian Paisley’s decision to make peace with his IRA enemies

His Save Ulster from Sodomy campaign would surely have been supported by many a Sunni imam