Heart gene therapy trial begins


Carol Gedda: 'It could improve the heart muscle. I'm really pleased to be part of it.'

Related Stories

It is 18 months since Carol Gedda suffered a massive heart attack. It left her with just 20% of her heart functioning.

"I have a lot of trouble with stairs, and sometimes I can even run out of breath in a conversation", says Mrs Gedda, who is 65.

She is one of at least 750,000 people in the UK with heart failure. It occurs when the heart is damaged and becomes unable to pump blood adequately.

There are treatments for the condition but nothing so far that can reverse the damage.

Mrs Gedda, from Essex, is among 200 patients being enrolled on a gene therapy trial to test whether introducing genetic material into damaged heart cells can improve their function.

Researchers at Imperial College London found that levels of the protein SERCA2a are lower in patients with heart failure.

Royal Brompton Hospital in London, where Mrs Gedda is being treated, is one of only two British centres taking part in the international study, The Golden Jubilee National Hospital in Glasgow is also involved.

Before joining the trial Mrs Gedda had baseline measurements taken for her fitness.

She walked up and down a 30m hospital corridor for six minutes - the distance she travelled was noted by one of the hospital researchers.

Her heart function was also analysed.

At Royal Brompton, the gene therapy is delivered at the NIHR biomedical research unit, via a coronary angiogram under local anaesthetic.

Trojan horse

The researchers have 'hidden' the gene inside a genetically modified virus which is able to latch on to heart muscle cells but is believed to be entirely harmless.

The virus acts like a Trojan horse, delivering the extra DNA into the nucleus of the heart cells.

The hope is the gene will prompt the heart cells to produce more of the SERCA2a protein and repair some of the damaged heart muscle.

Half of the patients will receive the gene therapy, while the rest will get a placebo or dummy drug.

"I'm delighted to be on the trial," says Mrs Gedda. " Of course I don't know whether I've received the gene therapy or the placebo but it is exciting to be part of it. I have three sons and my heart problem is partly genetic so it could be helpful to my family in the future."

The trial, known as CUPID2, is funded by the US biotechnology company, Celladon. It will be around three years before the results are known.

Dr Alexander Lyon, British Heart Foundation senior lecturer and consultant cardiologist at Royal Brompton hospital and Imperial College stresses that the treatment is far from being a cure, but says there is great excitement about the trial.

"A few patients in the United States who received the same dose we are using appear to have done extremely well.

"Importantly, early trials suggest the treatment is safe. But we need this large study before we can be sure the gene therapy really works. If we could find an effective treatment, that would be very exciting."

David Palmer, from Norfolk was the first patient in the UK to have the treatment.

The left side of his heart is enlarged and he suffers arrhythmias - irregular heart rhythms.

He said: "My heart is forever jumping out rhythm - I don't pass out just feel faint and very weak.

"Once I fell into a fridge in Sainsburys. I wasn't hurt but it was a bit embarrassing."

Mr Palmer, aged 50, says he is unsuitable for a heart transplant because he has had the condition so long, which leaves the gene therapy trial as his best hope:

"For people like me who are in the last chance saloon it gives us the opportunity to stabilise our condition and live that bit longer. It's far from being a cure but if it works then it would be a major advance for huge numbers of patients."

David Palmer: "it's not a cure....it's a huge bonus"

Fergus Walsh Article written by Fergus Walsh Fergus Walsh Medical correspondent

Defeating cancer, the 'evil genius'

Can we win the war against cancer? Over the past 18 months, Panorama has followed a group of patients on drug trials. Some who'd been given months to live, are keeping cancer at bay for years.

Read full article

More on This Story

Related Stories

The BBC is not responsible for the content of external Internet sites


This entry is now closed for comments

Jump to comments pagination
  • rate this

    Comment number 30.

    Its great to c that Gene therapy is progressing leaps and bounds. I wish all the scientists working on such subjects that will help all of us one day or the other, all the best. And to the Government, Bureaucrats and Politicians, please stay out of their way and provide them with all possible help. But that might be difficult to do; they are not ur rich friends unlike the ones in The City.

  • rate this

    Comment number 29.

    Personally I'm torn between being amazed at the advancement of science, medicine and technology; and prolonging of life beyond it's useful and productive years. We all have to die sometime, and it won't all be in our sleep why do we keep fighting the most natural of processes?

  • rate this

    Comment number 28.

    Well done the team at Imperial!

    I am fortunate enough to attend a hospital within the 'Imperial group' and their research into this and orthopaedics is nothing short of transformational in changing lives. I speak from experience.

    Forget HS2, how much more could be done in medical research with £35billion?

  • rate this

    Comment number 27.

    Gene therapies pose danger of “insertional problems” i.e. accidental triggering of nearby gene may cause other conditions, like cancer.
    It's hard to assess value of gene therapy because of unresolved questions around placebo-tests (which is no test at all); gene therapy should be tested against best alternative therapy available =
    A. Does gene therapy truly work better?

  • rate this

    Comment number 26.

    No 21, Ronnie. This has nothing to do with blocking arteries. They have worked out how to unblock arteries, it is called surgery and a stent, or beta blockers, aspirin, and a number of other blood thinning measures. However, a good way to avoid blocked arteries in the first place and stop them re-occuring is by a healthy lifestyle, including a good diet and exercise.


Comments 5 of 30



Copyright © 2015 BBC. The BBC is not responsible for the content of external sites. Read more.

This page is best viewed in an up-to-date web browser with style sheets (CSS) enabled. While you will be able to view the content of this page in your current browser, you will not be able to get the full visual experience. Please consider upgrading your browser software or enabling style sheets (CSS) if you are able to do so.