Heart gene therapy trial begins


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

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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"

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  • 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.

  • rate this

    Comment number 25.

    Brilliant news! There are amazing advances happening all the time in this domain. I'm an ex by-pass patient, and very glad the doctors caught the problem before it caught me! The surgeons explained there is much money spent on heart research as of course without a heart, you don't live long! Let's hope this trial works, maybe they'll even find a cure for blocked arteries soon to!

  • rate this

    Comment number 24.

    'Researchers at Imperial College London found that levels of the protein SERCA2a are lower in patients with heart failure' - fantastic discovery, which is being applied practically to help the people of this country! It's research such as this that the UK should pump it's public money into, not bombs, wars, or slightly faster trains. Inspiring stuff! :)

  • Comment number 23.

    This comment was removed because the moderators found it broke the house rules. Explain.

  • rate this

    Comment number 22.

    This will likely cost a fortune. So it must not be given to people who have self inflicted diseases such as obesity and smokers.

  • rate this

    Comment number 21.

    If they can work out how to break down the blockages in arteries it would go a long way to sorting out heart damage from heart attacks. But it's a step in the right direction.

  • rate this

    Comment number 20.


  • rate this

    Comment number 19.

    I am currently being treated for Arrythmia, with warfarin, statins, and a wealth of other drugs too numerous to mention. I feel as fit as I think possible for my age, am very active and have not experienced a palpitation for about a year now. Apart from having the warfarin levels monitored, I no longer have to attend the Cardiac unit, so am left to assume that this situation is ongoing till I die!

  • rate this

    Comment number 18.


    I wish you the best of results as well.

    Don't worry too much about the -ve rated posts. To be honest, I would have thought the hurdle faced was the exact opposite of their content, in other words, that the immune system is actually pretty effective at dealing with things it thinks are viruses.

  • rate this

    Comment number 17.

    I am in the trial. Heart failure is the consequence of not only a heart attack but many other causes including genetics and viruses. The virus used to deliver the therapy is a natural virus and is not manufactured. The virus is doctored with the new protien so that it will deliver it to the heart muscle cells. Unlike other gene therapies this one does not splice it'self into your DNA.See SERCA2a

  • rate this

    Comment number 16.

    Excellent report from one of the BBC's finest correspondent who should have been promoted long ago.
    The analogy with a Trojan Horse is very effective and the whole subject is explained succintly, interestingly and in a way which makes us think of the way in which the future of medicine will develop as we use gene technology more and more.
    Thank you for an interesting article.

  • rate this

    Comment number 15.

    Good news. Good luck to all concerned. It's great to see this kind of thing actually being trialled.

    Of course, the more people we help, the more people we have - so we need to stop producing so many of ourselves. We'll need social advances along with the scientific ones and unfortunately that means controlling population, a very emotive subject that needs some hard decisions.

  • rate this

    Comment number 14.

    How much effect has the taking of statins on escalating heart failure cases ? Statins deplete the heart muscle of co q10 yet the medical profession are trying to get everyone to take them to keep cholesterol levels low. The drug companies are raking in fortunes via statins which could be doing more harm than good.

  • rate this

    Comment number 13.

    What a pleasant surprise. At last, with all the gloom and doom some good news to lift the spirits. Wishing all those involved on both sides the very best of luck.

  • rate this

    Comment number 12.


    Every time mankind introduces a new species (like this virus) ... it is a total disaster.

    There are 2 million feral camels destroying arable land in Australia.

    The majority of the seabed in the Mediterranean is now covered with kelp for fish tanks that has killed off anything else.

    Japanese beetles and African Slugs eating everything.

    Just STOP, mankind.

    Or die.

  • rate this

    Comment number 11.


    On the other side of the BBC is an article from Oxford University that says that whilst humans have Huge technological power, their responsibility is at Infant level.

    These Biotech solutions seem an example of that.

    So much financial pressure to monetise and make billions ... and reduce the safety checks to a minimum ... well it's all going to end in tears.


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