Gene therapy: Ask the experts

The Cystic Fibrosis Trust's chief executive Rosie Barnes and Dr Helen Wallace, deputy director of GeneWatch UK, answered your questions on gene technology.

Click here to read the transcript

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An extra 50 million pounds is being invested by the Government in developing genetic science.

The new Health Secretary, John Reid told the Commons that he wanted Britain to 'lead the world' in this field.

He accepted there were some moral objections, but pledged that the law would be changed to make it an offence to test a person's DNA without their consent.

You put your questions to Rosie Barnes, chief executive of the Cystic Fibrosis Trust and to Dr Helen Wallace from GeneWatch UK in an interactive forum.

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Transcript

Susanna Reid:
Hello and welcome to this BBC News interactive forum, I'm Susanna Reid. The new Health Secretary, John Reid, has announced a major expansion in the use of gene technology. Fifty million pounds will be made available for genetic research in England over the next three years. Of this £18 million will be spent on upgrading NHS genetics laboratories and more than £7 million on developing services in primary care and hospitals. The government believes that genetic technology has the potential to transform healthcare.

Well you've sent in your questions on this subject and joining me to help answer them are Rosie Barnes, chief executive of the Cystic Fibrosis Trust and Dr Helen Wallace from GeneWatch UK.

And if I can ask you both, before we dive in with the questions, to explain your interest. First Dr Wallace, GeneWatch UK, what does it do?

Helen Wallace:
We're a science policy research group and our aim is to ensure genetic technologies are used in the public interest, so with proper safeguards for the public in how they are applied.

Susanna Reid:
And Rosie why do those affected by Cystic Fibrosis have an interest?

Rosie Barnes:
Well Cystic Fibrosis is a genetic condition. It's caused by a faulty mechanism in the gene that controls mucus in all the places in our body where we're meant to have it. Those of us, the vast majority, who have a perfectly normal genetic mechanism produce clear, thin, lubricating mucus that keeps our organs functioning well and free from infection. In those with Cystic Fibrosis this mechanism is faulty and the mucus is thick, congealing and prone to infection which eventually leads to severe long-term damage of the organs. It's still a life-threatening condition sadly and although we can do a certain amount to control the symptoms of Cystic Fibrosis there is, as yet, no cure and the cure is possible via gene therapy.

Susanna Reid:
Okay, so that's both of your interests clearly defined. Let's have a look at some of the e-mails. Alf from the UK asks: "Is this a matter for the government to decide and pronounce on or should it be regulated by those that have a grounding in medicine and medical ethics?" And a similar question from John McPhillips, here in the UK: "Is genetic technology too potentially dangerous to be left in the hands of politicians and business?" Dr Helen Wallace.

Helen Wallace:
Well of course there are potential benefits and there are potential dangers and we think government should be involved but it needs to set clear rules and regulations to stop misuse of genetic information, for example your insurer or employer wanting to know your genetic test results or commercial interests perhaps misusing genetic testing and trying to expand it to cases where it's really not appropriate.

Susanna Reid:
Rosie Barnes do you think that it's all in the right hands or could get into the wrong hands?

Rosie Barnes:
I think the government are to be applauded for taking an interest in this area, it's very much appreciated by those affected by Cystic Fibrosis. And they have set up a number of bodies including a body called the Gene Therapy Advisory Committee, particularly relevant to our interests, which controls all the clinical trials involving human beings on any new research project for gene therapy in the UK. And that's made up very eminent doctors, scientists and lay representatives and I think has given a very thoughtful and careful approach but nevertheless an encouraging approach to try and develop gene therapy. So I think the mix is roughly right.

Susanna Reid:
Gary Horsley from the UK says: "A while ago it was claimed the human genome had been fully mapped. Why is gene therapy so slow off the mark in treating known diseases and other health problems?" Rosie Barnes has that been a source of frustration?

Rosie Barnes:
It has. I think the confusion here arises because the human genome project identifies the genetic makeup and what genetic predispositions may have but doesn't do anything in terms of altering them in any way. The gene that causes Cystic Fibrosis was identified in 1989 and it's been a source of great frustration to families that since then nothing has happened in terms of effective treatment being made available. And that's because it's very difficult to change genes, to modify them to correct a fault and to ensure you're doing that with absolute safety. It's a long hard process which is why it's taking us a long time and costing us a lot of money.

Susanna Reid:
Dr Wallace, Paul from the UK says: "How far has knowledge in genetic science reached applicability? It appears that merely a trial and error technology has been established which could create unwanted, uncontrollable side effects. Would it not be desirable to invest more in fundamental genetic research?" Well I suppose the Government's announcement goes some way to doing that but how close are we?

Helen Wallace:
Well it really depends what disorders that you're talking about, so, for example, Cystic Fibrosis we do know the gene and therefore we've got a good starting point to start working on some kind of treatment. But we're particularly concerned about some of the claims that we can expand these kind of genetic tests and technologies to common complex illnesses that everybody has and there's a real danger .

Susanna Reid:
Like what?

Helen Wallace:
Well like heart disease and cancer, some forms of cancer do run in families but most common forms of cancer have much more to do whether you smoke or not, your lifestyle and your environment and even your social status and poverty than they do to do with genes. And so there is a real that we put too much emphasis on genetic factors instead of tackling things like the growing incidence of obesity that we're seeing in the country. Now that's not to say that we shouldn't of course put the money into genetic disorders, that needs to go in those, but a lot of companies who are funding genetic research are actually funding genetic research on those common complex illnesses because they're the ones where they think they'll make the money.

Susanna Reid:
Well cancer is the holy grail isn't it? Let's put a point to you - M. Walmsley from the UK says he's the father of a child with Cystic Fibrosis but is very concerned about where that funding will go. This is his question: "How will the distribution of extra funding in this important area be controlled? Isn't there a danger that the government and researchers will wish to focus their efforts in finding a cure for cancer rather than concentrate on a genetic disorder like Cystic Fibrosis that only affects a relatively low proportion of the population?" Rosie Barnes is that your concern?

Rosie Barnes:
Well that has been our concern and I have written to the government on this matter regularly over the last two years and today is the first evidence we have had that they've been listening and thinking about it and have decided to do something about it. We were concerned that both the government's funding and the major pharmaceutical companies' funding was all going into conditions which affect a great many people and that the conditions like Cystic Fibrosis were losing out because to find a cure via gene therapy for a condition that only affects a few people is just as costly as finding a cure for one that affects millions of people. But today we have for the first time been given a promise of £2.5 million towards our £15 million gene therapy appeal by the government and we are very appreciative of that. I had asked them for £6 million but I don't want to be petulant today when people have listened and helped. So we do want to get ourselves on the government radar screen and I think today we have some evidence that they are taking us seriously.

Susanna Reid:
Dr Wallace what is significant about what's happened today that does provide a safeguard that the money won't just go into those illnesses like cancer?

Helen Wallace:
Well I think I certainly agree that it's good that public money has been put towards genetic disorders and that some money is also going into improving the services for people with other genetic disorders who can benefit from genetic testing. I think what continues to worry us is that the bigger safeguards aren't in place, like safeguards to prevent insurers and employers using your genetic test results. And there's also a lot of misleading hype that genetics will solve these other more common complex diseases and I think that's very questionable.

Susanna Reid:
Well Matt Bolitho from England says: "What in your opinion will be the first illness to be successfully treated by gene therapy and when do you think that might happen?"

Helen Wallace:
Well there already have been some young boys who have been successfully treated for, what's sometimes known as, bubble boy syndrome, which is - the technical name is SCID, it's an immune problem. But there have been problems with that therapy and two of those children have very sadly got leukaemia as a result, showing that it's very much still an experimental therapy. There's certainly hope, of course, that we can progress beyond that and make more difference to single gene disorders but I think it's highly questionable whether we can treat something complicated like heart disease in the same way.

Susanna Reid:
And Rosie Barnes, Liz Furnass from the UK and Fiona Keyzor from the UK, both directly affected by Cystic Fibrosis in their family, Liz asks: "What's the position of gene therapy as a treatment for Cystic Fribrosis and when is it likely to be available? My two and a half year old grandson was diagnosed last week." They're currently trying to understand how this announcement will affect them. And Fiona Keyzor from the UK says her seven-year-old son has the disease, only diagnosed last year, desperately happy to hear this news but do you know, at this stage, how much the Cystic Fibrosis Trust will receive in support? Well you've already said. And does this mean a cure is closer? Can you give some sort of hope and a timescale to people like that?

Rosie Barnes:
Well I can try, there are no guarantees with research because if we know exactly how to do it we wouldn't be having to do the research and we're trying to overcome very major problems. But the first clinical trials for gene therapy for Cystic Fibrosis were carried out in the mid 1990s and the results were encouraging but limited. They demonstrated that in a third of the patients there was a positive reversal of the defective gene into being a correct copy of the gene but it was brief and short-lived and fairly weak in effect. Since then we've gone back to the drawing board and we are now able to predict a much longer and a much stronger benefit from gene therapy and it being taken up by the lungs of more people with Cystic Fibrosis. The problem we face is that those with very severe lung damage may not be able to benefit and we expect that the biggest benefit will come to babies, children, who have as yet managed to protect their lungs from some of the ravages of Cystic Fibrosis. All the families in the UK will tell you that it's always five years and they get very frustrated by that. Some five years ago now we made them a five year pledge with a commitment of £15 million but it's taken us longer than we hoped to raise that £15 million, so we're behind schedule, not because of the scientists lack of ability or interest or willingness but because of our lack of money. But we're already paying for 37 scientists to work full-time on this project and the extra money we're going to get will enable us to recruit the extra 20 or so we desperately need. And we do think that we will have identified a treatment and proved it's safe and effective ready to be going in large scale clinical trials in between three and four years. So for the parents of babies and younger children and for teenagers whose Cystic Fibrosis has been well controlled there's a great deal of hope indeed.

Susanna Reid:
Dr Wallace do you share that hope?

Helen Wallace:
I think there's some reasons to be hopeful, there are other reasons to be doubtful, it's really difficult to know whether this kind of therapy is going to produce the intended benefits, there have been many setbacks, we don't want to throw away people's hopes but we also don't want to raise false hopes. I'd be really reluctant to suggest any kind of timescale, we just don't know.

Susanna Reid:
Rosie one of our viewers is interested in just challenging you on a point, Bernard Wasney from Hampshire says: "Rosie Barnes stated that more of the money is being directed towards gene therapy as they see this treatment as the main hope. I do regard this as worrying, gene therapy does not cure the disease but rather alters the patient's genetic makeup to avoid the disease. If insufficient resources are available for proper investigation of cures then such cures may never be forthcoming." What's your response to that?

Rosie Barnes:
Well firstly I think stopping the symptoms developing is effectively a cure, so I don't want to play with semantics. And I think it's important for people to know that the Cystic Fibrosis Trust, whilst it's currently prioritising gene therapy as the major source of new funding to be released, of our current research funding this year three quarters goes into science and clinical control research other than gene therapy. So there's a great deal of research into the basic science, what goes wrong in the lungs and other organs of those with Cystic Fibrosis and how that might be corrected by other means as well as gene therapy. So we've always been very aware of the danger of having all our eggs in one basket. I think we've avoided that danger with putting a few more eggs in the gene therapy basket at the moment because it's been under-egged or under-funded, so to speak, and we feel it does offer the best hope for a significant improvement in both length and quality of life. But I'd like to assure all concerned that that's not being done at the expense of exploring all other potential benefits for those with cystic fibrosis.

Susanna Reid:
Let's have a look at just some other conditions Dr Wallace because obviously there's people who may be affected who have other conditions apart from Cystic Fibrosis. Stephen Irons from Scotland says: "I'm a diabetic. What I'd like to know is will gene therapy cure or prevent diabetes?" And Sonny from the UK says: "Will genetic therapy have any effect on diseases like Thalassaemia and Sickle Cell?" What do you think?

Helen Wallace:
Well this is where it starts to get more complicated because things like Thalassaemia are genetic disorders but they're also very complicated ones, they involve a lot of different genetic changes and it's very difficult to say at this stage of the research whether there will be real benefits from gene therapy or whether there won't. Your question from the person with diabetes doesn't make it clear whether - what type of diabetes they may have and for some types of diabetes genetics is quite important but for the most common type of diabetes that we think of, as adult onset diabetes, the biggest risk factors really are your diet and what you eat and your lifestyle. We're seeing a growing increase in diabetes, we're seeing it happening or even beginning to happen in children, the type of diabetes we only see in the elderly and that would be the kind of disease where GeneWatch would be worried that too much emphasis is going on genetic factors and we need to look at ways to address public health and cut down on the bad foods that we're all eating.

Susanna Reid:
Rosie, Liz Beatty from the UK says that she doesn't disagree with the possible use of gene therapy to treat such conditions as Cystic Fibrosis but she asks the ethical question, because the logical extension is that you could then remove genes from embryos for conditions such as deafness. How do you cope with the argument that these then get into value judgements of over what conditions you let your child have and those which you prevent?

Rosie Barnes:
Well I think there will always be ethical discussions and by and large the Cystic Fibrosis Trust always supports the principle of informed choice within the law. At the moment if a family have a child with Cystic Fibrosis they are then entitled to have genetic tests on further unborn children to see if they too carry Cystic Fibrosis and if they do and if the parents wish they may have that pregnancy terminated. That's by no means everybody's choice because children live much longer than they used to do and have full and happy lives and go on into adulthood and we've got doctors, barristers, teachers, sportsmen and women with Cystic Fibrosis. So people think very hard and seriously about taking those decisions. But this is an area where I don't think the government should be the overall deciding body, I think they should lay down the framework of the law within which decisions can be made and then ethical decisions should be made by the families concerned. And on the question of deafness, on a personal note I have a son with a very severe hearing loss and it certainly has made life very difficult for him in a whole variety of ways and I think people have to think about not just the family situation but the world in which we live and the difficulties that people have in adjusting, being accepted by their peers. And people have to make decisions as to when a quality of life is appropriate. But I think we get into a complicated area there. In relation to Cystic Fibrosis what we want to do above all else is to cure the disease for those who've already got it, so that we avoid having to make these very tough decisions.

Susanna Reid:
Okay, let me put the final question to you Dr Helen Wallace. Steve Goulding from England says: "Do you foresee a time when human life can be extended by gene therapy?" Not just for those who have short-term life threatening conditions but for all of us and is that a good prospect?

Helen Wallace:
Well I don't see that really happening, I think that's part of the hype around genetics so that we'll be able to tackle common sufferings like ageing itself and that it really will be too complicated and difficult to try and extend those kind of things. But yes we do also have to ask to what extent do we want to extend our lives, it's probably better to concentrate on a good quality of life and treating people who are actually suffering from serious diseases than it is to simply think in terms of everyone living that little bit longer.

Susanna Reid:
Very interesting debate. Well I'm afraid that's all we have time for. My thanks to our guests, Rosie Barnes and Helen Wallace, and to you for your many e-mails. From me Susanna Reid and the rest of the news interactive team, thanks for joining us and goodbye.