HC Deb 10 February 2004 vol 417 cc381-8WH 3.28 pm
Kevin Brennan (Cardiff, West) (Lab)

This is the second time that I have secured an Adjournment debate on the issue of research into treatments and a possible cure for Duchenne muscular dystrophy, but I make no apology for returning to the subject for a second time in the Chamber. I do so not only because this is a terrible disease of which people know little, and which many have never heard of, although it is of a similar prevalence to better known conditions such as cystic fibrosis, but because I believe, as do many of the young people and parents who are affected by the disease, that for the first time in many years there is genuine hope of an effective treatment, and perhaps even a cure. More importantly, that belief is shared by many eminent scientists working in the field, to the extent that they are setting aside their traditional differences and, in many cases, their individual pursuits, to pull together and to pool their efforts to find a treatment and perhaps eventually a cure for Duchenne muscular dystrophy. Perhaps the only thing that they lack is adequate resources for undertaking the necessary research to unlock the prize of a treatment and eventual cure for the disease.

Duchenne muscular dystrophy is the result of a gene defect that causes the body not to produce a vital substance called dystrophin. Without that substance, muscle cells become weaker and gradually waste away. The condition is regarded, wrongly, as relatively rare. In fact, it affects about one in every 3,000 boys throughout the world, although there is some dispute about the exact figure. In other words, every Member is likely to have constituents who are affected by this disease. I certainly have such constituents. This is a very cruel disease —if it is appropriate to attribute a human trait to the result of a genetic lottery.

Most parents who are affected believe that they have a healthy baby son, as any normal parent would, until the symptoms begin to emerge when the child is a toddler. I say "most" because a test, which is now carried out routinely in Wales, can inform parents of the presence of the disease while the child is still an infant. That can be very important, because parents may want that information if they are planning to have more children. The test may help them to take decisions about their family in future.

Whenever the diagnosis is made, the result is the same. Boys with Duchenne muscular dystrophy can expect to be wheelchair-bound by the age of 11 and to become progressively more disabled throughout their teens as their muscles gradually waste away. At this stage of their lives, the availability of the latest high-tech wheelchairs can have a huge impact. Many of these boys are high achievers in school and do very well in their studies if they have the right support. According to parents to whom I have spoken, however, the availability of such wheelchairs varies greatly throughout the country. I ask the Minister to look into that issue.

In their final years, young men with Duchenne muscular dystrophy will require 24-hour care, night-time ventilation and feeding support. Although improved ventilation helps to extend life, the disease is fatal. Young adults are likely to die in their early 20s as a result of heart or lung failure as the vital organs become too weak to sustain life.

The disease is often hereditary, but it can also occur by spontaneous mutation, so even very careful genetic screening could not eradicate it completely. My interest in the disease arose when my constituents, Nick and Janet Caplin, brought their son Saul to my surgery a couple of years ago. It was very hard to believe that the lively toddler whom they brought to see me had, as they put it, a death sentence on his head. Nevertheless, he had been diagnosed with the disease, and Nick and Janet helped to set up a charity, Parent Project UK, to campaign with other parents and families affected by the disease for even more research into treatments and eventually a cure. They have now moved out of Cardiff, West, and it is a testimony to both the closeness of the parental network of Duchenne muscular dystrophy sufferers and its prevalence, which makes my point that the disease is not that rare, that their home was bought by another couple from Cardiff, West, Lisa and Gareth, whose son loan also has Duchenne muscular dystrophy. This is an extremely poignant week for that family, because it is the first anniversary of Ioan's diagnosis, which was made through the screening to which I have referred.

No one could fail to be moved and inspired by the courage and humanity of couples such as Nick and Janet and Lisa and Gareth, and of many other families whom I have met through my interest in this terrible disease. Sympathy is not enough, particularly now that the scientists are telling us that new developments offer hope for sufferers of Duchenne muscular dystrophy. Since the previous debate on the subject, Parent Project UK, the Muscular Dystrophy Campaign and the Duchenne Family Support Group, the three main charities with an interest in the condition, have got their act together and joined forces to act as a catalyst for more research to be undertaken into the disease. It is highly commendable that they have pulled together to try to make progress and to maximise the synergy that they can obtain from working together.

Last September, a group of us met Lord Warner at the Department of Health. I went along as chair of the all-party group on muscular dystrophy, which was set up as a result of my meeting with Nick and Janet. It was a fruitful meeting and an exciting research proposal was put together. There was a fairly stiff deadline, and I pay particular tribute to Jenny Versnel of the Muscular Dystrophy Campaign for her work in burning the midnight oil to submit the proposal by the deadline. The proposal was put together to bid for £2.5 million of funds that were set aside and earmarked in the Department of Health's commendable genetics White Paper. which was published last year. It is also commendable that the money was set aside specifically for research into single-gene disorders such as Duchenne muscular dystrophy.

I understand that the bid is under consideration and that it is going through peer review, so the Minister is probably limited in what he can say about it at this time because it will be considered with all the other bids for that money. Before a final decision is taken. I want to emphasise to him what a successful bid would mean to the morale of families affected by Duchenne muscular dystrophy, as well as of the scientists who formed the consortium, joining together centres of excellence from around the United Kingdom.

Let me illustrate the calibre of some of the people involved who are champing at the bit to get on with the research project. The project will be overseen and managed by the highly regarded Professor Kay Davies at the university of Oxford, and individual parts of the research will be undertaken by Professor Francesco Muntoni at Hammersmith hospital; Professor Kate Bushby at Newcastle medical centre; Dr. Qi Lu at Imperial college, London, who is a tenured member of the research staff of the Medical Research Council; Dr. Dominic Wells, reader at Imperial college, London; Professor George Dickon at Royal Holloway college, London, with Dr. Ian Graham of the same institution; Dr. Matthew Wood, also at the university of Oxford; and Jenny Versnel, whom I mentioned earlier, the head of research at the Muscular Dystrophy Campaign. Many of those eminent researchers came to a conference organised by Parent Project UK and the Muscular Dystrophy Campaign in the autumn, which I attended.

Recently, Professor Terry Partridge of Imperial college, who is also part of the consortium, came to the House of Commons to address the all-party muscular dystrophy group. At that meeting, at which many parents of boys affected by Duchenne were present, he said that he was usually extremely cautious about raising hope, particularly with regard to something like Duchenne muscular dystrophy, because it would be cruel to raise anybody's hope about a certain piece of research. However, he saw real promise in the approach suggested in this research proposal, perhaps for the first time ever.

I want to outline what the research proposal involves. In recent years, studies undertaken in mice and on human cells in the laboratory have shown that it is possible to produce a sort of molecular patch that could enable dystrophin to be produced in muscle cells, which would modify the form of muscular dystrophy suffered by the boys. If the technique were successful —all the evidence from the laboratory and the experiments involving mice suggest that it could work and is promising—it would modify Duchenne muscular dystrophy into the much less serious Becker form, which would considerably lessen the symptoms, because in the Becker form of muscular dystrophy some dystrophin is produced. The quality and length of life of boys suffering from Duchenne muscular dystrophy would therefore be greatly enhanced. The scientists believe that up to 60 per cent. of Duchenne muscular dystrophy sufferers could benefit from the treatment.

The principle has been established, but the scientists need the financial support to advance the research to human trials. The project submitted to the Department of Health would do the following: first, it would improve the design of the molecular patches; secondly, it would find the best delivery techniques to the muscles; thirdly, it would do further tests in animals and Duchenne muscular dystrophy human cells in the laboratory; and, fourthly, it would carry out the first human safety trials and see whether functional dystrophin is produced by that technique.

For those involved in Duchenne muscular dystrophy, including parents and researchers, this is the first flicker of light at the end of a long and dark tunnel. The possibilities of genetic research to do good, correctly identified in the Government's White Paper last year and too often overshadowed by media scare stories about cloning and so on, give hope where before there was only despair, but I cannot emphasise enough the need for that hope to be kindled.

To giant pharmaceutical companies, Duchenne muscular dystrophy is a rare disease, because their definition of rarity is anything that is unlikely to yield an easy profit. That is why it is so vital that the Government, through Department of Health White Paper money and the Medical Research Council, support this vital work. The scientists to whom I have spoken are convinced that the project will have spin-offs in other areas and for other genetic disorders.

This is not the only line of research. We believe that £20 million should be spent over the next few years to support other promising research projects in this area too. However, this £2.5 million is on the shelf, ready to run, and it is backed up by all the charities and the all-party group, and has 18 of the nation's top scientists ready to carry it out. I believe that it deserves to be funded in full.

Finally, I hope that the Minister will ask the Secretary of State to make time in the near future to meet the families of boys with Duchenne muscular dystrophy, as his colleague Lord Warner did last September, so that he can hear their stories, listen to their concerns, understand their plight and, most importantly, do whatever he can to help them.

3.45 pm
The Parliamentary Under-Secretary of State for Health (Dr. Stephen Ladyman)

I begin, as always, by commending my hon. Friend the Member for Cardiff, West (Kevin Brennan) on securing the debate. Those congratulations are well deserved. As he said, he has raised the subject before, has remained close to it in recent years and continues to work hard to press the Government on it. I welcome the opportunity to bring him up to date on the state of the issues raised.

My hon. Friend asked whether the Secretary of State would meet the families of children with Duchenne muscular dystrophy. I have no doubt that he would be happy to do so. He meets people who suffer from all manner of long-term conditions. However, he needs no convincing of the importance of the issue. That is why he was so enthusiastic about launching the Government's genetics White Paper. He is committed to doing everything that we can to deliver aid, support and, where possible, cures for such distressing illnesses. This is a distressing condition. As my hon. Friend said, boys with this genetic disorder have a very limited life span.

Voluntary organisations active in the area, such as the Muscular Dystrophy Campaign, Parent Project UK and the Duchenne Family Support Group, should also be congratulated on their work. It is important to raise public awareness of that dreadful disease and I hope that initiatives such as "The Right to Survive" will help to achieve that objective. I know that my hon. Friend will continue to do everything he can to ensure that people are aware of the issues involved.

As my hon. Friend said, Duchenne muscular dystrophy is an inherited severe and progressive muscle wasting disease. It is estimated that about 100 boys with Duchenne muscular dystrophy are born in the United Kingdom each year and that about 1,500 known boys with the disorder are living in the UK at any one time. It affects around one in 3,500 male births every year. It is caused by a genetic disorder characterised by progressive weakness and degeneration of the skeletal or voluntary muscles that control movement. Most boys with the disease will be in a wheelchair by the age of 10.

My hon. Friend will be pleased to know that among other things in my portfolio is responsibility for community equipment, including the wheelchair service. I heard what he said and am aware of the patchiness of the wheelchair service around the country. I assure him that I am already looking into that and trying to devise ways to improve it. I hope that he will pass that message back to the parents and the boys themselves.

Tragically, there is no effective cure or treatment for Duchenne. I understand, therefore, why the families of patients with Duchenne muscular dystrophy call for a greater research effort to be applied. Indeed, the Government are committed to investing money in genetic research.

Genetics offers enormous potential to improve our health and health care. Increasing understanding of genetics will bring more accurate diagnosis, more personalised prediction of risk, and more targeted and effective use of existing drugs. It will give us new gene-based drugs and therapies as well as prevention and treatment regimes tailored according to a person's individual genetic profile.

Unfortunately, market forces tend to militate against industry funding trials for rare disorders and inherited disorders. None the less, there remains an unmet need for effective, less burdensome treatments, and an estimated 750,000 patients in the UK suffer from incurable single-gene disorders. Gene therapy offers hope for those people and, to address that, the Department of Health supports gene therapy research into single-gene disorders.

As with any medical research, it is not possible to predict accurately when such treatments might become available or which approaches will ultimately prove effective. That is the nature of research. However, I share the hopes of the many parents, carers and patients that gene therapy will become a viable treatment for inherited disorders such as DMD, although we should not build up expectations and must realistically expect those outcomes to be a decade or more away.

I am aware that researchers have submitted a bid for funding for Duchenne muscular dystrophy research, the bid that my hon. Friend so eloquently described. He is right; I am not in a position to announce whether that bid has been successful. All I can tell the House is that the commissioning group for gene therapy research has met and considered the proposals. The outcome of the commissioning group's deliberations must remain confidential until the funding for each successful applicant has been agreed, and that has not yet been achieved.

My hon. Friend will recall that in April 2001 we announced £30 million to develop specialised genetic services. We have made further funding available for this important area. In June 2003, the White Paper "Our Inheritance, Our Future —realising the potential of genetics in the NHS" set out the Government's commitment to developing genetics knowledge, skills and provision within the NHS by investing more than £50 million over the next three years.

Kevin Brennan

I understand that the Minister is unable to discuss the outcome of the deliberations on the applications, but is he able to give any idea when the announcement might be expected on the final outcome?

Dr. Ladyman

I cannot give a precise date, but it is relatively imminent—weeks rather than months.

The White Paper details the following commitments: £3.5 million to fund 90 new grade A trainees in laboratory genetics and the equivalent of 10 full-time trainer posts; up to £18 million capital to upgrade NHS laboratory facilities in England; up to £1 million in information technology for genetics laboratories in the genetics testing network; £2 million of start-up funding over three years for other initiatives to bring the benefits of genetics to mainstream clinical areas; up to £2 million of start-up funding over three years specifically for primary care genetics initiatives; £4 million to fund pharmacogenetic research on existing medicines; £500,000 to pilot near-patient genetic testing in the NHS; £1.5 million to fund a range of research projects in the area of genetics-based health services; £3 million to support gene therapy research on single-gene disorders; £2.5 million for cystic fibrosis gene therapy research; and £4 million to provide access to clinical grade gene therapy vectors.

The emphasis for Department of Health funding is "translational", which means bringing the benefits of research to the clinic. Our vision is for the NHS to lead the world in taking maximum advantage of the safe, effective and ethical application of the new genetic knowledge and technologies for all patients as soon as they become available. In time, we should be able to assess the risk that an individual has of developing disease not just for single-gene disorders such as DMD and cystic fibrosis, but for our country's biggest killers—cancer and coronary heart disease—as well as those, such as diabetes, that limit people's lives.

The potential in genetic research is immense. While genetics will never mean a disease-free existence, greater understanding of genetics is one of our best allies in the war against disease. I hope that, over time, research will provide a breakthrough in the understanding and treatment of genetic diseases such as DMD.

The main agency through which the Government support medical and clinical research is the Medical Research Council. That independent body receives its grant-in-aid from the Office of Science and Technology. It is, however, a long-standing and important principle of successive Governments that they do not prescribe to individual research councils how they should distribute resources between competing priorities. That is best decided by researchers and research users.

The MRC spend relevant to DMD research for 2001–02 was around £2 million. That included two major groups of researchers: those of Professor Kay Davies at the MRC functional genetics unit in Oxford and Professor Terry Partridge at the MRC clinical sciences centre based at Imperial college in Hammersmith. Research projects are also being carried out on muscular dystrophy and basic underpinning work that could apply to all forms of the disease. The MRC does not, as a rule, earmark funds for particular topics; research proposals in all areas compete for funding. When appropriate, high-quality research in the areas that we are promoting may be given priority in competition for funds, but research excellence and importance to health will continue to be the primary considerations in funding decisions. In addition, it is important to note that while prevalence of a particular condition is one important factor, the MRC's funding decisions are set largely on the scientific opportunity and the likelihood of significant development.

There are, of course, other lines of research that may help in the future, especially with long-term conditions. The report by the chief medical officer's expert group, "Stem Cell Research: Medical Progress with Responsibility", was published in 2000. This recognised that the early research on stem cells is exciting and provides a real hope of new treatments becoming available in a few years for people with chronic diseases.

The Government are investing an additional £40 million in stem cell research in 2004 to 2006. We believe that stem cell research offers enormous potential to deliver new treatments for many diseases for which there are no effective cures. The Government are therefore funding the creation of a national stem cell bank. The first of its kind in the world, it will hold all types of stem cell as a resource for researchers. The Science and Technology Committee, in its recent report on the work of the Medical Research Council, welcomed the stem cell bank and called it a world leading venture that we wholeheartedly support. The Government want research to use all sources of stem cells, including embryonic stem cells. It is far too early to know where useful results will come from. That position is supported by published scientific findings. Any activity that involves the use or creation of an embryo outside the body—be it for treatment of infertility or for research—is permitted in the UK only under a strict system of licensing by the Human Fertilisation and Embryology Authority.

In February 2001 we announced the development of a national service framework for long-term conditions. NSFs are set out in the White Paper, "The new NHS". They are intended to set national standards and define service models for particular services or care groups. This NSF will have a particular focus on the needs of people with neurological conditions and brain and spinal injury. We are also clear, however, about the fact that it must tackle some generic issues that affect a wide range of people with long-term conditions and their families and carers. We hope that work to establish standards for neurological conditions will provide patterns of service provision that have wider application and will also benefit people with non-neurological conditions.

I hope that my hon. Friend agrees that the Government are sympathetic to the needs of patients with Duchenne muscular dystrophy and other longterm conditions and that we are backing that sympathy with real effort and resource. There has been progress since we last debated the subject. The initiatives I have described—the genetics strategy, the important work under way in the research field, developments in stem cell research and the national service framework—will help to make life better for patients with Duchenne muscular dystrophy, their families and their carers.

I am sorry that I cannot give my hon. Friend the answers that he wants on the the exciting research project that he described, but I hope that we have them for him soon. I certainly assure him, the parents and all sufferers with Duchenne muscular dystrophy that the Government have every sympathy and want to do everything they can to help them, both with the services that make their lives better and with the research to ensure that there are effective therapies in the not too distant future.