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Duchenne Muscular Dystrophy

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 young 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 Ioan 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 how 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 chomping 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 now 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 both 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, now 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.