Duchenne Muscular Dystrophy
The final item of business is a members’ business debate on motion S4M-04027, in the name of Jim Eadie, on caring for and curing boys and men with Duchenne muscular dystrophy. The debate will be concluded without any question being put.
Motion debated,
That the Parliament commends the work of Action Duchenne in supporting boys and men across the Lothians and the United Kingdom who are living with Duchenne muscular dystrophy (DMD), which affects one in every 3,500 male births in the UK; welcomes what is considered groundbreaking research such as the AAV U7 trial being conducted at Royal Holloway College, University of London, into potentially highly effective “exon skipping” treatment for the currently incurable muscle-wasting disease; recognises that DMD is caused by a genetic variation in the dystrophin gene and can result in boys affected requiring a wheelchair by the ages of 8 to 11; notes that the current research is expected by Action Duchenne to produce the first wave of genetic medicines by 2014, with the potential to benefit at least 14% of patients, and has the potential to realise even more successful treatments in the future; believes that a simplified means of clinical trials for genetic medicines should be explored to promote further research, and looks forward to continued work toward improvements in the care and treatment of the boys and men living with DMD and the ultimate goal of an effective cure.
17:08
Parents are not meant to outlive their children. It is impossible to imagine what it must be like for any parent to contemplate living longer than their child, but that is the exactly the position that tens of families across the country face.
I am grateful to my colleagues in all parties—45 of whom have signed the motion—for allowing this debate on Duchenne muscular dystrophy to take place.
Duchenne, which is a currently incurable muscle-wasting disease, affects one in every 3,500 male births in the United Kingdom. The condition, which is devastating and is the most common form of muscular dystrophy, mostly affects boys whose bodies do not produce dystrophin, which is a protein that helps to protect muscle. Without it, the muscles weaken, break down and are replaced by fibrous tissue. The first symptoms appear between the ages of one and three, and they usually result in the boys who are affected requiring a wheelchair by the age of eight to 11. Those who are affected may eventually need spinal rods to keep upright. By the time the person is 30, Duchenne is life threatening, as their heart and lungs are affected. In the most severe cases, people need respirators to breathe.
The motion commends the work of Action Duchenne, which is a charity that supports boys and men who live with DMD. As well as paying tribute to Dean Widd, the charity’s regional development officer, I give special thanks to my constituent John Miller, advocacy officer for Action Duchenne Scotland, his grandson Lee, aged 14, who has Duchenne, and Lee’s dad, Gary, all of whom are in the public gallery this evening. I also thank Lee’s mum, Joyce, who is looking after Lee’s brothers this evening. John Miller has done a phenomenal amount to raise awareness of the condition while lobbying for DMD to achieve the clinical and political priority that it deserves.
I also pay tribute to the boys and men living with Duchenne, their families and the care support workers and healthcare professionals who are involved in their care. I also record my thanks to the Muscular Dystrophy Campaign for all its work.
I am grateful to Dr Keith Foster of the University of Reading—formerly of Royal Holloway College, University of London—for making me aware of his groundbreaking research
“such as the AAV U7 trial into potentially highly effective ‘exon skipping’ treatment”,
which is highlighted in the motion.
The landscape for the development of gene medicines based on adeno-associated viruses has changed considerably over the past two years. Earlier this month, the European Commission licensed an AAV gene medicine—Glybera—for the first time. Although not in the field of DMD, that is a landmark moment as it defines a clear pathway for AAV-based gene medicines from the research laboratory bench to regulatory approval.
Two life science companies, GlaxoSmithKline and Prosensa, have already started treatment in a phase 3 clinical study in ambulant boys with DMD who have a dystrophin gene mutation amenable to an exon 51 skip—up to 13 per cent of boys with DMD. That randomised, placebo-controlled study has enrolled 180 patients from 18 countries and is currently the most advanced on-going study. I understand that all of the phase 3 clinical trial data are expected to be submitted to the European Medicines Agency—the evaluation agency—by early 2014.
Two further AAV-based research programmes have recently been approved and are recruiting patients for clinical trials that will start early in 2013. One research programme, based in Paris, will try to mask the mutation within the boys’ muscle cells. The other research programme, based in the United States, will deliver a truncated DMD mini-gene using AAV into the muscles of DMD patients. In respect of clinical translation, an important point is that parameters to safely deliver adeno-associated viruses systemically to humans have also recently been defined and approved.
According to Dr Foster,
“Within the field of DMD research, progress has been astounding ... For the first time, I have developed a method to restore a full length DMD gene to muscle using three safe AAVs.”
Dr Foster states that this research is highly significant,
“such that a programme will be applicable for all patients with DMD and is long overdue.”
There is an opportunity for Scotland to be part of that groundbreaking research. High-quality testing relies on clinical trials involving large numbers of patients. For muscular dystrophy, Scotland does not have the patient population required to achieve that on its own, but that does not mean that we cannot or should not contribute to United Kingdom and international research.
Contributing to international research is one of the main functions of the Scottish muscle network, which has been involved in encouraging participation in global trials in a number of ways. The network also promotes the Duchenne registry, which ensures that families can be contacted about trials in which they might participate. There are currently around 70 people on the register.
Such collaborative approaches are where the focus must lie, and that is where the Scottish Government, through the chief scientist office and the health research strategy, has an important role to play. Action Duchenne is contributing £161,000 to support Dr Foster’s research at the University of Reading. I would like to see Scotland play its part through co-funding arrangements so that we can support the joint initiative between the University of Reading and the University of Glasgow. Future studies in Glasgow will evaluate improvements to cardiac tissue. A co-funding arrangement would allow greater value and greater resource to be generated by the chief scientist office, working in partnership with medical research charities and potentially other health departments in the UK, to support this pioneering research. That has the potential to deliver the genetic medicines that will benefit boys and men with DMD.
The motion refers to the need to explore a simplified means of clinical trials for genetic medicines. I am delighted that progress is being made through the development of a road map that highlights the checkpoints and blockages that currently exist in the gene medicine developmental pathway for rare diseases so that we can fast-track clinical research programmes while ensuring the first principle of safety.
If we invest in research and improve the means for clinical trials, we will meet the ambition that the First Minister set out when he stated in June 2008:
“We are determined that standards of care and life expectancy match those in other countries.”
The debate is primarily concerned with the breakthrough in research that is giving hope to people with Duchenne and their families, but I will end as I began, by putting the debate into its proper context. My constituent Mark Chapman, who spoke eloquently when lobbying Parliament earlier this year, said:
“As adults we need support with social inclusion, we need access to education and employment, we need equipment such as cough assists and ventilators at point of need, we need access to new drugs and treatment. We have a right to live as best we can, be that independent living or not. We have the right to relationships, sex and our own families should we choose. We demand our right to live life. Dealing with Duchenne should be a big enough fight on its own.”
By supporting the research, the Scottish Government can leverage the value of every Scottish pound that is spent by matching it with research funding from elsewhere in the UK. If we do that, we will be meeting our moral and practical obligations to a group of exceptional and inspirational individuals and their families by finding a cure for this horrendous disease.
17:16
I will be the first to congratulate Jim Eadie on securing the debate. I acknowledge the work of Action Duchenne in supporting boys and men with Duchenne muscular dystrophy. I am proud to be the convener of the cross-party group in the Scottish Parliament on muscular dystrophy. We benefit from the experience of people such as John Miller, who as we know is the Scottish advocacy officer for Action Duchenne and who never stops talking about Lee. I associate myself with Jim Eadie’s remarks about John’s work and the work of his family.
Working with the Muscular Dystrophy Campaign, the cross-party group has spent much of its time focused on what we can do to improve the experience of people with muscular dystrophy. Inevitably, a lot of that has been about services. We have focused on the development of specialist multidisciplinary care, better transition services, better access to physiotherapy, the need for specialist muscle care advisers and the need for health and social care to be better joined up. We produced “The Mackie Report: Access to specialist neuromuscular care and social care in Scotland”, which clearly set out what could be achieved, based on evidence from health and social care professionals and, more important, from those with muscular dystrophy and their families.
The Scottish Government’s response was helpful, although there is a continuing problem with the employment of specialist muscle care advisers. The minister will recall that he gave a hugely welcome commitment to provide three full-time care adviser posts in this financial year. My understanding is that, currently, two advisers are in place, but that someone has yet to be employed for the north of Scotland. There might have been recruitment difficulties, but the minister will know just how important the advisers are to those with muscular dystrophy, as they provide essential care and support. If the minister has time, it would be helpful if he could tell us what action is being taken to ensure that the appointment is made as quickly as possible and that the long-term future of the posts is secure.
I return to Duchenne specifically. The thought that the groundbreaking research that Jim Eadie has highlighted might lead to the production of genetic medicine that will improve the treatment and condition of those with Duchenne’s is truly fantastic. I do not pretend to understand what exon skipping is all about or what AAV-based medicine actually does, but what matters is that it will make a huge difference. The lesson for us all is to ensure that we continue to invest in research and to consider the impact of services and how to improve services for those with muscular dystrophy. Like Jim Eadie, I know that Action Duchenne will do just that, as will the Muscular Dystrophy Campaign, which also has an extensive research programme.
I join Jim Eadie in paying tribute to the campaigners, fundraisers and researchers because, without their efforts over the years, many things would have been so much more difficult to achieve. He is absolutely right that we should contribute to international research and participate in global clinical trials. Co-funding of such initiatives working in partnership with others will, undoubtedly, lead to quicker and more significant progress. I, too, recommend that to the Scottish Government.
Jim Eadie and I appear to be mentioning the same people tonight—we are at one on the matter. I recommend that those who have not yet seen Mark Chapman’s film watch it. It is called “A Life Worth Living: Pushing the Limits of Duchenne”. I was not able to make the screening in the Parliament in June—indeed, that might even have been its premiere—but I have seen the film since then, and I recommend it to colleagues.
Mark Chapman is not defined by his disability or his condition. Rather, he is a shining example of what it is possible to do and of the richness of life. Therefore, I am very pleased to participate in the debate and, once again, congratulate Jim Eadie on bringing it to the chamber.
17:20
I confess that my knowledge of Duchenne muscular dystrophy was, until recently, rather sketchy. I suspect that such ignorance of that awful disease is widespread. Given the fact that, as the motion notes,
“one in every 3,500 male births in the UK”
is affected by DMD, that is a regrettable state of affairs.
Awareness of the condition and its devastating consequences for the victims and their families has increased markedly over the past few months in the major population centre in the constituency that I represent, for the most unfortunate of reasons. In Arbroath, we have in our midst one of the most heartbreaking examples of the devastating impact of DMD. The thought of seeing a child or loved one suffer and then die from a degenerative genetic condition is simply too painful for most of us to imagine. However, it has been a reality for Norman and Yvonne Mathieson, along with their daughter Claire, not once but twice, and the pain for the family goes on.
In little more than two years, the Mathiesons have lost two of their three sons to the condition. The remaining son, Daniel—their firstborn—is wheelchair-bound and they recognise that he is living on borrowed time. Daniel and his family have lived with his life-shortening condition for 20 years, and the family has spent the past few months fundraising to grant him his last wish. In 2007, Norman and Yvonne took the entire family to Florida; Daniel wants to return there one more time. The family set about trying to raise the £10,000 that it would take to meet the cost of a 29-day trip to the USA.
I talked to Mr Mathieson yesterday in preparation for the debate, and it emerged that the sum involved has successfully been raised in the space of only 11 weeks. In four weeks’ time, the family will head to the sunshine state. I am sure that all members wish the trip to be everything that Daniel and his family would hope for.
Although I am sure that making preparations for the Florida holiday will be uppermost in the family’s minds, I am equally sure that they will take an interest in the debate, as they have been actively campaigning to raise awareness of DMD for some time. Back in 2009, they were part of a lobby of Parliament for that purpose, and they tell me that they will return to the Parliament next year to pursue that agenda.
I hope that the staging of the debate—I congratulate my colleague Jim Eadie on securing it—will add momentum to the efforts that are being made not only to raise awareness of DMD but, more important, to develop some form of effective response to the disease.
The campaigning group Action Duchenne argues that a centre of excellence specific to the disease should be created in Scotland. I understand that there are only 150 identified sufferers north of the border and I am not sure whether it would be viable or practical to have such an arrangement with that number. It strikes me that, for geographical reasons and because of the travel challenges that such a set-up might pose some patients and their families, the best solution might be to ensure that the national health service seeks to deliver a quality service at a more localised level.
We must, of course, acknowledge that there are other forms of muscular dystrophy. Any commitment of resources in the direction of a centre of excellence would surely have to take account of that. However, such judgments can be made only on the advice of experts in the field. Although we are less expert, in this debate, we can acknowledge the advances that are being made in genetic medicine and commit ourselves to supporting them.
It seems that we are still nowhere near finding a cure for the disease but, as Jim Eadie highlighted, experimental treatments are being trialled and show promise. The sad reality is that whatever progress is made in that area will come too late for Daniel Mathieson. However, it seems that there is some hope on the horizon for other, much younger DMD sufferers.
I ask the minister to offer us the Government’s perspective on the advances that are being made in search of a treatment and to indicate how we might contribute to them, as well as outlining how services in Scotland for people with DMD may develop.
17:24
Like others, I welcome the debate and congratulate Jim Eadie on bringing this important issue to the chamber. Chronic neuromuscular conditions often feature as a subject matter for members’ business debates, but this evening’s debate is one of the few to highlight a condition that strikes in childhood and which predominantly affects young boys.
All such conditions have a common thread—they are all relatively rare in population terms, and they tend to be the Cinderellas of the NHS, whose focus is inevitably on the more common diseases that affect the bulk of the population.
The needs of the groups of people who suffer from such diseases are similar. Many of the diseases are progressive, most are currently incurable, and top-class research is vital if the underlying causes are to be found and treatments and potential cures developed. It is very exciting that such research is now producing results.
The motion sends out a message of hope of a very real health improvement to people who suffer from Duchenne muscular dystrophy, with current research likely to result in the development in the next few years of a series of genetic medicines that have the potential to benefit a proportion of patients and, I hope, with the possibility of more to come in the not-too-distant future.
Such genetic medicines were undreamt of until fairly recently, and they are a significant step forward in the search for an effective cure for such a debilitating condition. I fully support Jim Eadie’s plea for Scotland to be part of collaborative clinical trials.
In the meantime, DMD sufferers deserve the best possible care and support and state-of-the-art equipment to help them cope with their disabilities. Advice from specialist nurses and physiotherapists, adaptations to housing on the recommendation of occupational therapists and, in the case of DMD sufferers who also have learning difficulties, the support of specialist teaching staff can all make a tremendous difference to quality of life and can enhance life expectancy.
In preparing for the debate, I read once again the muscular dystrophy campaign’s daily living factsheet, produced a few years ago, which made it clear that boys with DMD do best when they are educated in school and that, with careful planning, it is possible to ensure that a child’s time at school can enhance his range of experiences and quality of life, and can provide opportunities to enjoy a wide variety of activities and develop friendships. Without that, life for those children would be restricted and isolated.
Many boys with DMD have done very well at school, passing exams and attaining university degrees. Some move on to jobs and many gain enormous pleasure from swimming, reading, painting, playing musical instruments or operating computers. Such achievements are possible only if their talents and abilities are recognised and supported from an early age but, unfortunately, Scotland has been some way behind other countries in that respect. As a result, the choice of school—mainstream or special needs, day or boarding school—is important. The fact that pupils with DMD have different needs should be taken into account when their education is being planned, although it is reckoned that, with the right planning, most boys can be educated in a local mainstream school.
Planning is also essential to ensure that, as a condition progresses, the necessary facilities, equipment and support are readily available. Regular reviews and assessments are therefore required and, as the children grow, there should be early planning for transition between school and college or university. Moreover, children should not just become the passive recipients of care; they must be allowed to develop independence of thought and given the freedom to make choices.
Social integration is another essential ingredient of a happy, fulfilled life, and ensuring that that takes place will prove to be easier the earlier a child can be integrated into school. They certainly deserve no less.
I recall a debate that was initiated into this matter a few years ago by a former colleague, Sylvia Jackson MSP. At the time, I raised the issue that, by the age of 12, most boys with DMD need to use a powered wheelchair at least some of the time. I ask the minister what progress has been made on wheelchair supply in Scotland and, in particular, in our rural areas, such as Aberdeenshire, which I represent. I hope that the minister will give a commitment in the interests not only of the DMD sufferers for whom I am principally speaking but of the many people with chronic neuromuscular conditions who might need appropriate modern wheelchairs to achieve mobility.
This debate must give real hope for the many people afflicted with DMD, and I look forward to hearing about the future developments in the care and treatment of men and boys who are diagnosed with the condition. Once again, I thank Jim Eadie for sponsoring the debate. I support the motion.
17:29
I, too, thank Jim Eadie for bringing this debate to the chamber and giving us the opportunity to discuss this important condition.
Seventy families in Scotland are registered with Action Duchenne, but that does not mean that each family has only one son with Duchenne muscular dystrophy. We know what can cause DMD and we know the symptoms of the disease, but not enough is known about its treatment and cure.
Small steps are being taken, and it is encouraging that new gene therapy techniques such as exon skipping could be on the market in the UK by 2014. However, that is likely to help only 14 per cent of DMD sufferers. We need to think about different models of care while a cure is found.
Denmark has the same population size and birth incidence of DMD as Scotland, but the difference in the life expectancy of those with DMD is striking. Denmark has twice the number of adult DMD survivors—79 compared with 39 in Scotland. In Scotland, adult patients can live until their early 30s; in Denmark, they can live into their late 30s.
There is no difference between Denmark and Scotland in the treatment that is delivered or the drugs that are available. The difference appears to be down to levels of care. I hope that the Social Care (Self-directed Support) (Scotland) Bill will help us to make improvements in that regard.
In Denmark, all DMD patients have round-the-clock care, and their houses are adapted to make them suitable. If the house cannot be adapted, the family is moved to a new home.
It would appear that Scotland is lacking in expert care provision for DMD patients. The UK has two centres of excellence in treating DMD: one in Great Ormond Street hospital in London and one in Newcastle. According to Action Duchenne, in the past 18 months it has been found that life expectancy for DMD patients near Newcastle is much better than life expectancy for those in Scotland. DMD patients can experience respiratory problems, motor problems and, in some cases, scoliosis. Those who live near the centres of excellence have the advantage of access to a core team of experts who are on hand to help them.
It is not only in times of crisis that the centre helps. The teams carry out routine check-ups of DMD patients in the surrounding area and monitor their condition. It is worrying that Scottish patients do not have access to the same expertise in their own areas. In Scotland, patients have to wait to weeks and sometimes months to be individually assessed by different doctors.
In Northern Ireland, the recent McCollum report, which was based on NHS data, found that emergency unplanned admissions to hospital of neuromuscular patients, including those with DMD, cost £2.27 million a year. The report states that 40 per cent of that cost could be saved with better monitoring of the condition. Investing in and joining up neuromuscular services would lead to a large overall saving, while also helping those with DMD to live longer and to have a better quality of life.
I feel that it would be to the benefit of all DMD patients if we had a centre of excellence in Scotland—a place that could offer expert care, advice and information.
Once DMD is diagnosed, a comprehensive care plan can be created for the child that details everything from the early years up to the teenage years. It can also be used to liaise with the local authority in order to give maximum support. Even then, however, there is a postcode lottery when it comes to local authority services. Stirling Council provides free hydrotherapy sessions for DMD patients, whereas patients in the Renfrewshire Council area either have to pay or receive their hydrotherapy through private medical care.
Scotland has gaps in its care for DMD patients. Small but important initiatives such as the free provision of hydrotherapy can extend life expectancy, and important research is being carried out to find a cure for DMD. Until a cure is found, however, we need a care model that is structured around the needs of sufferers. That will give patients a better life expectancy and a fighting chance.
17:34
I congratulate Jim Eadie on bringing to the chamber this important and interesting motion, which has a hopeful and encouraging message at its heart.
When confronted with this tragic genetic disease, our first thoughts are for the boys who are affected and their families. I first became aware of the condition when I was approached by my constituent, Eileen McCallum, who some members might know as a well-known actress. She has two grandsons who have Duchenne, and she has campaigned tirelessly for better care and support as well as for funding for research.
As part of that campaign, Eileen went to Denmark to make a film about the superb care that young people receive in that country. We should certainly learn from that—Mary Fee has already referred to that in her speech. She also mentioned the fact that boys in Denmark who are affected live longer into adulthood, and I am sure that we can learn many lessons by looking outwith Scotland.
Having said that, I believe that we should also acknowledge the developments in care and treatment that we have seen over the past few years in Scotland, particularly through the Scottish muscle network, which is one of a large number of managed clinical networks that have greatly improved the standard of treatment and care in Scotland over the past 10 years.
The focus of this evening’s debate is on recent developments in gene therapy. I have been aware of gene therapy as an exciting area of medicine for some time, particularly in relation to cancer, but I was not aware of the specific developments in relation to Duchenne muscular dystrophy until I read Jim Eadie’s motion and looked into the background.
The motion highlights the use of the adeno-associated viral vector for gene therapy. The aim is to mask the deleted exon or exons that are a feature of muscular dystrophy to facilitate production of the dystrophin protein. It is really encouraging to read in the motion and some of the background papers that gene therapy is already producing some exciting developments and results. That is why there is such a message of hope in Jim Eadie’s motion.
Jim Eadie also made some positive and helpful suggestions about carrying the work forward, and I hope that the minister will respond to them in the winding up speech. We need to pay tribute to the super work of Action Duchenne in the context of the debate. It has been involved in partnership funding on research and, as Jim Eadie has suggested, the Scottish Government should pursue that model.
The largest sums of money for medical research clearly sit with the UK Government and funding councils, but the chief scientist office has a significant budget for research and its current chief scientist, the brilliant Professor Andrew Morris, has a particular interest in the genetics of medicine. I hope that the Scottish Government will respond positively to Jim Eadie’s call for the involvement of the Scottish Government and the chief scientist in partnership funding for research.
The other aspect that Jim Eadie referred to was the importance of clinical trials. Again, the motion refers to some new developments that give us encouragement. I hope that the Scottish Government will do all that it can to ensure that those who are affected in Scotland are involved in clinical trials.
Once again, I pay tribute to Jim Eadie for bringing the motion to Parliament, and to Action Duchenne and the many people who work tirelessly for that organisation. That includes John Miller, who is in the public gallery and who prompted me to speak in the debate, for which I thank him.
17:38
Like other members, I begin by congratulating Jim Eadie on securing the time for such an important debate and on the way in which he has raised the issues that he wanted to highlight in the debate and his motion.
I have no doubt that all members who are in the chamber share the same ambition: that a cure for Duchenne muscular dystrophy will be developed. DMD is a devastating disease that, at any one time, affects around 150 boys and young men in Scotland. Jim Eadie set out the impact that it can have on individuals, and Graeme Dey highlighted the challenges and difficulties that the Mathieson family faced in losing their son James while their son Daniel has the same condition. I wish Daniel well for his trip to Florida in the next few weeks.
However, these are also exciting times in the search for a treatment for Duchenne muscular dystrophy. As Jim Eadie highlighted, it is around 25 years since the genetic variation in the dystrophin gene was identified as being the cause of DMD. Advances in medicine mean that experimental treatments are now being trialled in ways that were unimaginable at that time. For example, DNA patches have been designed to override the genetic variation; they have shown promise in laboratory trials and are currently being tested in clinical trials. They are part of the exon skipping approach to which Jim Eadie referred.
Jim Eadie’s motion refers to the AAV-U7 clinical trial that is being conducted by Royal Holloway College, University of London. The basis of the trial is that the DNA patches are packaged into a virus that allows them to be effectively delivered into the patient’s cells. That involves very exciting technology that is at the forefront of research into an effective treatment.
It is only right that we record our thanks to those with DMD who are participating in the present trials. Those trials are an essential step in developing a safe and effective treatment, and we must go through a process of evaluating the effectiveness of that type of treatment. It is through the dedication and selflessness of those patients and their families that we will eventually arrive at the point at which—I believe—we will achieve more effective treatment.
A particular challenge in the treatment of DMD is how to protect and restore the function of the heart muscle. I am aware that Dr Keith Foster, who is one of the leaders of the AAV-U7 trial, is developing a research project on that subject with a group of Glasgow cardiologists.
I reassure members that the Scottish Government’s chief scientist office is happy to consider co-funding arrangements with leading UK medical charities in particular areas of research. I also reassure members that a large amount of the research that takes place in Scotland involves international collaboration.
I can confirm that officials from the chief scientist office have spoken to Action Duchenne with a view to co-funding such a project. Action Duchenne provides an example of the importance of the charitable sector in the area of medical research. Its role in partnership with the Muscular Dystrophy Campaign and the Duchenne Family Support Group in establishing the MDEX consortium to develop treatment for DMD is particularly worthy of recognition.
Action Duchenne funds cutting-edge research and has established a registry of DMD patients—to which some members have referred—that plays an essential role in identifying those who are most suitable for particular clinical trials that may be available or are being considered at any given time.
The motion calls for simplified governance for trials of genetic medicines. Ethical oversight of proposals to conduct clinical trials that involve gene therapy is currently a reserved matter. However, the Scottish Government notes that operational responsibility for gene therapy in the form of the gene therapy advisory committee was transferred to the NHS Health Research Authority in December last year, and we are monitoring the effectiveness of that new arrangement.
Clinical trials need to obtain NHS research and development approval. I assure members that we have recently streamlined that process so that it is co-ordinated centrally in Scotland. The streamlined approach means that approval times for multicentre clinical trials in Scotland are among the quickest in Europe, which makes Scotland an attractive place in which to conduct world-leading clinical research.
I assure members that we recognise the importance of ensuring that people with Duchenne muscular dystrophy get access to the care and support that they need.
I highlight the important role of the Scottish muscle network, which Malcolm Chisholm mentioned, in strengthening the specialist neuromuscular centres in Scotland, through a collaborative approach. The network brings together clinicians in various disciplines that contribute to the care of those with muscular dystrophy. It provides on a Scotland-wide basis the multidisciplinary focus that Action Duchenne is looking for, and it gives families a strong voice in the development of services.
I heard what Mary Fee said about the creation of a centre of excellence. In Scotland, our population is dispersed over a much greater area than is the case in England, where populations are much more concentrated. That makes it challenging to provide a single centre of excellence in the way that has been done in England. We regard the muscle network as Scotland’s virtual centre of excellence, because it links the five paediatric centres in Scotland that deliver specialist services for those who have any form of muscular dystrophy or neuromuscular disorder. I am sure that members appreciate that it would be difficult to set up a single centre in Scotland, given the impact that that could have on families, who might have to travel a considerable distance to access such a centre.
The Scottish muscle network has developed multidisciplinary care standards for the management of Duchenne that are based on the care standards of the international network of translational research in Europe: assessment and treatment of neuromuscular diseases, which is known as TREAT-NMD. The implementation of the standards will be key to improving healthcare services for people with Duchenne muscular dystrophy.
We have agreed two years’ funding to pilot neuromuscular care advisers posts in the south-east and Tayside, the west of Scotland and the north of Scotland. Two posts are already in place, and we remain committed to posts in all three regions. Jackie Baillie asked about the post in the north of Scotland regional planning area. I advise her that there have been recruitment difficulties, but the north of Scotland has confirmed that it intends to re-advertise the post. We will monitor the situation closely, because we want the care adviser post in the north of Scotland to be filled as soon as possible.
Many people with DMD will benefit from the £16 million that we have invested in the wheelchair and seating service, to support the timely provision of wheelchairs in a personalised way, responding to individuals’ needs at an early stage. Nanette Milne expressed concern about the issue. We recently issued a wheelchair and seating services quality improvement framework, to which standards are attached. That approach will bring further improvements for people with neuromuscular conditions.
We have also put £1.5 million towards improving services for people with complex respiratory conditions. That investment will help to recruit new specialists, whether they are consultants, dieticians, physiotherapists, nurses or allied health professionals, so that people can be treated more effectively locally.
Progress has been made, but it is clear that we need to continue to build on that progress to improve services. We remain committed to doing that. I look forward to working with members and the charities and organisations that seek further improvement in Scotland, to ensure that people receive the best quality of service that they can receive in Scotland.
Meeting closed at 17:49.