Select Committee on Science and Technology Written Evidence

Memorandum 16

Submission from the Muscular Dystrophy Campaign


  1.1  The Muscular Dystrophy Campaign (MDC) believes that stem cell research may lead in time to new treatments and effective cures for muscle disease and neuromuscular conditions in addition to other serious illnesses and diseases. However, there is a shortage of donor eggs for research purposes which slows and delays the vital research effort.

  1.2  The MDC believes that the existing legislation should be extended to allow this problem to be addressed through the use of chimeras which use the existing technique (already licensed by the HFEA) of cell nuclear replacement. These embryos would not in any circumstances be allowed to develop beyond 14 days, of course, and would never be implanted in a human or animal womb—they are simply a source of cells for research. This research should be tightly regulated by the HFEA. We also believe that there is a need for greater public understanding of the issues involved.


  2.1  Stem cell therapy offers real hope for the hundreds of thousands of individuals who suffer from chronic or disabling conditions such as the muscular dystrophies, diabetes, osteoarthritis, Alzheimer's disease, Parkinson's disease and Multiple Sclerosis. Since stem cells harbour the possibility to become one of many different types of cell, there is the potential to use them to replace damaged or diseased tissues, for example restoring damaged muscle in Duchenne muscular dystrophy1 potentially prolonging ambulation and increasing quality of life. They may also provide an invaluable research tool for the study of specific populations of cells in both health and disease, for instance the study of nerve cells and neurodegeneration which currently relies heavily on the use of animals which are not always the most appropriate model.

  2.2  In addition to treating debilitating conditions, stem cells could be used as a source of pure populations of human cells to be used in drug testing. The use of animals for the toxicity testing of new drugs does not always highlight potential problems so using these populations of cells, high throughput drug screens could be designed to screen drug effects in specific human cell types especially those that may be vulnerable to toxicity or those that form the drug's target tissue.

  2.3  Stem cells can be found in some, but not all, tissues in adult humans (Frequently asked Questions, International Society for Stem Cell Research) and although research in this area has been promising there is still a need to explore other areas such as the use of embryonic stem cells since these cells are more versatile than adult stem cells2. At the current time, there is no evidence to show that one area of stem cell research will be more successful than another and at this early stage it would seem sensible to keep all avenues open until more is known, a view that is shared by a number of interested organisations such as the Royal Society3, Association of Medical Research Charities4 and the Medical Research Council5.

  2.4  Adult stem cell therapy, in the form of bone marrow transplantation, is already being used in the treatment of some types of leukaemia6 and, while not yet a risk-free procedure, for some patients it can lead to prolonged disease-free survival7. The types of conditions that stem cell therapy could potentially treat directly affect many hundreds of thousands of individuals in the UK, not only those with the disease but also their carers and families who have to live with the effects of these disabling and life threatening conditions.


  3.1  Duchenne muscular dystrophy is a devastating and life limiting condition affecting around 1 in every 3,500 boys born in the UK. It is caused by mutations in the gene for dystrophin, a protein with a vital role in the maintenance of muscle membrane integrity. Boys with Duchenne are not expected to live beyond their mid-twenties and are generally expected to be in a wheelchair by the age of 138. There is no cure for this condition and in recent years researchers have looked to stem cell research as a means by which a treatment to this disease may be sought.

  3.2  Recent research by an Italian group lead by Giulio Cossu 9 has fuelled the debate over whether stem cells could be used to treat Duchenne and other muscular dystrophies. Golden retrievers with a mutation in the dystrophin gene that causes a disease similar to Duchenne muscular dystrophy were injected with a particular type of stem cell, called a mesoangioblast. Although these were preliminary results, they clearly showed a potential for the treatment of Duchenne muscular dystrophy as a number of the dogs given donor stem cells, from healthy dogs, showed an improvement in muscle function. Despite the fact this particular research was carried out using so-called adult stem cells it still goes some way to demonstrating the value of stem cell research and the need for further investigation to fully explore the potential of using the chimera embryos as a source of stem cells.


  4.1  Although embryos are a source of stem cells that can be used for research and potentially the development of therapies, there is currently a shortage of donor eggs that can be used for research purposes and often researchers must rely upon using eggs that have been deemed unsuitable for IVF10. Researchers at the Newcastle Centre for Life investigating stem cells as a therapy for a number of conditions have been given permission by the HFEA to ask women having IVF to donate any "spare" eggs if they produce 12 or more during their treatment. The licence will also allow the scientists to offer couples the chance of having their IVF part-funded in exchange for some of their eggs. However, this approach still does not meet the demand for eggs by the research community11. Women who are not undergoing IVF can also donate eggs for research but there has been some concern over the risk of developing a rare but potentially lethal condition called ovarian hyperstimulation syndrome caused by the drugs that must be taken in order to stimulate the production of eggs for harvest.


  5.1  The generation and use of chimera embryos overcomes these issues since it negates the requirement for human eggs to be used in this type of research. Animal eggs, from cows and rabbits for instance, are available in much greater numbers than donor eggs from humans. The human genetic material is also very easily obtainable from cells such as skin fibroblasts, which can be obtained via a skin biopsy, a procedure that is less invasive and potentially far less harmful than the harvesting of eggs.


  6.1  Given the potential benefit to the many people affected by muscular dystrophy, we would support, with careful regulation by the HFEA, the use of chimera embryos for the generation of stem cells and would encourage dialogue with scientists who can fully explain the potential this technology holds. These are not human embryos that will be implanted into a womb—either human or animal—but are artificially created chimeras that must be activated to divide as an embryo would. In addition they would only be studied for a limited amount of time, up to 14 days, in the laboratory and would not be allowed to develop beyond this point. The technique that would be used to create the chimera embryos, cell nuclear replacement, is already known to the HFEA since licences have been granted to researchers to use this technique using human eggs and genetic material12.

  6.2  The Muscular Dystrophy Campaign strongly believes that the existing legislation should be clarified, to ensure that it includes the generation of chimera embryos implying that this research is subject to the same strict regulation as research using human embryos. We would also encourage the education of the general public about the issues surrounding the use of chimera embryos and stem cells, giving clear explanations of why these techniques could prove vital in the fight against certain diseases, allowing a more informed debate to take place.

  6.3  While it is currently unclear as to whether embryonic stem cells will in time yield the "Holy Grail" of a cure for chronic and disabling conditions such as the muscular dystrophies it would be an extremely disappointing move to ban the use of chimera embryos without proper and thorough investigation into their potential. We would urge, therefore, that very careful consideration is given to the people and families affected by these devastating conditions and the potential wealth of knowledge that could be gained from research using stem cells derived from chimera embryos.


  7.1  The MDC covers more than 60 different neuromuscular conditions and was founded in 1959 (as the Muscular Dystrophy Group). These conditions are genetic in origin and there are currently no known cures for them. The MDC invests more than £2 million a year in research projects in the UK and campaigns for improved care and support for the more than 30,000 people directly affected by muscular dystrophies and related neuromuscular conditions.

  7.2  The MDC provides care and emotional support for people living with the conditions, their families and carers and we also produce high quality information and advice. We have more than 4,500 members and a national network of more than 80 local branches across the UK.

  7.3  The MDC campaigns with its members and supporters for increased research funding for muscle disease, improved treatments and access to high quality care and support for all people living with a neuromuscular condition.

REFERENCES  1  Davies K & Grounds M "Treating muscular dystrophy with stem cells?" Cell (2006) 127:1304-6.

  2  Pickrell J "Instant Expert: Stem Cells". News Service. (September 2006)

  3  "Royal Society response to HFEA ruling on human-animal hybrid research." Royal Society Science News. (11 January 2007)

  4  Petit-Zeman S. "AMRC response to scientists' concerns about embryo research" Association of Research Charities Press Release. (5 January 2007)

  5  "Debate over Human Fertilisation and Embryology Act reform fires up". MRC News, Views and Events (12 January 2007)

  6  "Frequently Asked Questions". International Society for Stem Cell Research.

  7  Iida H, Sao H, Kitaori K, Gotoh S, Yazaki M, Kojima S, Wakita A, Morishima Y, Kodera Y, Morishita Y "Twenty years' experience in allogeneic hematopoietic stem cell transplantation for Philadelphia chromosome-positive acute lymphoblastic leukemia in the Nagoya Blood and Marrow Transplantation Group." Int J Hematol. (2004) 79:79-84.

  8  Bushby K, Bourke J, Bullock R, Eagle M, Gibson M, Quinby J. "The multidisciplinary management of Duchenne muscular dystrophy." Current Paediatrics (2005) 15:292-300.

  9  Sampaolesi M, Blot S, D'Antona G, Granger N, Tonlorenzi R, Innocenzi A, Mognol P, Thibaud JL, Galvez BG, Barthelemy I, Perani L, Mantero S, Guttinger M, Pansarasa O, Rinaldi C, Cusella De Angelis MG, Torrente Y, Bordignon C, Bottinelli R, Cossu G. "Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs." Nature (2006) 444:574-9.

  10  "`Donor eggs for science' debated". BBC News at (Thursday, 7 September 2006)

  11  "Cloning team's IVF deal for eggs". BBC News at (Thursday, 27 July 2006)

  12  Human Fertilisation and Embryology Authority.

January 2007

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