Submission from the Muscular Dystrophy
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
wombthey 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
2. NEW TREATMENTS
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
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. MUSCULAR DYSTROPHIES
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. SHORTAGE OF
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
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 wombeither human or animalbut 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. THE MUSCULAR
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
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.
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2 Pickrell J "Instant Expert: Stem
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3 "Royal Society response to HFEA
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Science News. (11 January 2007) http://www.royalsoc.ac.uk/news.asp?year=&id=5792.
4 Petit-Zeman S. "AMRC response
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Int J Hematol. (2004) 79:79-84.
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Barthelemy I, Perani L, Mantero S, Guttinger M, Pansarasa O, Rinaldi
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10 "`Donor eggs for science' debated".
BBC News at bbc.co.uk (Thursday, 7 September 2006) http://news.bbc.co.uk/1/hi/health/5323894.stm
11 "Cloning team's IVF deal for
eggs". BBC News at bbc.co.uk (Thursday, 27 July 2006)
12 Human Fertilisation and Embryology Authority.