Stem cell research - questions & answers

We are aware that many people will have questions about the stem cell research being carried out in Italy. Here we hope we can clarify some of the issues.

Q: Will UK scientists be involved in the work?
A: This particular research has been carried out by an Italian team and a research team from the UK was not involved. The results are welcomed by the charity - they are a move in the right direction and any potential future benefits will be beneficial to every person with Duchenne, independent of where this person lives. The Muscular Dystrophy Campaign is currently supporting a number of projects that explore the possibility of using muscle stem cells as a treatment for muscular dystrophies. Two scientific teams in the UK receive an annual funding of about £140,000 a year to carry out the research and we are happy to provide further information.

Q: When will clinical trials be taking place and is there a timeline?
A: A: This is a very difficult question to answer and the charity does not want to raise expectation too soon for the thousands of people with muscular dystrophy in the UK. The research is still at a very early stage and a lot of issues need to be addressed and resolved before the research can be taken forward into a clinical trial. If the research proves to be successful, it certainly will take several years before any therapeutic benefits will be available to people with muscular dystrophies. Some newspapers reported that a clinical trial may be possible within the next two years. However, some researchers’ response was that it might well take longer than two years before this technology can be tested in humans.

Q: How can I get involved in clinical trials?
A: If this line of stem cell technology shows any potential of developing into a treatment for Duchenne boys, the first step will be to test whether it is safe. Hence the first part of a clinical study will be a safety trial, which generally involves only a very small number of people. The opportunity to participate in a clinical trial will depend on where the trial is carried out. The Muscular Dystrophy Campaign aims to keep its members informed about any new developments and we will make you aware of any new advances. Being a Member of the Muscular Dystrophy Campaign will keep you up to date with research, both independently funded projects and those funded by the charity.

Q: How will this research affect someone with Duchenne now?
A: The Italian research team has provided initial evidence that in the future it might be possible to use stem cell therapy as a treatment for Duchenne and other muscular dystrophies. But, as you are probably aware, the experiments in Italy were carried out in dogs, and a lot of the future achievements will depend on how well this technology will translate to humans. So, although the results are positive, unfortunately there will be no immediate therapeutic benefit for boys with Duchenne.

More information
For more information on this development or to become a Member, please contact the Muscular Dystrophy Campaign. Staff in the Research department or our Information Officers will be happy to help.
Call: 020 7720 8055
Email: info@muscular-dystrophy.org

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Read about the research - Duchenne stem cell research published