Stem cells
A lot of the research is looking at ways to prevent or reduce the effects of neurodegenerative conditions such as the ataxias, therefore stopping them progressing further, but there are also scientists investigating ways to repair the damage that has already been done. If you damage your skin cells for example, with a cut or scrape, your skin can repair itself and return to how it was before it received the injury, but in conditions involving the nervous system this is impossible because nerve cells cannot regenerate.
At present, stem cells offer the most promise for being able to repair this type of permanent damage.
What are stem cells?
Stem cells are unspecialised cells that have not yet differentiated into any specific type. They are unique because they can reproduce themselves infinitely. They can also generate more specialised cell types, such as muscle, nerve or bone cells. They are found in a number of the body’s tissues and organs and occur from the earliest stages of development (embryonic stem cells) to adulthood (adult stem cells).
Theoretically, stem cells derived from early embryos could be developed into most or all types of tissue. Those extracted from an adult may have more limitations, but may be easier to obtain. It is important to ensure that research in all types of stem cells is allowed in order to maximise chances of finding treatments.
How could they be useful?
The ability to create new body tissue means stem cells have a potential future use to generate cells to replace diseased and damaged body tissues. These tissues could treat patients whose tissue is diseased or damaged through injury.
Currently, clinical work with stem cells is at a very early stage. While possible benefits have been highly publicised in the press, there have been no clinical trials proving the effectiveness of stem cell therapy for Friedreich’s ataxia or any of the other ataxias. Nor have any of the potential risks been fully debated.
Click here[link to statement] to see Ataxia UK’s position statement on stem cell therapy
Current Research
Research on cells in the laboratory has looked at the possibility of using stem cells in other neurodegenerative conditions such as Parkinson’s and Huntington’s diseases (Freed et al 2001; Dunnett et al 2006), but there are many questions still to be answered before it will be known if they are a safe and effective treatment for human patients. For example: can they be delivered to the precise place they are needed to repair damage, and can their regeneration be in a controlled manner? How long will the effects last and will the new cells eventually succumb to the same disorder as the patient’s original cells? What is the likelihood of dangerous complications such as tumour development or rejection of the new cells?
Ataxia UK is currently funding a one-year project looking at the potential of stem cells to protect nerve cells from degeneration when they are exposed to damage similar to that seen in cerebellar ataxia. Dr Wilkins and Professor Scolding at the University of Bristol will be using cerebellar cells taken from rodents and subjecting them to toxins which cause damage to test the effect of stem cells on their survival. If this avenue of research appears useful, it will subsequently lead to testing the effect of stem cells in more complicated models (such as in whole animals which have been modified to be ataxic) and then eventually it may lead to human trials.
See here for more information on this research.
References
Freed CR et al. N Engl J Med 2001; 344:710-9.
Rosser A, Dunnett S. The Lancet Neurology 2006; 5 (4): 284-5.
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