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Gene therapy trial and Alzheimer’s disease

Published 27 April 2004

Alzheimer’s Society comment on research by Mark Tuszynski, M.D., Ph.D. et al reported at the American Academy of Neurology meeting in San Francisco on 27 April 2004.

'Professor Tuszynski treated eight volunteers diagnosed with early-stage Alzheimer's disease in an exciting study where patients' own skin cells were genetically modified in culture to produce a growth factor that protects nerve cells and encourages regeneration.

This tissue was surgically placed directly into the brains of these individuals. In this preliminary study, the procedure appeared to be safe and resulted in increased metabolic activity in the brain, with cells apparently responding to the insertion of growth factor-producing grafts. This is one of a number of innovative treatment approaches involving grafts of growth factors, stem cells or delivering genes that can modify key processes in nerve cells.

Although many of these lines of investigation are at a relatively preliminary stage at the moment, there has been enormous development of the techniques over the last few years, and such approaches hold huge promise for the development of effective treatments. Many of these studies are being undertaken in cells cultured in the laboratory or in animals. It is therefore also very encouraging that Professor Tuszynski's study suggested that this could be a safe and viable treatment approach for people with Alzheimer's disease.'

Professor Clive Ballard
Director of research