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Gene therapy shows early success in Parkinson's trial

16 April 2012
Appeared in BioNews 652

A woman with Parkinson's disease is reportedly able to write again for the first time in 15 years after receiving pioneering gene therapy at Addenbrooke's Hospital, Cambridge. Mrs Shelia Roy took part in an early stage clinical trial of ProSavin - a treatment developed by biopharmaceutical company, Oxford BioMedica.

Roy was diagnosed with Parkinson's 17 years ago in her mid 40s. During the trial, a modified virus was injected into Roy's brain containing coding instructions for dopamine-producing proteins. Patients with Parkinson's have a lack of dopamine in the brain which leads to symptoms including tremors and an inability to co-ordinate movement. This can make even everyday tasks - like writing - very difficult.

Speaking of her experience, Roy said: 'Parkinson's disease changes the ability and capability of the individual affected. You lose confidence, dignity and hope. The [treatment] has given me hope'. Although Roy's experience is an encouraging result, scientists have warned that it may still be at least five years before ProSavin becomes available.

Dr Philip Buttery, from the Cambridge Centre for Brain Repair, University of Cambridge, told Sky News that while the treatment was still in its early stages the results look promising. 'It seems to be having an overall beneficial effect in smoothing out people's days, probably allowing a slight dose reduction in medication, and in some patients a better sleep pattern and a better quality of life for all', he said.

Roy took part in a small Phase I/II clinical trial with 14 other people with mid-stage Parkinson's who were no longer responding well to existing treatments. Five additional patients in the UK received a high dose of ProSavin. Although the trial was designed to test the dose needed and the safety of the treatment, when assessed these patients showed an average 29 percent improvement in motor function over a three month period. Larger studies are now needed to confirm these results.

Commenting on the results, Dr Kieran Breen, director of research and innovation at the charity Parkinson's UK, said: 'Gene therapies hold great promise for people with Parkinson's in the future, as they could mean an end to the daily regime of drugs that most people with the condition currently face'.

He added: 'In addition to ProSavin, there are three other gene therapy trials under way at the moment. So far all the therapies appear to be safe - now the challenge is to see whether they are more effective than the medications we already have for Parkinson's'.

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Parkinson's disease sufferer can write for the first time in 15 years thanks to new gene therapy
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