Sheila Roy, a woman on an experimental gene therapy treatment for Parkinson’s disease, never imagined three years ago that she would be well enough to write a book.
The 66-year-old from Bedfordshire, England, is to speak today at an international conference hosted by NUI Galway.
She has experienced progressive improvements since she volunteered for the radical treatment in 2011, she said.
" It goes in stages, but I took a big step up just two weeks ago," Ms Roy told The Irish Times .
Her neurologist, Prof Roger Barker of the University of Cambridge, who will participate in the presentation, notes she has already been able to reduce her medication by 50 per cent.
“I had Parkinson’s for 17 years, and was not well at all, when I heard about this treatment,” Ms Roy said of her decision to volunteer for trials of ProSavin, a viral gene therapy manufactured by Oxford BioMedica.
Virus injection
It involves injecting a virus into the brain which induces production of the neurotransmitter dopamine.
Deficiency of nerve-controlling dopamine causes the neurodegenerative condition, which affects an estimated four million people worldwide.
Ms Roy, who was in her 40s when first diagnosed, was inquiring about deep brain stimulation treatment when she heard about the trials.
She is one of 15 people selected for the treatment in Britain and France.
“At that stage, my breathing and my vocal cords had been affected, I was getting very little sleep and my brain felt very fuzzy from the medication I was on,”she said.
“I had become socially invisible as I was unable to break into a conversation, so I felt I had nothing to lose.”
The risks associated with the treatment included the danger that her condition might worsen, but she started to feel the benefits almost immediately after her operation.
“I had a headache for a couple of days, but I began to function very quickly,” she said.
She still has involuntary movements, which tend to vary in relation to the timing of her medication, and this is one side effect, Prof Barker said.
“ProSavin doesn’t cure Parkinson’s, but does offer an opportunity to deliver or reproduce dopamine in the brain,”he said.
Ms Roy and Prof Barker will give their joint presentation at the 24th Annual Meeting of the Network of European Central Nervous System Transplantation & Restoration (NECTAR) in Galway’s Hotel Meyrick.
NECTAR is a major international gathering for researchers working on the development of cell and gene therapies for neurodegenerative diseases such including Alzheimer’s disease and Parkinson’s disease.
Prof Anders Björklund of Lund University, Sweden, is set to outline progress in a separate experimental type of gene therapy that involves infusing proteins which have a protective effect on dopaminergic neurons.
If successful, the therapy could block the progression of Parkinson’s disease, he says.
Prof Björklund is also working with Prof Barker on an EU-funded project aimed at developing a type of stem cell therapy which may be able to repair the brain.
“Some 24 years ago, when I first started coming to this conference, the brain was seen as irreparable and we were regarded as mavericks”he said.
“Now, with such international progress in stem cell research, the outlook is much better, and we think Parkinson’s may be a good first candidate to apply this technology.”
