Revolutionary New Treatment For Parkinson's
A team of Oxford scientists believe they have created a groundbreaking form of therapy that could revolutionise the way Parkinson's disease is treated.
Sky News has been given exclusive access to one of the patients involved in the gene therapy trial, who says it has been like turning back the clock 10 years.
Sheila Roy was diagnosed with Parkinson's in her forties. After 17 years with the disease she suffered from severe tremors and a lack of balance made simple tasks like writing impossible.
She says she felt trapped in a "Jekyll and Hyde" state, lost her confidence and was only getting three hours' sleep a night.
She is one of only 15 people worldwide to have had the radical new treatment, which effectively creates a medicine factory in her brain.
She told Sky News: "People would take knives off me in the kitchen because I was everywhere with the knife.
"My vocal cords would suddenly shut so I can't breathe.
"If I hit a wall of people then I can't function, I just stop, but I'm starting to see a glimmer of the person I used to be, which is exciting."
Parkinson's disease occurs when the brain gradually stops producing the nerve-controlling chemical dopamine.
Over time symptoms such as tremors, slow movement and stiffness get worse.
ProSavin, the new treatment, uses a "stripped-down" virus to transport dopamine-making genes into the brain.
It is injected into a region called the striatum that helps control movement.
Once the virus gets into the brain cells, it reprogrammes them to gradually start producing their own dopamine.
The procedure has been carried out in Cambridge and France.
Dr Philip Buttery, from the Cambridge Centre for Brain Repair, says it is early days but the treatment appears to be having positive results.
"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."
The study is at an early stage and has only been used to treat a small number of patients.
More studies, involving hundreds of patients, are likely to be needed to confirm that the treatment is safe and effective. So, even if the trials progress as well as scientists hope, it is likely to be at least five years before this becomes a routine clinical treatment.
But the principle of creating medicine factories inside patients' bodies is extremely exciting.
The side effects from current drug treatments often result from the high doses needed and their indiscriminate effects on the body. By injecting genes into particular cells - where the chemical is needed most - the doses are lower and the effects are localised.
The scientists at Oxford BioMedica are also developing gene therapy treatments for other degenerative illnesses.
Chief scientific officer Stuart Naylor told Sky: "Rather than popping lots of tablets, the idea of a single shot therapy, single shot treatment, single shot placement of a gene therapy that provides that long term therapeutic correction is something that hasn't been ever experienced before in medicine.
"I think this is a wonderful opportunity to change the face of the physician's armoury."
Parkinson's disease is the second most common neurodegenerative disease after Alzheimer's.
Most people who have it are aged 50 or over but younger people get it, too. One in 20 sufferers is under the age of 40.
Worldwide it currently affects 4.1 million patients, a number that is predicted to more than double to 8.7 million by 2030.
Sheila Roy will not need any more operations but still takes medication.
She will also have constant checks and brain scans to try and assess the amount of dopamine she is producing. Doctors will also monitor any potential side-effects.
Making sure the therapy is safe is vital for future research.
Other gene therapy trials are taking place in America but this is the only treatment in the world that aims to reproduce dopamine in the patient's brain.
Other treatments are aimed at protecting the dying cells, which gradually become unable to produce the chemical.