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Initial positive results indicate that new gene therapy could revolutionise treatment for Parkinson’s disease

A story has appeared on Sky News on April 12th on Oxford BioMedica’s innovative gene-based treatment for Parkinson’s disease. The programme, which was broadcast ahead of Parkinson’s Awareness Week starting on 16 April, includes interviews with Sheila Roy, a patient who has been treated with ProSavin®, Dr Philip Buttery, a clinician at Addenbrooke’s Hospital, and Stuart Naylor, Chief Scientific Officer at Oxford BioMedica.

What is Parkinson’s disease?
After Alzheimer’s disease, Parkinson’s disease (PD) is the most common neurodegenerative disease. It is caused by the loss of brain cells that produce the chemical dopamine, a neurotransmitter which makes other parts of the brain that coordinate movement work properly. A patient with Parkinson’s disease develops stiffness, tremors and slow movement that can become worse over time. Most people who get Parkinson’s disease are aged 50 or over but younger people can get it too; one in 20 is under the age of 40. Parkinson’s disease currently affects 4.1 million patients globally which is projected to rise to 8.7 million by 2030.

What is ProSavin®?
ProSavin® is an innovative gene-based treatment for Parkinson’s disease. In Parkinson’s disease, there is degeneration of the cells in the brain that produce dopamine. Using Oxford BioMedica’s LentiVector® gene delivery technology, ProSavin® delivers the genes for three enzymes that are required for the synthesis of dopamine directly to the region of the brain called the striatum, converting cells into a replacement ‘dopamine factory’ within the brain, thus replacing the patient’s own lost source of the neurotransmitter.
ProSavin® is being evaluated in a Phase I/II trial in patients with Parkinson’s disease at two centres of excellence for neurosurgery; Addenbrookes Hospital in Cambridge, UK, with Professor Roger Barker as Principal Investigator and at the Henri Mondor Hospital in Paris with Professor Stéphane Palfi as Principal and Coordinating Investigator.

Sheila Roy was diagnosed in 1995. Commenting on her experiences with PD Sheila said: “Early in 2011 I was rapidly deteriorating. My medication was being less effective, there was increased involuntary movement, where I frequently hit myself but also other people, and had a four second switch from extreme movement to being ’OFF’ and very still. This lasted for some time, up to two hours and more and I could do nothing. These unpredictable shifts were like a ‘Jekyll and Hyde’ transition, and outside of my control. At night there was no relief as I had terrible nightmares, and often woke my husband up with screaming or punching him.”

She added: “Parkinson’s disease changes the ability and capability of the individual affected. You lose confidence, dignity and hope. The ProSavin® experience has restored my confidence, enabled better motor function and has given me hope. I can function more normally and, for the first time in 15 years, I can write.”

Cambridge Neuroscientist 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."

Who is Oxford BioMedica?
Oxford BioMedica is a biopharmaceutical company, which has established a platform of technologies in gene delivery and immunotherapy, and have a broad pipeline of gene-based product candidates for the treatment of age-related or inherited neurodegenerative and ocular diseases. In addition, Oxford BioMedica and its collaborators are developing novel targeted therapies and therapeutic vaccines to treat multiple types of cancer.

Adapted from statement released by Oxford BioMedica, April 12th 2012.

Parkinson’s awareness week will take place from April 16th-22nd. For more information, please click here. As part of Parkinson's Awareness week, Parkinson's UK will be hosting a public lecture entitled "Brain rhythms in Parkinson's: from bedside to bench and back again" at Fitzwilliam College in Cambridge. The lecture will be aimed at a lay audience but will be entertaining and interesting to all. For more information, please click here.

Posted on 13/04/2012