A major research project getting underway at the Royal Free could bring a breakthrough in the hunt for a new treatment which could better slow or stop the progression of Parkinson’s disease.

The research is a ‘proof of principle’ study which will test a drug and investigate the hypothesis that Parkinson’s symptoms could be treated by reversing the impact of a mutation of the GBA gene. It’s already well known that the mutation causes Gaucher’s disease, but researchers now know it also increases the risk of a person developing Parkinson’s by 20 to 30 times.

It’s believed that the mutation leads to an accumulation of protein which promotes the degeneration of nerve cells in the brain that produce dopamine, a cause of many Parkinson’s symptoms. It’s thought that the drug, which has already been safely tested in humans for another condition, may increase activity associated with the GBA gene and therefore reduce the protein accumulation.
As many as 10% of people with Parkinson’s in the UK have the gene mutation, but the research will also test the hypothesis that the drug may also help Parkinson’s patients who don’t fall into this group.

The work has been made possible by a £660,000 award given to an international team of researchers by the Centres of Excellence in Neurodegeneration (CoEN) initiative, launched in 2010 by the Medical Research Council, and builds on a similar grant awarded in 2011.

Professor Anthony Schapira, professor of clinical neurosciences at the Royal Free, is principal investigator for the international research collaboration, which includes representatives from the universities of Toronto and Pavia, Italy as well as Deutsche Zentrum fur Neurodegenerative Erkrankungen, a German research institute.

Professor Schapira, who is also vice dean of University College London (UCL) Medical School and director of UCL’s Royal Free Campus said: “I’m delighted that our international team has been given a grant from such a prestigious funding body, which will allow this exciting research to be carried out at the Royal Free. 

“To me, it’s a clear indication of the excellent reputation our research has around the world – and indeed the team has recently been awarded a separate grant from the Michael J. Fox Foundation in the US. I think both of these grants are in part due the Royal Free and UCL both being members of UCLPartners, one of the world’s leading centres of medical discovery and healthcare innovation.

“This approach is unique in that it is initially designed to target a specific sub-group of Parkinson’s patients with certain genes. Its applicability to the wider Parkinson’s community will also represent the first treatment designed based on a particular biochemical defect thought to be responsible for Parkinson’s.” 

It was possible for researchers to develop their hypothesis because of a close collaboration with two other Royal Free clinicians, Professor Atul Mehta and Dr Derralyn Hughes, who run the lysosomal storage disorders unit which sits within the new UCL Institute of Immunity and Transplantation at the Royal Free.

Gaucher’s disease patients being treated in the unit provided samples used by the researchers, in an early sign that investment in the institute and the close links it has established between researchers and patients is paying off.

Professor Schapira concluded: “We hope that over the course of the next 12-18 months our hypothesis will be proven and we will be able to move onto human trials, taking us a step closer to a better drug for Parkinson’s and a light at the end of the tunnel for the millions of people worldwide affected by the disease.”


Notes to editors

Please contact rf.mediaenquiries@nhs.net.

Interviews and photographs are available with Professor Anthony Schapira on request.

Parkinson's is a progressive neurological condition. People with Parkinson's don't have enough dopamine because some nerve cells in their brain have died. The loss of nerve cells in the brain causes the symptoms of Parkinson's to appear as without dopamine people can find that their movements become slower so it takes longer to do things. The main symptoms of Parkinson's are tremor, rigidity and slowness of movement.

There's currently no cure for Parkinson's and experts don’t yet know why people get the condition. However drugs and treatments are available to manage the symptoms.

One person in every 500 has Parkinson's, or about 127,000 people in the UK currently. Most people who get Parkinson's are aged 50 or over but younger people can get it too – one in 20 people with the condition is under the age of 40.

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UCL and the Royal Free are members of UCLPartners, a leading academic health science partnership that supports the healthcare system serving over six million people. Together, its member organisations from higher education and the NHS form one of the world’s leading centres of medical discovery, healthcare innovation and education.  They have come together through UCLPartners to improve health outcomes and create wealth for the UK at scale and pace. Designated in 2009, UCLPartners’ centre of discovery is one of five accredited Academic Health Science Centres in the UK. Its Academic Health Science Network is one of the largest in the country, covering parts of London, Hertfordshire, Bedfordshire and Essex.