Study aims to develop new Parkinson's drug

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 is 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. 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 and vice dean of the UCL Medical School, 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 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.”