Hope on Parkinson’s
Could UK trial be first step to reversing the disease?
A BREAKTHROUGH British trial has provided the first glimmer of hope that Parkinson’s disease could be reversed.
For the first time, neurologists regenerated brain cells damaged by the condition, using an implant that injects drugs deep into the brain.
The researchers, led by experts at Bristol University, admitted they had failed to prove the treatment actually improved patient’s symptoms.
But scans showed they had reversed six years’ worth of damage to key parts of the brain. They believe a stronger dose of the drug, and a trial designed in a new way, could prove it will make a huge difference.
Around 145,000 Britons have Parkinson’s, which causes tremors, slow movements and muscle rigidity. More than 1million people alive today will develop it later in life. Despite years of trials, there is no cure and no way to stop it progressing.
Lead researcher Dr Alan Whone said: ‘The spatial and relative magnitude of the improvement in the brain scans is beyond anything seen previously in trials of surgically delivered growth-factor treatments for Parkinson’s. This represents compelling evidence that we may have a means to possibly reawaken and restore the dopamine brain cells that are gradually destroyed in Parkinson’s.’
The £3million trial of 41 people showed that those given GDNF – an artificial version of a protein that boosts the development and survival of brain cells – experienced a 17 per cent improvement in symptoms in nine months.
But this was not judged to be significantly better than the 12 per cent improvement in those given a placebo. Because they had aimed for a 20 per cent difference, the trial was seen to have failed.
But the scientists remain optimistic. While the average impact was modest, some patients showed a dramatic response such as being able to ride a bike again.
The scientists also said the drug regenerated cells in the whole of the area of the brain affected by Parkinson’s, reversing the damage by roughly six years. Unusually, they have already received regulatory approval to further test the drug on hundreds of people, although they need roughly £76million of funding.
MedGenesis, the small company that makes GDNF, is keen to continue with the tests but cannot fund such a huge trial.
Professor Steven Gill, a neuroscientist at Southmead Hospital in Bristol who designed the device that delivers the drug, said: ‘This work urgently needs funding.’
His ‘convection enhanced delivery’ implant lets doctors inject drugs through a port in the side of the head, down a tube directly into the key part of the brain, bypassing the blood-brain barrier which usually keeps drugs out.
The initial trial, the results of which are published in the Brain journal and the Journal of Parkinson’s Disease, was funded by Parkinson’s UK and the Cure Parkinson’s Trust. Dr Arthur Roach of Parkinson’s UK said: ‘While the results are not clear-cut, the study has been a resounding success.’
But Professor Kevin McConway of the Open University said that while the research was valuable, ‘it hasn’t really been able to move the new treatment beyond the level of being promising’.
‘Beyond anything seen previously’