A new treatment that can halt and even reverse Parkinson’s disease is giving sufferers fresh hope.
Researchers found boosting levels of a naturally occurring protein can regenerate dying brain cells – if delivered directly into the brain via tubes.
The £3million study, funded by Parkinson’s UK and pharma firm MedGenesis, had a five-year trial at Bristol’s Frenchay and Southmead hospitals.
And by the end of it, some participants had stopped having tremors and could walk from one side of a room to the other without help.
The protein Glial Cell Line Derived Neurotrophic Factor has been tested before but it was the first time it had been delivered directly to the centre of the brain.
Professor Steven Gill, who came up with the delivery method, said: “This trial has shown we can safely and repeatedly infuse drugs directly into patients’ brains over months or years through a small implanted port that emerges behind the ear.
“This is a significant breakthrough in our ability to treat neurological conditions such as Parkinson’s because most drugs that might work cannot cross from the bloodstream into the brain due to a natural protective barrier.”
GDNF was given to 41 participants. Among them was Tom Isaacs, 49, who was diagnosed aged 27 and co-founded The Cure Parkinson’s Trust in 2005.
Tom was instrumental in making the trial happen, working with Prof Gill, and had one of the most severe cases.
But after several rounds of GDNF infusions, he went from having violent tremors and barely being able to walk to running around the garden.
Tragically, Tom died from an unrelated heart condition in 2017. Prof Gill said: “Tom was absolutely central to making this happen. Without Tom, there’s no question, we wouldn’t be here.”
Christine Proctor said taking part in the trial changed her life.
The 57-year-old, diagnosed in 2004, was initially given a placebo treatment and did not see any improvements.
After nine months, she was offered GDNF.
Ms Proctor, from Consett in County Durham, said: “Within just two days of having the infusion, I noticed an improvement in my mobility. I wake up like a lump of wood normally but I could actually sit up without assistance and move quite nimbly.”
She went from driving an automatic car to a manual and even cut down on her normal medication after treatment.
“The difference GDNF made was life-changing for me,” she said. “It gave me mobility but also confidence as a result of that mobility. It took me from dependence to independence.”
Her last infusion was two years ago but she believes the progression of her Parkinson’s slowed after the trial.
Half the participants of the study, which started in 2012 and ended in 2017, were randomly assigned to get monthly infusions of GDNF and the others placebos.
All were then offered a nine-month course of GDNF. Brain scans showed signs of improvement, according to findings published in journal Brain and the Journal of Parkinson’s Disease.
Principal investigator Dr Alan Whone said: “This represents some of the most compelling evidence yet that we may have a means to reawaken and restore the dopamine brain cells that are gradually destroyed in Parkinson’s.”
Another trial is being planned and Prof Gill believes the technology could also be used to administer chemo to brain tumour patients or test drugs for Alzheimer’s and stroke patients.
He said: “Now we’re looking at moving on with trials and increasing the dosage.
“Hopefully, this will be a definitive study to bring this therapy to people. We’re feeling very positive about this.”
Research is the key
Tom Phipps, 63, of Bristol, was the first person to undergo the surgery.
He said: “During the trial, I noticed an improvement in my mobility and energy levels, and I was even able to reduce my medication.
“Clinical trials are so important because it’s not going to go away.”
My Tom knew there was a cure
A few years ago, Tom Isaacs could barely walk and he would have violent shaking episodes every day.
But halfway through the trial, Tom was no longer shaking, he could walk and, remarkably, he managed to run for the first time in 17 years without any medication.
His widow, Lyndsey, 55, from Herts, said: “Tom knew there would be a cure.
“When Tom started having more GDNF in 2015, he was definitely much better. And what was amazing was even when he stopped having it, his Parkinson’s didn’t really progress. It stayed at the same level.”
Tom set up the Cure Parkinson’s Trust to fund more research into GDNF.
Lyndsey said: “It is an amazing legacy. Tom didn’t accept Parkinson’s would stop him from doing anything. I wish he was here today to see what he has achieved.”
It works – we need it
Vicki Dillon was devastated when she was diagnosed aged 35.
The former nurse, now 47, said: “My world just stopped turning, I was horrified.” But after being on the GDNF trial, she insisted: “My Parkinson’s got better.
“The trial changed my life and a lot of other people’s lives. It does work. There’s no doubt in my mind: We need it.”