Initiative set to recharge brain to halt Parkinson’s disease
LONDON: Pioneering scientists have moved a step closer to a radical medical intervention that promises to halt the progression of Parkinson’s disease by “recharging the batteries” of brain cells.
Research will begin within weeks into a form of gene therapy for Parkinson’s patients that will prevent the degenerative disease process that starves the neurons of energy and leads to cell death.
If scientists can show they can provide the brain cells with an alternative continual source of energy that prevents brain cell death, they will have proven Parkinson’s disease can be not only treated, but halted.
“We are really looking at a way to provide a new, reliable sustainable source of energy for the brain cells that die in Parkinson’s disease,” said Professor Carolyn Sue AM, Kinghorn Chair, Neurodegeneration at Neuroscience Research Australia. “What happens in Parkinson’s disease is that parts of the brain degenerate and don’t work anymore, particularly those bits that help control movement.
“When brain cells start degenerating it’s because they lose energy, just like if you have a mobile phone that loses charge. Energy fuels active things like making the synapses work, it allows proteins and molecules to be transported around the cells, and helps get rid of the rubbish that stops the cell from working properly. In Parkinson’s disease, all of those processes are slowly or gradually lost.
“This new treatment works on a new way to recharge the batteries.”
Professor Sue will begin preclinical research on the new gene therapy in coming weeks after receiving a $1.7m grant jointly funded by Australian charity Shake it Up and the Michael J. Fox Foundation. Hollywood actor Fox was diagnosed with Parkinson’s disease in 1991 aged 29.
The NeuRA research is aiming to provide the cells with a sustainable source of energy to combat the cell death that occurs in Parkinson’s disease that results from the impaired function of the dopaminergic neurons due to energy loss.
Professor Sue is now testing her hypothesis that a cell protein known as Nix can restore cell mitochondrial function and prevent the widespread cell death that causes Parkinson’s disease’s debilitating symptoms, which include tremors or shaking, muscle rigidity or stiffness, the slowing of movement and balance problems.
Professor Sue’s research puts her at the precipice of a major medical advance after she established overproduction of Nix restored a cell’s ability to regenerate its own battery power and remove its damaged ones. The discovery was made from studying a woman who naturally over-expressed the Nix protein whose Parkinson’s disease did not progress.
Parkinson’s disease is a disorder of the nervous system characterised by damage to the brain cells that produce dopamine. Currently, the symptoms can be treated, but there is no way to stop the disease progressing. The NeuRA scientists will be testing in cell models affected by Parkinson’s whether over-expression of the Nix protein can restore the function of dopaminergic neurons by providing an alternative way for mitochondria to recharge themselves and prevent the neurons running out of energy.
“If this research is successful, it will be a game-changer,” Professor Sue said. “This will be the first time we’ll be able to protect cells from dying.
“All therapies at the moment for Parkinson’s disease are truly to treat symptoms, but they don’t actually get to the crux of the problem, which is that it’s a degenerative process in the first place. So this will be a treatment that could be given to all patients with Parkinson’s disease to stop the disease process.”
Clyde Campbell AM was diagnosed with Parkinson’s disease 13 years ago when he was only 44.
“The first time I noticed it was doing a major opening address speech; the notes started to shake in my hand,” Mr Campbell said.
“It was a shock to be diagnosed because I was quite young.”
Father of three Mr Campbell, founder of the Shake It Up Foundation and chair of its board, exercises every day with his dog Zebby to help slow the progression of his disease, and is grateful for the drugs that bring him symptom relief but said if his disease could be halted via gene therapy it would be incredible and grant him years of joy being able to play with his grandchildren. “If we can stop the cell loss it can make a mass difference to us all.”
If the preclinical studies replicate what has been observed in the patient who naturally overexpresses the Nix protein, the therapy will progress to human clinical trials.
The Shake It Up Foundation has contributed $22m to Parkinson’s disease research. “It’s a great opportunity to be able to change the progression of disease,” Mr Campbell said.
