New Gene Therapy Research Targeting the Mitochondrial Function could Halt the Progression of Parkinson’s
Wednesday, 11th January 2023

Shake It Up Australia Foundation and The Michael J. Fox Foundation have committed $1.67M in funding for a new research project that targets the mitochondrial system and aims to stop cell death.

The research will be conducted by Professor Carolyn Sue AM, Kinghorn Chair, Neurodegeneration at Neuroscience Research Australia (NeuRA). The study will explore if the Nix protein restores mitophagy and mitochondrial function in people with the PINK1/PARKIN gene mutation. The team is now working on the theory that overexpressing the Nix protein using gene therapy could stop the progression of Parkinson’s.

“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.”

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The research will test the 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, including tremors or shaking, muscle rigidity or stiffness, and slowing of movement and balance problems.

“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.”

Founder of Shake It Up Australia Clyde Campbell AM was diagnosed with Parkinson’s at the age of 44 and exercises daily with his dog Zebby to help slow the progression of his disease. Clyde 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. “If we can stop the cell loss it can make a mass difference to us all.”

Read the full article in The Australian


Clyde Talks to 10 News on his diagnosis and hopes for the future.

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Media Contacts:

Troy Whittaker Media & Communications Manager – GoodPR – 0406 742 003

Vicki Miller CEO Shake It Up Australia Foundation – 0413 417 486