“Stopped in its tracks”: New drug for Parkinson’s disease

UQ researchers find promising new treatment for Parkinson's disease


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Drug prevents loss of brain cells in Parkinson's patients, improving motor function.

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A PROMISING new therapy to stop Parkinson’s disease in its tracks  has been developed at the University of Queensland.

Researchers at UQ Faculty of Medicine have found that a small molecule, MCC950, stopped the development of Parkinson’s in several animal models.

“We have used this discovery to develop improved drug candidates and hope to carry out human clinical trials in 2020,”  said researcher Associate Professor Trent Woodruff. 

Parkinson’s disease is the second-most common neurodegenerative disease worldwide, with 10 million sufferers. 

The disease is characterised by the loss of brain cells that produce dopamine, which is a chemical that co-ordinates motor control, and is accompanied by chronic inflammation in the brain.

“We found a key immune system target, called the NLRP3 inflammasome, lights up in Parkinson’s patients, with signals found in the brain and even in the blood, said Dr Woodruff.

MCC950, given orally once a day, blocked NLRP3 activation in the brain and prevented the loss of brain cells, resulting in markedly improved motor function.

There are no medications on the market that prevent brain cell loss in Parkinson’s patients, with current therapies focusing on managing symptoms rather than halting the disease.

UQ Institute for Molecular Bioscience researcher Professor Matt Cooper said drug companies had traditionally tried to treat neurodegenerative disorders by blocking neurotoxic proteins that build up in the brain and cause disease.

“We have taken an alternative approach by focusing on immune cells in the brain called microglia that can clear these toxic proteins,” he said.

“With diseases of ageing such as Parkinson’s, our immune system can become over-activated, with microglia causing inflammation and damage to the brain.

“MCC950 effectively ‘cooled the brains on fire’, turning down microglial inflammatory activity, and allowing neurons to function normally,” said Prof. Cooper.

The study is published in Science Translational Medicine.

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