Probucol: Hope wonder drug can treat both Parkinson's and Alzheimer's diseases in new Perth trial
A new clinical trial to treat Parkinson's disease will start in Perth next year using a drug currently under investigation as a ground-breaking treatment for Alzheimer's disease.
Probucol, a drug originally used in the 1980s to treat cardiovascular disease and lower cholesterol, has since been found to have potent anti-inflammatory and anti-oxidant effects on the brain.
The Probucol in Alzheimer's disease study, which started in 2021 and has so far recruited around 100 people, has just received ethics approval to extend treatment access for participants for an additional two years.
Professor John Mamo, the trial's chief investigator, said results from the two-year double blind trial were not yet available, but the open label extension was an 'exciting opportunity' for those taking part.
'The scientific significance lies in combining data from both the PIA study and the open-label extension. We'll gain insight into the efficacy of four years of treatment and also understand the impact of delaying treatment by two years — as in the case of those originally randomised to placebo,' Professor Mamo said.
Professor Mamo, Head of Neurovascular and Metabolic Diseases at the Perron Institute and Curtin University, said Probucol has properties that are highly relevant across several neurodegenerative conditions and Parkinson's disease was the next target for investigation.
In the context of Alzheimer's, Professor Mamo and his research team discovered that amyloid, a key protein implicated in the disease, is secreted outside the brain as part of fat-carrying particles called lipoproteins.
They showed that when amyloid-rich lipoproteins are excessively abundant in the blood, they compromise the integrity of microscopic brain vessels, allowing amyloid to leak into the brain and trigger inflammation.
'We found that Probucol powerfully suppresses the synthesis and secretion of amyloid into the blood, preserving brain capillaries and preventing brain cell loss due to inflammation,' he said.
But Professor Mamo said that amyloid plaque build-up in the brain likely represents the end stage of this damaging process — suggesting it may be the metabolic process beforehand, not the plaque itself, that principally drives the disease.
Preclinical studies have shown that Probucol can profoundly preserve cognitive function in models of Alzheimer's.
He said new monoclonal antibody therapies available to treat Alzheimer's were highly effective at reducing plaque burden but only had modest effects on halting cognitive decline.
'It's still unclear whether plaques are more a consequence than a cause,' he said.
The Parkinson's trial will start in 2026 through the Perron Institute and Curtin University with Perth being the main research site.
Alzheimer's disease is the biggest killer of women in Australia, and the second biggest for men and women. Professor Mamo said women with the disease deteriorate at three times the rate of men.
Parkinson's disease is the fastest growing neurogenerative disease with 150,000 people living with it in Australia and 20 per cent of them are below retirement age.
The Probucol in Alzheimer's study is looking to recruit around 50 more people who can contact the PIA study team on piastudy@curtin.edu.au.
They do not need a doctor's referral or a diagnosis of Alzheimer's or dementia to be considered and can contact the PIA study team on
piastudy@curtin.edu.au
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