Groundbreaking research by the University of Sydney has identified a new brain protein involved in the development of Parkinson’s disease and a way to modify it, paving the way for future treatments for the disease.
With the aim of finding new treatments to slow or stop its progression, the research team has spent more than a decade studying the biological mechanisms underpinning the condition—which is the second most common neurological condition after dementia.
In 2017, they identified for the first time the presence of an abnormal form of a protein—called SOD1—in the brains of patients diagnosed with Parkinson’s disease.
Normally, the SOD1 protein provides protective benefits to the brain, but in Parkinson’s patients it becomes faulty, causing the protein to clump and damage brain cells.
The newest study by the same team, led by Professor Kay Double from the Brain and Mind Centre, was just published in Acta Neuropathologica Communications. It found that targeting the faulty SOD1 protein with a drug treatment using copper improved the motor function in mice.
“We hoped that by treating this malfunctioning protein, we might be able to improve the Parkinson-like symptoms in the mice we were treating – but even we were astonished by the success of the intervention,” said Professor Double in a media release.
“All the mice we treated saw a dramatic improvement in their motor skills, which is a really promising sign that it could be effective in treating people who have Parkinson disease too.
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The study involved two groups of mice with Parkinson-like symptoms. One group was treated with a special copper supplement for three months, while the other received a placebo.
Throughout the study (which was partly funded by the Michael J. Fox Foundation), the mice receiving only the placebo saw a decline in their motor symptoms. The mice receiving the special copper supplement, however, did not develop movement problems.
“The results were beyond our expectations,” said Prof. Double. “They suggest, once further studies are carried out, this treatment approach could slow the progression of Parkinson’s disease in humans.”
At present there is no known cure and only limited treatments for Parkinson’s disease, which is a degenerative disorder in which dopamine-producing cells in the brain die, leading to a range of symptoms including tremors, muscle stiffness, slow movement and impaired balance.
But researchers hope understanding the causes of the disease will lead to improved treatments.
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“As our understanding of Parkinson’s disease grows, we are finding that there are many factors contributing to its development and progression in humans – and faulty forms of the SOD1 protein is likely one of them.
“Just as researchers found with HIV, Parkinson’s disease is a complex condition that likely requires multiple interventions. A single treatment may have a small effect when used alone but, when combined with other interventions, contributes to a significant overall improvement in health.”
The researchers’ next step is to identify the best approach to targeting the faulty SOD1 protein in a clinical trial, which could be the start of a new therapy to slow the development of Parkinson’s disease.
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