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2 mins read
Researchers from the Universities of Sydney and Stanford have developed promising therapies targeting key proteins and mutations involved in Parkinson’s disease. Results in animal models show significant improvements in motor and neuronal function, paving the way for more effective combined treatments for a condition that continues to rise
Parkinson’s disease is a degenerative neurological condition that affects nearly 10 million people worldwide. Its incidence has doubled over the past 25 years, and it is estimated that cases could exceed 25 million by 2050. With no known cure and limited treatment options, it poses a growing challenge for healthcare systems—not only due to its impact on patients’ quality of life but also because of the high associated costs, estimated at around $10,000 per person annually.
Characterized by the progressive loss of dopamine-producing neurons, the disease causes symptoms such as tremors, muscle stiffness, slowed movements, and balance disturbances. For over a decade, the team led by Professor Kay Double at the University of Sydney has been investigating the biological mechanisms underlying this condition, aiming to develop new therapeutic strategies.
In 2017, Professor Kay Double’s team identified for the first time an abnormal form of the SOD1 protein in the brains of people with Parkinson’s disease. Normally, this protein acts as a protective antioxidant enzyme, but when altered, it loses that function. Instead of protecting neurons, it accumulates and damages them, contributing to disease progression.
Building on this discovery, the team explored the therapeutic potential of copper—an essential element for SOD1 function. They developed a drug called CuATSM, capable of crossing the blood-brain barrier and delivering copper directly to the brain. The study, published in Acta Neuropathologica Communications, was conducted in two phases: first, determining the optimal dose to trigger a brain response; then, applying that dose to genetically modified mice exhibiting Parkinson-like symptoms.
For three months, one group received CuATSM treatment while another received a placebo. The control group showed progressive motor deterioration, whereas the treated mice maintained normal movement and preserved dopaminergic neurons in the substantia nigra—a key region for motor control and cognitive functions. Researchers concluded that the treatment restored SOD1’s protective properties and halted neuronal damage.
This finding complements parallel research on genetic variants of Parkinson’s. A study led by Suzanne Pfeffer at Stanford University focused on the LRRK2 gene mutation, the most common genetic subtype of the disease. This mutation causes overactivity of the encoded enzyme, disrupting cellular structure and impairing communication between dopaminergic neurons and the striatum—a region involved in movement, motivation, and decision-making.
Treatment with an experimental inhibitor called MLi-2 successfully restored key cellular structures—primary cilia—and reactivated neuronal communication. After three months, researchers observed recovery of synaptic connections and increased markers of dopaminergic nerve endings. While preliminary, these results suggest therapeutic potential to stabilize and even reverse symptoms, especially if treatment begins early.
Both lines of research point in the same direction: Parkinson’s is a complex disease likely to require combined therapies. A single intervention may have limited impact on its own but could contribute significantly when integrated into a broader approach. Understanding the mechanisms that trigger and sustain the disease remains an urgent priority for science.
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