New international research led by Western Australian neurologists and scientists highlights the potential for precise and personalised treatment of Parkinson’s disease.
As Parkinson’s is a complicated disorder driven by multiple processes, comprehensive therapeutic strategies are likely to be required in parallel with a personalised approach, the research suggests.
The perspective paper, ‘A personalised and comprehensive approach is required to suppress or replenish SNCA for Parkinson’s disease’, was published in Nature journal, NPJ Parkinson’s Disease.
Lead author is Dr Dunhui (Oliver) Li, until recently with the Perron Institute and now Associate Research Scientist at the Department of Neurology and Adams Center for Parkinson’s Disease Research, Yale University, United States.
“The connection between alpha-synuclein (α-synuclein), a neuronal protein encoded by the SNCA gene, and Parkinson’s was discovered in 1997, representing a milestone in Parkinson’s research,” Dr Li said.
“Since then, researchers have been working to understand the function and underlying mechanisms of how α-synuclein causes Parkinson’s and to develop effective disease-modifying therapeutic strategies.
“Reducing α-synuclein levels and removing its aggregates has been the current focus of new experimental therapies for Parkinson’s disease. However, evidence has been emerging that the process of α-synuclein aggregation may in fact lead to a deficiency of the soluble functional form of the protein in neurons, and that this may need to be replenished to sustain neuronal function.
“Based on our research findings, we propose a personalised and comprehensive approach for treating different Parkinson’s subgroups based on whether α-synuclein is likely to contribute to disease pathogenesis through an excess (“gain-of-function”) or a depletion of the protein (“loss-of-function”), or through a combination of both mechanisms.
“Our perception is that the ideal disease-modifying strategy should comprise a combination of personalised approaches. We believe a biological or biochemical definition of α-synuclein could be the key to the discovery of novel therapies targeting underlying disease pathogenesis.
“Getting the right treatment for Parkinson’s is challenging, with huge gaps still to be filled, but we are optimistic that continued investigation of therapeutic approaches targeting α-synuclein will lead to improved quality of life for people with Parkinson’s,” Dr Li said.
Perron Institute senior advisor and Clinic co-founder Professor Frank Mastaglia is senior author of the research paper. Other authors are Perron Institute Consultant Neurologist Dr Wayne (Wai Yan) Yau, Professor Steve Wilton (Perron Institute and Murdoch University), and Professor Shengdi Chen, Department of Neurology and Institute of Neurology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.