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Schneider, Susanne A. and Alcalay, Roy N. (2020): Precision medicine in Parkinson’s disease: emerging treatments for genetic Parkinson’s disease. In: Journal of Neurology, Vol. 267, No. 3: pp. 860-869 [PDF, 781kB]


In recent years, numerous clinical trials for disease modification in Parkinson’s disease (PD) have failed, possibly because of a “one-size-fits all” approach. Alternatively, a precision medicine approach, which customises treatments based on patients’ individual genotype, may help reach disease modification. Here, we review clinical trials that target genetic forms of PD, i.e., GBA-associated and LRRK2-associated PD. In summary, six ongoing studies which explicitely recruit GBA-PD patients, and two studies which recruit LRRK2-PD patients, were identified. Available data on mechanisms of action, study design, and challenges of therapeutic trials are discussed.

Parkinson’s disease (PD) is the second most common neurodegenerative disorder, affecting more than 6 million people worldwide [1]. Numerous drugs for the treatment of PD are avilable on the market. While drugs targeting the dopaminergic pathway treat motor symptoms, there is no evidence that they modify disease progression. This “one-size-fits all” approach may very well explain why clinical trials for disease modification in PD have failed. Treatments that target the underlying pathophysiology are required. Since the pathophysiology of PD may be different in different patients, studies should be designed that assess PD treatment on a more individual basis. Therefore, a precision medicine approach in PD is very timely.

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