Precision medicine in Parkinson’s disease: emerging treatments for genetic Parkinson’s disease
Susanne A. Schneider, Roy N. Alcalay
Abstract
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.
Topics & Concepts
LRRK2DiseaseParkinson's diseaseClinical trialNeurologyMedicinePrecision medicineNeuroradiologyIntensive care medicineBioinformaticsInternal medicinePathologyPsychiatryBiologyLysosomal Storage Disorders ResearchParkinson's Disease Mechanisms and TreatmentsNeurological diseases and metabolism