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Precision medicine in Parkinson's disease: emerging treatments for genetic Parkinson's disease.

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.

SUBMITTER: Schneider SA

PROVIDER: S-EPMC7035220 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature

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Precision medicine in Parkinson's disease: emerging treatments for genetic Parkinson's disease.

Schneider Susanne A SA Alcalay Roy N RN

Journal of neurology 20200123 3

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 ...[more]

PMID: 31974807

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