Emerging Targeted Therapeutics for Genetic Subtypes of Parkinsonism

Schneider, Susanne A.; Hizli, Baccara; Alcalay, Roy N.

In recent years, a precision medicine approach, which customizes medical treatments based on patients’ individual profiles and incorporates variability in genes, the environment, and lifestyle, has transformed medical care in numerous medical fields, most notably oncology. Applying a similar approach to Parkinson’s disease (PD) may promote the development of disease-modifying agents that could help slow progression or possibly even avert disease development in a subset of at-risk individuals. The urgent need for such trials partially stems from the negative results of clinical trials where interventions treat all PD patients as a single homogenous group. Here, we review the current obstacles towards the development of precision interventions in PD. We also review and discuss the clinical trials that target genetic forms of PD, i.e., GBA-associated and LRRK2-associated PD. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s13311-020-00920-8) contains supplementary material, which is available to authorized users.