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Several clinical trials aimed at disease modification in Parkinson’s disease (PD) have been unsuccessful in recent years, possibly due to a universal treatment approach that does not consider differential pathophysiologies in different people. Alternatively, adopting a precision medicine strategy, which tailors treatments based on the specific genotype of each patient, could be beneficial in achieving disease modification. In this review, we examine clinical trials focusing on genetic forms of PD, namely, SNCA-, GBA1-, and LRRK2-associated PD. Overall, we have identified no ongoing trials dedicated to SNCA-PD, two studies that will recruit GBA-PD patients that will begin soon (with others already completed), and one study recruiting LRRK2-PD patients. The available information regarding the mechanism of action, study design, and challenges encountered in therapeutic trials is also discussed.

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Translating Therapeutic Strategies for Genetic Parkinson’s Disease Syndromes

  • 实验方案

  • 发表日期：

    2025年

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  • Susanne A. Schneider,
  • Roy N. Alcalay

摘要

Several clinical trials aimed at disease modification in Parkinson’s disease (PD) have been unsuccessful in recent years, possibly due to a universal treatment approach that does not consider differential pathophysiologies in different people. Alternatively, adopting a precision medicine strategy, which tailors treatments based on the specific genotype of each patient, could be beneficial in achieving disease modification. In this review, we examine clinical trials focusing on genetic forms of PD, namely, SNCA-, GBA1-, and LRRK2-associated PD. Overall, we have identified no ongoing trials dedicated to SNCA-PD, two studies that will recruit GBA-PD patients that will begin soon (with others already completed), and one study recruiting LRRK2-PD patients. The available information regarding the mechanism of action, study design, and challenges encountered in therapeutic trials is also discussed.