Progress in Disease-Modifying Therapies for Parkinson's Disease

Parkinson's disease (PD) is a highly prevalent neurodegenerative disorder, and current therapeutic approaches fail to prevent the progressive loss of dopaminergic neurons. To date, no disease-modifying therapies (DMTs) have been approved for PD. Developing effective DMTs remains the foremost objective in PD research. Here, we review the rationale for α-synuclein, LRRK2, GBA1, the PINK1-Parkin axis, and GLP-1R as potential therapeutic targets for PD. Additionally, we summarize the functional alterations observed in cellular organelles, including mitochondria, lysosomes, and the endoplasmic reticulum, in the context of PD. We also highlight the progress in drug development targeting these therapeutic candidates and the associated organelles. Furthermore, we discuss the advancements in stem cell-based therapeutic strategies in the field of PD research. It is believed that deepening understanding of disease mechanisms, combined with the development of novel technologies, offers promising potential for more effective solutions to this debilitating condition.