Advancing disease-modifying therapies for Parkinson’s disease: Current strategies and future directions

Parkinson's disease (PD) is the second most prevalent neurodegenerative disorder, characterized by the accumulation of α-synuclein (α-syn) aggregates and the loss of dopaminergic neurons in the midbrain. PD affects more than 10 million individuals worldwide, but no therapy has been proven to slow down its progression, posing a significant socioeconomic burden. Developing interventions to delay or halt disease progression remains a top priority for researchers. Recently, several disease-modifying approaches have been proposed for PD treatment, such as passive immunization, small-molecule inhibitors or gene editing therapy directly targeting α-syn aggregation, mitochondrial-targeted strategies, and cell replacement therapy. Our recent research has identified FAM171A2 as a novel neuronal receptor for pathological α-syn, offering a potentially viable target for disrupting α-syn transmission that is previously not known. Further development of these strategies could offer new hope for effective treatments of PD in the near future.