Gaps in the Parkinson's disease therapeutic clinical pipeline: A focus on approaches targeting disease pathobiology

The understanding of the pathobiology of Parkinson's disease (PD) is evolving, based largely on genetic discoveries. Despite a rich pipeline of potential therapies addressing aspects of the underlying biology of the disease, there is currently no available disease-modifying therapy for PD. In this short commentary, we review the status of the relevant pipeline and highlight areas where alternative or complementary approaches could also be advanced to the stage of clinical trials, with the ultimate goal of providing meaningful clinical benefit to patients and their families. The further evolution of biomarkers, cellular and animal models of the disease and delivery methods will be crucial to this end. Plain language summary The understanding of the background of Parkinson's disease (PD) is evolving, based largely on genetic discoveries. Despite numerous potential therapies addressing aspects of the underlying biology of the disease, there is currently no available therapy able to modify PD clinical course. In this short commentary, we review the status of the relevant therapeutic trials landscape and highlight areas where alternative or complementary approaches could also be advanced to the stage of clinical studies, with the ultimate goal of providing meaningful clinical benefit to patients and their families. The further evolution of biomarkers, cellular and animal models of the disease and delivery methods will be crucial to this end.