A New Target for Parkinson’s Disease

Abstract

LRRK2 blockade has emerged as a potential target for developing disease-modifying therapies against Parkinson’s disease. Both in cellular and animal models, DNL201- a first-in-class central nervous system–penetrant LRRK2 kinase inhibitor, successfully mitigated the LRRK2 function resulting in re-establishing lysosomal activity as per the findings of Jennings and colleagues. In both healthy volunteers and patients with Parkinson’s Disease, LRRK2 kinase was inhibited by DNL201 and the drug candidate was also shown to be safe and tolerable at doses that improved lysosomal functioning. These findings argue for advanced studies on LRRK2 inhibitors including early as well as late-stage clinical investigations in patients with Parkinson's disease. Sci. Transl. Med.14, eabj2658 (2022).