Chronic inhibition of LRRK2 as a therapeutic target in PD: how safe will it be in humans?

image of a human astrocyteWe are excited to share the publication of our latest research that explored the molecular effects of long-term pharmacological LRRK2 inhibition in vivo. This comprehensive study was completed by Ph.D. candidate Jillian H. Kluss in the lab of Dr. Mark R. Cookson at the National Institute on Aging, NIH. Adamantios Mamais, Ph.D., assistant professor in the department of neurology and the center for translational research in neurodegenerative disease (CTRND), and team explain how looking at the effects of chronic drug administration in the brain, kidney and lung of preclinical models of Parkinson’s disease helped us identify alterations in endolysosomal and trafficking pathways that are sensitive to LRRK2 inhibition. What remains to be explored is whether these effects are beneficial in pathology and how relevant they are to what happens in human disease.

Read the article in Molecular Degeneration.