A small fraction of patients with Parkinson's disease have mutations affecting their LRRK2 genes, which drug companies have been exploring as a therapeutic target, and Scientific American now reports that drugs influencing this target could potentially help more than just the patients with this particular genetic variant.
Researchers from the University of Pittsburgh recently reported that the LRRK2 kinase activity is altered in the brains of people with idiopathic Parkinson's disease. They used a series of proximity ligation assays to find wild-type LRRK2 kinase activity was heightened within substantia nigra dopamine neurons from post-mortem brains of people with idiopathic Parkinson's disease, as they reported in Science Translational Medicine last month. This suggested to them that LRRK2 kinase inhibitors could represent a treatment approach even for Parkinson's disease patients without a LRRK2 gene mutation.
"[T]hese data suggesting that LRRK2 plays a role in perhaps the other 95 percent…could mean a broader impact across the board," Todd Sherer, CEO of the Michael J Fox Foundation for Parkinson's Research, which funded the study, tells Scientific American.
Scientific American notes, though, that LRRK2 has a role the immune system and the side effects of turning the protein off are not yet clear.