Potential New Treatment Target for Parkinson's Disease Discovered

LRRK2 inhibitors could stop the progression of Parkinson’s disease and other related diseases.

Researchers have discovered that the interaction between a mutated gene and a brain protein are similar to indicators of Parkinson’s disease.

It is believed that Parkinson’s disease can be caused by environmental factors and some genetic factors, as well. The most common genetic factor that contributes to the disease is a mutated leucine-rich repeat kinase 2 (LRRK2) gene.

In the study, which was published by the Journal of Neuroscience, researchers discovered that the mutated LRKK2 gene can cause inclusions in the neurons of the brain. The inclusions are composed of α-synuclein proteins. After death, these proteins are found in patients’ brains with Parkinson’s disease.

In the study, researchers applied fibrils of α-synuclein to neurons. Researchers tested mutated LRRK2 kinase and G2019S-LRRK2 to determine the effect on α-synuclein inclusion creation.

Researchers discovered an experimental drug that inhibits mutated LRKK2 genes can reduce the creation of α-synuclein inclusions. They also found that G2019S-LRRK2 increased α-synuclein formation in dopamine neurons in the substantia nigra pars compacta, which dies in patients with Parkinson’s disease.

Researchers believe that this may slow down the progression of similar conditions, such as Alzheimer’s disease.

“These data give us hope for the clinical potential of LRRK2 kinase inhibitors as effective therapies for Parkinson's disease. The LRRK2 kinase inhibitors may inhibit the spread of pathologic alpha-synuclein, not only in patients with LRRK2 mutations, but in all Parkinson's disease patients,” concluded researcher Laura A. Volpicelli-Daley, PhD. “Future studies to validate the safety and efficacy of the LRRK2 inhibitors will be necessary before testing the inhibitors in human clinical trials.”