Once again “our” alpha-synuclein (SNCA)—the protein that my team linked to Parkinson’s back in the ’90’s—is in the forefront of potential treatment approaches! A team at UCLA has reported improvement of PD-like symptoms in a mouse model (mice that over-produce SNCA and demonstrate Parkinson disease-like symptoms) with a drug being studied to treat a rare genetic disorder, Gaucher disease.
Gaucher disease occurs when two carrier-parents pass on a gene which prevents the body from producing an essential enzyme, glucocerebrosidase (GCase). In 2011, a team at Massachusetts General Hospital described the interaction of Gauchers and Parkinson’s in the same pathway: disruption of the GCase pathway results in SNCA deposits and as SNCA levels rise, the Gaucher’s associated pathway is inhibited. This suggested that medications used for one might benefit the other.
The drug, AT2101, is a “chaperone” drug which helps guide malfunctioning enzymes to where they need to function. In the mice that over-express SNCA, (as in PD patients) the drug improved motor function, stopped brain inflammation, and reduced SNCA, suggesting its potential effectiveness for treating Parkinson’s.
Keep the faith.