Researchers Find Unexpected New Drug to Treat Parkinson’s Disease

In a new study published in the journal Neurotherapeutics, researchers from UCLA found that a drug being evaluated to treat an entirely different disorder also helped slow the progression of Parkinson’s disease in mice. The study found that the drug, AT2101, which has also been studied for Gaucher disease (a rare genetic disorder), improved motor function, stopped inflammation in the brain and reduced levels of alpha-synuclein, a protein critically involved in Parkinson’s disease. Researchers determined that there may be a close relationship between Gaucher disease and Parkinson’s due to an enzyme called β-glucocerebrosidase, or GCase. “This is the first time a compound targeting Gaucher disease has been tested in a mouse model of Parkinson’s disease and was shown to be effective,” said the study’s senior author. “The promising findings in this study suggest that further investigation of this compound in Parkinson’s disease is warranted.” The drug AT2101 is a first-generation “pharmacological chaperone” — a drug that can bind malfunctioning, mutated enzymes and lead them through the cell to their normal location, which allows the enzymes to carry on with their normal work. To read more about this study, click here.