University of Colorado School of Medicine researchers have found that phenylbutyrate stops the progression of PD in mice. The drug, previously approved for treatment of a rare metabolic disease in infants, has been found to increase alpha-synuclein in the bloodstream of both patients with early PD and age matched controls. Alpha-synuclein accumulates in excess in the brain's of patients with PD. Therefore, the finding of an increase in alpha-synuclein in the blood of phenylbutyrate treated patients with PD suggests that this protein that accumulates in excess in their brain's is being cleared by the drug. This new research follows the earlier study of Zhou as published in 2011.
J Biol Chem 2011;286:1491-1451