RNA biomarkers of Parkinson's disease: developing tools for novel therapies

Abstract

By 2030 the number of individuals with Parkinson's disease (PD) will nearly double to approximately 9.3 million because of aging populations. No medications have been approved that address the progressive neurodegeneration that underlies the disease and existing symptomatic treatments are only partially effective. Reliance on insensitive and confounded clinical assessments has obstructed the development of novel therapeutics designed to prevent, delay or slow the disease. While PD symptoms reflect preferential neuronal death, DNA, RNA and biochemical traits of the disease are detectable in blood cells. To systematically search for lead RNA biomarkers of PD, genome-wide expression changes in the blood of patients with early-stage PD and controls have been probed by microarray. This scan identified a candidate gene signature, as well as lead single gene biomarkers associated with PD. Efforts are underway to refine and develop these hits into biomarkers that will enable risk-modifying therapies. This development process will progress through discovery, cross-sectional and prospective clinical biomarker studies, to Phase III clinical trials.