Study Rationale:
Multiple candidate Parkinson’s disease (PD) biomarkers have been identified over the years, but none have been properly validated. Using a targeted mass spectrometry platform, a multiplexed assay (experimental set-up) will be developed to accurately quantify approximately 170 candidate protein markers in the cerebral spinal fluid (CSF) of Parkinson’s disease patients and control subjects. The goal will be to identify one or more proteins that will serve as markers of disease and disease progression.
Hypothesis:
Of the many proteins associated with Parkinson’s disease and neurodegeneration in general, it is expected that a small subset will prove useful in monitoring disease progression and eventually therapeutic efficacy.
Study Design:
An advanced mass spectrometer will be used that can accurately identify and measure signals from each of these proteins in CSF, the fluid that bathes the brain and spinal column. Proteins will be tested for their ability to distinguish disease from control subjects. If successful, those that pass this first test will be validated on additional patient and control samples in a future study.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
A biomarker protein or panel of proteins that are associated with PD could eventually serve as an early diagnostic, a means of measuring disease progression and/or a measure of drug efficacy in a clinical trial.
Next Steps for Development:
A panel of one or more proteins that can classify Parkinson’s disease patients will be further tested for sensitivity and specificity and eventually validated using additional patient and control subject samples, including those from other neurodegenerative diseases.