Validating Measurements of Peripheral GCase Longitudinally Under Clinical Trial Conditions

Study Rationale:  An enzyme known as GCase is a key protein that stops working properly in people with Parkinson’s disease (PD). However, determining exactly how impaired the enzyme’s activity might be is difficult. This project aims to measure GCase in blood cells and determine how its activity changes over time in people with PD.

Hypothesis: Our hypothesis is that we will be able to measure GCase in blood cells from people with PD and that this approach may be a valid way to monitor the activity of the enzyme over time.

Study Design: We have been collecting and storing blood samples from people with PD undergoing a clinical trial. These samples have been collected at different time points. We will use a test that we have developed and optimized to measure GCase in these samples and determine how the activity of the enzyme changes over time and how this change relates to PD symptoms.

Impact on Diagnosis/Treatment of Parkinson’s disease: A blood test that can monitor GCase could be helpful in the treatment of PD as new drugs targeting the GCase enzyme are in development. This test may help to determine who would benefit most from these potential new therapies.

Next Steps for Development: If successful, the test we have developed could be used in clinical trials testing new drugs that target the GCase enzyme.