Skip to main content
Funded Studies

Charting a Path to Success for Clinical Trials in Patients with Inherited, GBA-linked Parkinson's Disease: PPMI Supplement

Promising Outcomes of Original Grant:
The original study is ongoing.

Objectives for Supplemental Investigation:
We aim to determine whether fatty molecules called sphingolipids can serve as biomarkers -- disease indicators -- for tracking the progression of early-stage Parkinson's disease (PD) with an unknown cause. We will analyze the sphingolipid levels over time in people with PD and healthy people enrolled in the Parkinson's Progression Markers Initiative (PPMI), The Michael J. Fox Foundation's landmark clinical study to find biomarkers, critical missing links in the search for better Parkinson's treatments. We will analyze sphingolipids in cerebrospinal fluid, which bathes the brain and the spinal cord, and in the blood, and the resulting data will be analyzed using statistical methods. Movement and cognition will be evaluated using clinical tools to measure disease progression.

Importance of This Research for the Development of a New PD Therapy:
Identified biomarkers will be useful in evaluating multiple GBA-targeting therapeutics that are entering clinical trials.

Progress Report


Final Outcome



  • Clemens Scherzer, MD

    Boston, MA United States

Discover More Grants

Search by Related Keywords

Within the Same Program

Within the Same Funding Year

We use cookies to ensure that you get the best experience. By continuing to use this website, you indicate that you have read our Terms of Service and Privacy Policy.