Funded Studies
This grant builds upon the research from a prior grant: Alpha-Synuclein PET Tracer Discovery
Study Rationale: The discovery of new treatments for Parkinson’s disease (PD) has been hampered by the amount of time it can take (typically a number of years) to determine whether a drug works in a clinical trial. Having tests that clinicians can use to gauge the effectiveness of therapeutics would speed the discovery of new interventions for PD. In this project, we aim to develop a new imaging tool that can be used to measure toxic clumps of alpha-synuclein protein, a defining marker of PD, in the brains of living patients.
Hypothesis: We hypothesize that the ability to monitor changes in alpha-synuclein aggregation in an individual’s brain will provide a new test for diagnosing PD and assessing the effectiveness of therapeutics.
Study Design: First, we plan to conduct clinical studies in individuals with PD using a molecule we discovered in our previous MJFF-funded project. We hope this molecule will demonstrate, for the first time, an ability to monitor alpha-synuclein pathology using PET imaging in people with PD. Second, we plan to improve on this molecule to create an alpha-synuclein imaging agent optimized to be useful in clinical studies of PD therapeutics.
Impact on Diagnosis/Treatment of Parkinson’s Disease: If successful, the discovery of an alpha-synuclein imaging agent can help determine more quickly and confidently whether a new treatment is effective. Further, it will improve doctors’ understanding of the disease by relating an individual’s symptoms and disease progression to changes in alpha-synuclein.
Next Steps for Development: If approved, we will require approximately one year to test our proof-of-concept molecule in formulation, safety, and human imaging experiments, as well as optimizing our current series of compounds.
Trial Phase: Preclinical / Clinical