Currently, diagnosis of Parkinson’s disease relies on extensive neurological and physical examinations. A hallmark of Parkinson's is misfolded alpha-synuclein that forms aggregates, or clumps, and impairs the ability of brain cells to function. We plan to target alpha-synuclein as a biological marker (biomarker) for Parkinson’s by developing a simple method for imaging alpha-synuclein aggregates in the eye, which would be be a major step forward in Parkinson’s diagnostics.
We propose, and have validated in small models, to use fluorescent probes to visualize alpha-synuclein deposits as a diagnostic tool for Parkinson’s.
We plan to advance our program with three important pre-clinical studies of formulation, pharmacokinetics (what happens to drugs within the body) and toxicity. These are critical studies required to advance a compound into clinical testing.
Impact on Diagnosis/Treatment of Parkinson’s disease:
A fluorescein angiograph is a medical procedure that uses a fluorescent dye to highlight the blood vessels in the back of the eye so they can be photographed. If successful, this method will provide an early, accurate, and cost-effective diagnostic compared to the current standard of care in Parkinson’s diagnosis. We envision our diagnostic test to have two major impacts. First, we anticipate identifying patients with Parkinson’s, potentially years earlier than current methods. Secondly, we plan to use our diagnostic in collaboration with therapeutic agents to assist in the selection of patients, thereby improving the likelihood of success in Parkinson’s drug development.
Next Steps for Development:
The next steps for this program are a pre-IND (pre-investigational new drug application) meeting with the Food and Drug Administration and IND-enabling studies to secure approval to conduct a Phase I clinical trial.