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Funded Studies

The Effects of a LRRK2 Inhibitor on Alpha-synuclein-induced Dopamine Deficits in a Pre-clinical Model of Parkinson's Disease

Study Rationale:
In a previous study, we demonstrated that PFE360, an LRRK2 inhibitor, has neuroprotective benefits. However, several questions remain unanswered as follows: (1) Did the vehicle (substance) used to mix the PFE360 solution affect the results of the study? (2) Can we develop a way to deliver PFE360 that would not affect the results of the study? (3) Would the neuroprotective effects of PFE360 be maintained and/or enhanced if a vehicle is not used to mix the PFE360 solution? This study will address these questions.

The PFE360 vehicle affects the magnitude of deficits produced by delivery of alpha-synuclein into a pre-clinical model of PD.

Study Design:
In the first study, we will deliver a vehicle, an alternate vehicle (different from the vehicle used in previous research), delayed alpha-synuclein or water as the control to pre-clinical models to determine the effect of the PFE360 vehicle. The results of this study will determine the strategy used in the second study.

Impact on Diagnosis/Treatment of Parkinson's disease:
We can inform drug development by providing a better understanding of how drug formulations may impact pre-clinical models,.

Next Steps for Development:
The results of this study will allow us to better interpret pre-clinical research conducted to date using this compound and its associated vehicle.


  • James B. Koprich, MA, PhD

    Toronto ON Canada

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