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.
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.