Study Rationale: Persistent activation of the immune cells that reside in the brain leads to a state of chronic inflammation that contributes to the degeneration of neurons in Parkinson’s disease (PD). We have identified a novel inflammatory target in the brain and designed an inhibitor that enters the brain to reduce neuroinflammation and neurodegeneration.

Hypothesis: This study aims to validate the efficacy of our anti-inflammatory preclinical candidate drug in preclinical models of PD.

Study Design: Initial experiments will apply our novel anti-inflammatory drug to human and mouse inflammatory cells to determine the minimal effective dose required to inhibit inflammation. The ability of the anti-inflammatory drug to reduce inflammation in the brain will then be determined in two preclinical models of PD. If reduced brain inflammation is observed in these models — which are induced by acute exposure to different toxins — the ability of the anti-inflammatory drug to improve PD-related motor performance will then be investigated in a long-term preclinical model of the disease.

Impact on Diagnosis/Treatment of Parkinson’s disease: Therapy with an anti-inflammatory drug will provide a unique, neuroprotective, disease-modifying approach for the treatment of PD.

Next Steps for Development: Demonstration of efficacy in acute and long-term preclinical models will complete the preclinical package and allow clinical development in people with PD to proceed.