Efficacy and Safety of Chronic Treatment with LRRK2 Inhibitors

Study Rationale:
LRRK2 inhibitors are among the most promising in the current generation of potential novel treatments for Parkinson's disease (PD). In pre-clinical models, long-term treatment with LRRK2 inhibitors alleviates Parkinson's symptoms. The present work has two purposes: first, to confirm that LRRK2 inhibitors can alleviate Parkinson's symptoms; and second, to assess if the treatment has any negative effects on the lungs.

Hypothesis:
We hypothesize that chronic treatment with LRRK2 inhibitors will alleviate Parkinson's symptoms in pre-clinical models.

Study Design:
We will develop pre-clinical models with Parkinson's symptoms and treat them with LRKK2 inhibitors for an extended period. Following the treatment period, we will evaluate changes in movement and markers of PD in the brain. After the evaluation is complete, we will examine the lungs of the models for any side effects.

Impact on Diagnosis/Treatment of Parkinson's disease:
This research can bring LRRK2 inhibitors closer to clinical use, help design clinical studies and, eventually, determine the optimal treatment dose.

Next Steps for Development:
To take this research a step further, we would use models that share traits with Parkinson's, such as Chronic Obstructive Pulmonary Disease (COPD). This would help us better understand how these circumstances affect treatment efficacy and safety.