Study Rationale: LBT-3627 is a disease-modifying therapeutic candidate that has been shown to reduce brain inflammation and immune imbalance in preclinical models of Parkinson's disease (PD). A recent biomarker study found that about half of people with PD display an immune imbalance comparable to that seen in preclinical PD models. In this project, we aim to translate LBT-3627 into clinical trials and obtain preliminary clinical evidence of immune transformation in people with PD.
Hypothesis: We hypothesize that restoring immune balance using LBT-3627 in people with PD will lead to improved clinical outcomes.
Study Design: We will initiate a Phase I clinical trial and enroll both healthy volunteers as well as people with PD who exhibit a dysfunctional immune system. Participants will be treated with a single dose of LBT-3627 or a series of increasing doses. The primary objective is to determine the safety and tolerability of LBT-3627. Secondary objectives include following patients both immunologically and using a unified PD rating scale to assess disease severity and progression. Results will be analyzed to determine any treatment effects.
Impact on Diagnosis/Treatment of Parkinson’s Disease: Should this project be successful, it could help usher in a new immune-focused approach for both diagnosing and treating PD.
Next Steps for Development: Next steps for this project would include proceeding with a larger Phase II clinical study to further evaluate the efficacy of LBT-3627. In addition, immunological information obtained from this study could lead to additional diagnostic capabilities in terms of future PD clinical trials.