Study Rationale:
This study aims to compare the immune system of Parkinson's disease (PD) patients and their caregivers in an effort to determine differences between these groups. Several recently published clinical research efforts have implicated the immune system as a potential source of PD biomarker information. This study aims to expand on these findings and attempt to correlate the findings with traditional clinical measurements. Should this clinical study be successful, it may be possible to identify a blood test for Parkinson's disease. In addition, we will incubate cells to test the ability of drug candidate LBT-3627 to convert PD immune cell profiles into 'caregiver' profiles.
Hypothesis:
This study will attempt to separate PD patients from their environmentally matched caregivers based on blood-based inflammatory markers. Furthermore, we will test LBT-3627, an immunomodulatory therapeutic candidate that has demonstrated the ability to improve immune function and be neuroprotective in models of PD.
Study Design:
We will collect blood samples from PD patients and their caregivers who share the same environment. These samples will be evaluated for multiple inflammatory and immunological endpoints. We will then attempt to correlate these results with 1) alpha-synuclein levels and 2) the Unified Parkinson's Disease Rating Scale (UPDRS) Part III score. We aim to identify statistically significant differences between these two groups based on these markers. In addition, a subset of blood cells will be incubated and evaluated before and after dosing with LBT-3627 to determine the ability to transform these cells from a "PD" profile to a "healthy" profile.
Impact on Diagnosis/Treatment of Parkinson's Disease:
If this project is successful, we may be able to identify and track the progression of Parkinson's disease based on a specific blood test - while also performing a preliminary evaluation of a unique immunomodulatory therapeutic strategy for PD. This would have a significant impact on early diagnosis, clinical trials and treatment progress alike.
Next Steps for Development:
If successful with this initial clinical study, we would then expand into a much larger cohort of patients for additional therapeutic and biomarker validation. In addition, we would be pursuing a U.S. Food and Drug Administration-regulated clinical diagnostic and/or therapeutic program.