Study Rationale:
Adaptive and innate immune responses play important roles in neuroinflammation and can be harnessed to improve outcomes in Parkinson's disease (PD). Daily administration of LBT-3627 increases function of the vasoactive intestinal peptide receptor 2 (VPAC2), which affects the immune system by turning neurotoxic (causes nervous system damage) activities to neurotrophic (promotes nervous system growth) activities. Here, we aim to explore additional therapeutic options, such as a less frequent dosing schedule and different routes of administration. In addition to studying immunomodulatory benefits, neuroprotective capabilities of LBT-3627 will also be explored.
Hypothesis:
Preliminary data have suggested a prolonged immune shift to neuroprotection in PD after taking the drug, we aim to determine the minimum dosing frequency and other routes of administration that result in neuroprotective outcomes, while also determining neuroprotective features of the VPAC2 pathway.
Study Design:
This project aims to refine the therapeutic clinical product profile of this compound by optimizing dose frequency and route of administration and to determine the direct neuroprotective and neurotrophic activities of LBT-3627 in cell culture models.
Impact on Diagnosis/Treatment of Parkinson's disease:
Immunomodulation and inflammation hold great potential to alter Parkinson's progression. The VPAC2 pathway can rebalance the immune system toward a neuroprotective state. Furthermore, the direct neuroprotective capabilities of the VPAC2 receptor may also provide disease-modifying effects as well. Further development of the immune transformation and protection profile of LBT-3627 may help establish the case for clinical evaluation.
Next Steps for Development:
Should the first two aims of this proposal be successful, the next step would involve completion of a full Investigational New Drug set of studies and filing of the results with the FDA prior to Phase I clinical evaluation. Longevity Biotech has obtained quotes and secured agreements with leading Contract Research Organizations who are ready to initiate these studies.