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VPAC2 Agonists Neuroprotective and Anti-inflammatory Effects in Parkinson's Disease

Study Rationale:                   
The adaptive and innate immune responses play intricate roles in modulating neuroinflammation and may be harnessed for neuroprotection from Parkinson’s disease. We have developed a novel modulator (VPAC2 agonist) that transforms adaptive immune activities from neurotoxic to neurotrophic (supporting growth). This project aims to determine pharmacokinetic and pharmacodynamics parameters of the VPAC2 agonist, including formulation of dose response and blood-brain-barrier penetrance. We will also determine preliminary cardiovascular safety risk factors.  

Hypothesis:
This study aims to determine key mechanisms and safety of a Parkinson’s disease therapeutic drug candidate.

Study Design:
We will perform detailed immunological experiments to determine neuroprotective endpoints. Ultimately, direct protection of neurons will enable confirmation of the disease-modifying potential of the VPAC2 agonist. We will also complete FDA-quality preliminary pharmacokinetic evaluations and key cardiovascular safety studies.  

Impact on Diagnosis/Treatment of Parkinson’s Disease:             
Addressing brain inflammation, such as in this study, has the potential to slow or stop neurodegeneration seen in Parkinson’s and could possibly result in stabilization of symptoms and disease progression.  

Next Steps for Development:
Should this project confirm cardiovascular safety and further determine key inflammatory markers of Parkinson’s disease, our next steps would be to accelerate this program towards clinical evaluation. We would initiate FDA-mandated investigational new drug studies, and clinical trials would follow.


Researchers

  • Scott Shandler, PhD, MBA

    Philadelphia, PA United States


  • Howard E. Gendelman, MD

    Omaha, NE United States


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