Funded Studies
Study Rationale: The immune system displays an aberrant pattern of activity in the central nervous system of people with Parkinson’s disease (PD); this pattern includes activation of a danger-sensing signaling pathway called the “inflammasome.” Although short-term activation of the immune system can be beneficial in fighting infections or healing wounds, its long-term activation in PD contributes to the death of nerve cells.
Hypothesis: We hypothesize that the novel proprietary inflammasome inhibitors we have developed will limit the overactivation of the immune system and comprise a new therapeutic approach for PD.
Study Design: We have identified molecules with novel chemical and biological properties that decrease the activity of the inflammasome in cell models that mimic the overactivation of the immune system. We are now optimizing their chemical structures to ensure that they are safe, specific and active in the brain. Following this chemical optimization, we will test our prospective new medicine in preclinical models of PD to determine whether it inhibits the progression of disease signs and the corresponding nervous system pathology.
Impact on Diagnosis/Treatment of Parkinson’s disease: We expect that an inflammasome inhibitor can prevent damage to the nervous system in PD. Such a therapeutic could substantially improve the prognosis for individuals with PD.
Next Steps for Development: This project will bring us to the stage of preparing an Investigational New Drug (IND) application for submission to the U.S. Food and Drug Administration.