Study Rationale: We are developing novel therapeutics to reduce brain inflammation and enhance the survival and function of brain cells in Parkinson’s disease (PD). Studies over the past decade have revealed that a protein found on the surface of a type of brain cell called microglia produces a toxic local environment that increases brain cell loss. We are working to better understand the role that this protein, called potassium channel 1.3, has in these processes so we can develop treatments that block this protein from exerting detrimental effects in PD.
Hypothesis: We hypothesize that blocking potassium channel 1.3 selectively and safely will reduce brain inflammation and prevent neuronal loss in PD and perhaps other neurodegenerative diseases as well.
Study Design: We are studying potassium channel 1.3 in human microglial cells that are grown in the laboratory and in a new animal model of PD that we are using to monitor the activity of human microglia inserted into a rodent brain, where they produce much of the same pathology as we see in PD. We will then block this channel, or eliminate it, and study the resulting effects on microglial cell behavior and gene activity. This data will allow us to understand how this protein works in microglia, as well as in other cells from people with PD.
Impact on Diagnosis/Treatment of Parkinson’s disease: The work we conduct with support from MJFF will expand our understanding of how PD affects brain function and quality of life, and it will accelerate the development of novel therapeutics for PD.
Next Steps for Development: We plan to bring a potassium channel 1.3 blocker to Phase 1 safety testing in healthy volunteers, followed by testing in people with PD, in the next 18-24 months.