STATUS: Research Stage | CNS-Penetrant Oral Inflammasome Inhibition [CALL OUT BOX]
Parkinson's Disease is a long-term, progressive, neurodegenerative disease affecting approximately 1 million people in the US, generally occurring in people over 60 years of age. Cell death in the substantia nigra reduces dopamine levels, causing tremors, stiffness, and slow movement. Current medications including L-DOPA and monoamine oxidase inhibitors are off-patent, mask symptoms, and do not prevent progression. US drug sales for Parkinson's disease were $1.4 billion in 2020, while annual direct medical care costs exceeded $25 billion. Multiple neurotoxic elements implicated in Parkinson's disease, including alpha-synuclein, complement, iron, retrotransposons, gamma interferon, and reactive oxygen species, all induce inflammasome activation. Markers of inflammasome activation, specifically IL-1beta, IL-18, and caspase-1, are elevated in the CSF of patients with Parkinson's disease, pointing directly to inflammasome inhibition as a rational therapeutic strategy.