STATUS: Research Stage | CNS-Penetrant Oral Inflammasome Inhibition [CALL OUT BOX]
Alzheimer's Disease is the most common form of dementia, affecting 5.7 million people in the US and 1 in 10 people over 70 years of age, with no satisfactory treatments currently available. It has long been recognized that in AD, amyloid-beta and tau protein tangles accumulate in the brain, triggering inflammation and nerve cell death, which then triggers further inflammation and amyloid-beta accumulation. Biochemically, AD is multifactorial: elevated Alu-RNA, complement, iron, and reactive oxygen species are also present in the brains of patients and all of these factors induce inflammasome activation, triggering caspase-1, IL-1beta, and IL-18. Inappropriate inflammasome activation causes nerve cell death. In animal studies, inhibition of caspase-1 and inflammasome activation alleviates cognitive impairment and neuropathy in an Alzheimer's disease mouse model. Global AD revenue was $3.6 billion in 2017, with potential growth to exceed $12 billion by 2026 as disease-modifying therapies emerge.