Alzheimer’s disease participants did not show any serious adverse effects (primary endpoint), and showed improvements in MMSE and plasma amyloid-beta, total tau and UCHL1 (secondary endpoints), though changes on other clinical outcomes were more variable (e.g. ADAS-cognition and amyloid PET).42 Another Phase II trial (NCT04902703) is now ongoing to evaluate safety and efficacy longer-term sargramostim treatment in Alzheimer’s disease. The gene discussed is UCHL1; the disease is early-onset autosomal dominant Alzheimer disease.