NIH R01 · 2025
Efficacy and mechanisms of anti-IFN drugs in AGS treatment
Abstract Aicardi-Goutiéres syndrome (AGS) is a severe, often lethal genetic autoinflammatory encephalopathy. It typically has an infantile-onset and results in progressive and profound cognitive and physical disability. There is an urgent need to find an effective therapy for AGS, but currently, there is no FDA-approved therapeutic approach that effectively alters the pathological course of this disease. Clinical trials with IFN pathway inhibitors, such as the Janus kinase (JAK) inhibitors baricitinib and ruxolitinib, have been carried out in AGS; these JAK inhibitors reportedly resulted in improved systemic manifestations. However, their efficacies in treating brain injury, the most…
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