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A preclinical study from Karolinska Institutet offers hope for treating severe neurodegenerative diseases with an existing drug. The study suggests that the leprosy drug clofazimine may be effective in the treatment of Huntington's disease. The research group examined whether existing drugs could reduce the toxicity of so-called polyQ proteins. These proteins are found in patients with certain hereditary neurodegenerative diseases, including Huntington's disease, for which there is no cure. Screening hundreds of drugs, they found that the leprosy drug clofazimine reduces the toxicity of polyQ proteins and restores mitochondrial function in zebrafish and worms. The finding supports the previous hypothesis...

Testing in the lab at Sanford Burnham Prebys Medical Discovery Institute. Credit: Sanford Burnham Prebys Medical Discovery Institute Phase 2 clinical trial could begin immediately for clofazimine, an FDA-approved drug on WHO’s List of Essential Medicines. A Nature study authored by scientists at Sanford Burnham Prebys Medical Discovery Institute and the University of Hong Kong shows that the leprosy drug clofazimine, which is FDA approved and on the World Health Organization’s List of Essential Medicines, exhibits potent antiviral activities against SARS-CoV-2 and prevents the exaggerated inflammatory response associated with severe COVID-19. Based on these findings, a Phase 2 study evaluating...