King's College London neuroscientists have shown that KCL-286, a drug originally developed and safety-tested for spinal cord injury, repairs DNA damage and curbs inflammation in a mouse model of Alzheimer's disease. Because the compound already cleared Phase 1 human safety trials for its original indication, researchers say it could move into Alzheimer's clinical testing far faster than a drug starting from scratch.
The study, led by Professor Jonathan Corcoran with first author Natasha Hill, found that KCL-286 promotes repair of DNA double-strand breaks — damage Corcoran compared to "a rope snapping completely in two" — by activating a protein in the retinoic acid pathway, which the body also uses to process vitamin A. Dr Maria Goncalves noted the drug tackles both DNA damage and inflammation, two processes that occur very early in the disease, rather than targeting only amyloid or tau, the two hallmark proteins most current Alzheimer's drugs focus on.
The findings, published in FEBS Open Bio and funded by the Medical Research Council and Wellcome Trust, add KCL-286 to a small pool of repurposed compounds that could reach patients sooner precisely because their safety profile is already established.