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The drug has previously proven safe-to-use for humans.
A research team from King’s College London has found a way to combat Alzheimer’s early by repurposing a drug originally designed for spinal cord injuries.
This experimental drug, KCL-286, targets early brain damage and inflammation. Because it has already passed human safety tests, development could move exceptionally fast.
According to King’s College London, the treatment targets a pathway linked to how the body uses vitamin A. Professor Jonathan Corcoran explained how this speeds up clinical progress.
“KCL-286 is a first-in-class, orally bioavailable small molecule that has already successfully cleared Phase 1 human safety and tolerability trials. This will dramatically cut down the traditional multi-year timeline required for new drug development,” Corcoran said.
Snapping the rope
While most therapies focus on clearing late-stage proteins, KCL-286 repairs physical breaks in the brain’s genetic code. Corcoran compared this damage to a severe fracture.
“DNA double-strand breaks are like a rope snapping completely in two, rather than just fraying at the edges. We found that KCL-286 promotes repair of these breaks, allowing us to target a key feature of Alzheimer’s disease,” he explained.
Dr Maria Goncalves, who managed the drug’s development, believes this dual action is crucial. She noted: “Our findings demonstrate that KCL-286 not only targets DNA damage but also reduces inflammation, two processes that occur very early in Alzheimer’s disease progression. This highlights its potential as a disease-modifying therapy rather than simply addressing symptoms.”
Healing the mind
The researchers discovered that spinal cord injuries and Alzheimer’s actually share the same molecular pathways. This allows one drug to target both issues.
Researcher Natasha Hill stressed that this versatile approach is exactly what patients need, stating: “To develop an effective treatment for Alzheimer’s disease, we need to tackle multiple aspects of the disease. KCL-286 was able to target multiple disease-relevant cellular pathways, some of which are initiated very early in the disease course.”
Though currently tested only in mice, the drug’s safety profile gives scientists a massive head start. They are now working to see if these benefits translate to humans.
