Scientists have discovered an experimental drug with a completely new mechanism of action that has the potential to treat Alzheimer’s disease. According to a new study published in the scientific journal FEBS Open Bio, the medication specifically targets the repair of DNA damage in brain neurons — a process considered one of the primary causes behind the development of this severe neurodegenerative disease.
Researchers at the Institute of Psychiatry, Psychology & Neuroscience at King’s College London established through experiments on Alzheimer’s mouse models that a new molecule, named KCL-286, activates a specific receptor (RARβ). At the cellular level, this process triggers a mechanism that helps neurons repair damaged DNA strands. Furthermore, the drug significantly reduced neuroinflammation in the brain, which otherwise accelerates the progression of Alzheimer’s.
The scientists explained that the drug’s action is akin to repairing potholes on a road — once the underlying damage is fixed, the ‘traffic’ within brain cells returns to normal, allowing the entire system to reset and calm down. Researchers suggest that this unique principle of DNA repair could prove effective not only for Alzheimer’s but also for nervous system healing in general and the management of other neurodegenerative diseases.
The drug KCL-286 has already successfully passed Phase I clinical trials in humans, showing a favorable safety profile. Currently, the researchers are working on securing additional funding to test the new approach directly in patients during the next phase, evaluating how effectively it can improve the condition of people living with Alzheimer’s disease.
