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Scientists have identified a promising experimental drug that appears to repair DNA damage and reduce inflammation in the brain—two processes believed to play a key role in the earliest stages of Alzheimer’s disease.
The findings, published in the journal FEBS Open Bio, center on a compound called KCL-286, an oral drug developed by researchers at King’s College London.
In a mouse model of Alzheimer’s disease, the treatment reduced harmful DNA damage in neurons and calmed inflammatory activity in the brain, raising hopes that it could one day help slow the disease itself rather than simply manage symptoms.
Jonathan Corcoran, professor of neuroscience at King’s College London and a co-author of the study, told Newsweek that the biggest obstacle to testing the drug in Alzheimer’s patients is no longer science, but funding.
“KCL-286 has already successfully completed Phase I human safety trials in healthy participants,” he said. “The drug is completely ready to be given to Alzheimer’s patients today.”
Corcoran said a small proof-of-concept trial involving around 20 patients could provide an initial answer within a year if funding were secured, showing whether the drug activates the same DNA-repair pathways in people as it does in laboratory models.
Alzheimer’s disease is most commonly associated with the buildup of amyloid-beta and tau proteins in the brain.
While several recently approved treatments target amyloid, their benefits have been modest, prompting researchers to investigate other biological changes that occur earlier in the disease process.
One emerging target is DNA damage.
Researchers say neurons in people with Alzheimer’s can accumulate DNA double-strand breaks—serious injuries in which both strands of the DNA molecule are broken.
These breaks may contribute to genomic instability, impaired cell function and, ultimately, the loss of brain cells seen in Alzheimer’s disease.
The new study found that KCL-286 enhances the brain’s ability to repair that damage. The drug activates a protein known as retinoic acid receptor-beta (RARβ), which triggers cellular pathways involved in repairing DNA.
Scientists also found that treatment boosted levels of BRCA1, a protein best known for its role in DNA repair.
Beyond repairing DNA damage, the drug reduced neuroinflammation, another hallmark of Alzheimer’s.
The researchers observed improvements in the appearance of microglia and astrocytes—immune and support cells in the brain that can become abnormally activated during neurodegenerative disease.
Corcoran said repairing DNA damage could represent a fundamentally different approach to treating Alzheimer’s.
While existing therapies largely focus on clearing amyloid plaques, KCL-286 aims to help brain cells repair their internal machinery and continue functioning normally.
“For families, this could mean moving away from merely managing symptoms toward a true disease-modifying therapy,” he said. “It has the potential to delay disease progression, slow down memory loss, and—most importantly—preserve a patient’s cognitive independence for longer.”
Reference
Hill, N., AlMuallim, H.Y.O., Maddock, E., Hobbs, C., Clarke, E., Goncalves, M.B. and Corcoran, J.P.T. (2026). Treatment with KCL‐286, a first‐in‐class retinoic acid receptor‐β (RARβ) agonist, ameliorates neuronal DNA damage and inflammation in a mouse model of Alzheimer’s disease. FEBS Open Bio. doi:10.1002/2211-5463.70284.
Contact Newsweek editors on this story: Kara Dolman and Emma Lee-Sang
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