Scientists discover new Alzheimer’s drug that repairs DNA damage and reduces brain inflammation
Scientists have discovered that a drug originally developed for spinal cord injuries can tackle several key features of Alzheimer’s disease, including DNA damage and inflammation. Already tested for initial safety in humans, the experimental treatment could offer a faster path toward therapies that target the disease at its roots.
Key points
- King's College London scientists found KCL-286 repaired DNA breaks and cut brain inflammation in mice with Alzheimer's-like disease.
- KCL-286 has already passed Phase 1 human safety and tolerability trials, which may speed further clinical testing.
- The drug activates a protein in the retinoic acid pathway, linked to how the body processes vitamin A.
Scientists at King's College London have identified an experimental drug, KCL-286, that may address several damaging effects of Alzheimer's disease at once. In a mouse model of the disease, the drug helped repair breaks in DNA and reduced inflammation in the brain, according to research reported by ScienceDaily.
KCL-286 was originally developed for spinal cord injuries and works by activating a protein in the retinoic acid pathway, a biological system tied to how the body processes and uses vitamin A. The drug is a small molecule that can be taken by mouth.
Prior safety testing could speed path
KCL-286 has already completed Phase 1 human safety and tolerability trials as part of its earlier development. The article states that this could significantly shorten the path toward further clinical testing, though the drug's effectiveness against Alzheimer's in humans has not yet been established.
The findings remain preclinical, and additional studies and human clinical trials will be needed to determine whether the drug can safely slow disease progression or improve outcomes in patients.
KCL-286 is a first-in-class, orally bioavailable small molecule that has already successfully cleared Phase 1 human safety and tolerability trials.
— Professor Jonathan Corcoran
What the researchers said
Professor Jonathan Corcoran of King's College London said the drug's earlier Phase 1 clearance "will dramatically cut down the traditional multi-year timeline required for new drug development." Dr. Maria Goncalves, who project-managed the drug development, said the findings show KCL-286 targets DNA damage and reduces inflammation, two processes that occur very early in Alzheimer's progression.
The research builds on earlier work by the same team that identified molecular pathways shared by acute spinal cord injury and Alzheimer's disease, and on findings that KCL-286 promoted repair of DNA double-strand breaks in research involving neuropathic pain.
