Researchers at King's College London report that an experimental oral drug, KCL-286, repaired DNA breaks and reduced inflammation in a mouse model of Alzheimer's. Its efficacy in people is not yet known.
Scientists at King's College London report that an experimental drug first developed for spinal cord injuries showed encouraging effects in a mouse model of Alzheimer's disease. The compound, called KCL-286, helped repair breaks in DNA and reduced inflammation in the animals, two processes that the researchers say can appear very early in the disease. The team argues that this multi-target approach differs from treatments focused mainly on the build-up of amyloid-beta and tau proteins.
According to the researchers, KCL-286 is an orally available small molecule that activates a specific protein in the retinoic acid pathway, a system linked to how the body uses vitamin A. Earlier work by the group had shown that the drug promotes repair of DNA double-strand breaks, in which both strands of the DNA molecule are severed, in research on neuropathic pain. The team had also previously identified molecular pathways shared by acute spinal cord injury and Alzheimer's, which prompted them to test the drug against the disease.
The findings remain preclinical, and the researchers stress that the drug's effectiveness against Alzheimer's in humans has not been established. KCL-286 has, however, already completed Phase 1 human trials of safety and tolerability, which the team says could shorten the road to further clinical testing. Additional studies and human trials will be needed to determine whether it can safely slow the progression of the disease or improve outcomes for patients.
