Researchers at King’s College London report that KCL-286, an experimental drug first developed for spinal cord injuries, reduced DNA damage and inflammation in mice with Alzheimer’s-like disease. The findings point to a possible approach that targets several processes linked to the disease rather than focusing only on amyloid-beta or tau proteins.
In the study, the drug helped repair DNA double-strand breaks, a severe form of damage in which both strands of a DNA molecule are severed. It also reduced inflammation. Researchers say both processes can occur early in Alzheimer’s disease, though the results so far come from a mouse model and do not establish that the treatment will work in people.
KCL-286 works by activating a protein involved in the retinoic acid pathway, a system associated with vitamin A and the regulation of gene activity. The team investigated the drug for Alzheimer’s after earlier research found that it could promote DNA repair and identified biological pathways shared by spinal cord injury and the disease.
The drug has completed Phase 1 human safety and tolerability trials during its earlier development. That testing does not show whether it is safe or effective for people with Alzheimer’s; further studies and clinical trials will be needed to assess its potential in the disease. KCL-286 is an orally available small molecule.
The research, led by Natasha Hill and colleagues, was published in FEBS Open Bio. The authors describe KCL-286 as a candidate for further investigation because it affected multiple disease-related cellular pathways in the mouse study.
Sources: ScienceDaily.