Investigational drug that targets DNA damage help could treat Alzheimer's disease

by Wiley

edited by Stephanie Baum, reviewed by Andrew Zinin

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Accumulation of DNA damage in the brain's neurons may contribute to the development of Alzheimer's disease. New research in FEBS Open Bio demonstrates the therapeutic potential of a drug that targets this process.

Using a mouse model of Alzheimer's disease, investigators found that treatment with KCL-286, a molecule that activates the retinoic acid receptor-beta (RARβ), enhanced the repair of DNA damage in neurons. Activation of the receptor triggers a pathway that ultimately causes a protein complex to bind to DNA and promote the expression of certain repair genes. KCL-286 also reduced neuronal inflammation and abnormal brain immune activity, processes thought to contribute to Alzheimer's disease development and progression.

"We think of the drug as repairing potholes in a road—once the damage is fixed, normal traffic can flow again and the system settles down. By repairing the underlying damage, we can allow the system to reset," said corresponding author Jonathan Corcoran, Ph.D., professor of neuroscience at the Institute of Psychiatry, Psychology & Neuroscience at King's College London. "This principle may extend beyond Alzheimer's to nerve repair and neurodegeneration more broadly."

Corcoran noted that phase 1 clinical trials have already established a favorable safety profile for KCL-286 in humans. With appropriate funding, investigators can next test whether this approach delivers meaningful benefit to patients.

"The opportunity is immediate, and the science is ready to advance," he said.

More information

Treatment with KCL-286, a first-in-class retinoic acid receptor-b (RARb) agonist, ameliorates neuronal DNA damage and inflammation in a mouse model of Alzheimer's disease, FEBS Open Bio (2026). DOI: 10.1002/2211-5463.70284

Key medical concepts

Alzheimer's DiseaseDNA Damage

Clinical categories

NeurologyClinical pharmacology Provided by Wiley Who's behind this story?

Stephanie Baum

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Citation: Investigational drug that targets DNA damage help could treat Alzheimer's disease (2026, July 8) retrieved 8 July 2026 from https://medicalxpress.com/news/2026-07-drug-dna-alzheimer-disease.html This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.