Potential treatment for neurodegenerative diseases found in drug repurposing
Researchers at SciLifeLab have identified a potential treatment for Polyglutamine (PolyQ) diseases. Offering hope for patients with, for example, Huntington’s disease. These diseases are characterized by CAG repeat expansion and progressive neurological decline and pose significant challenges due to lacking therapies. However, a recent discovery might change that.
The research community has long been looking for effective treatments for neurodegenerative diseases, such as Huntington’s disease. Despite their efforts, effective treatments remained elusive. Recent developments in drug repurposing may offer a solution.
Using chemical screening, scientists found clofazimine, an anti-leprosy drug, as a promising PolyQ disease treatment. It mitigates toxicity associated with the Huntington’s disease protein, in lab and animal models (Zebrafish and Caenorhabditis elegans worms). Clofazimine stimulates mitochondrial biogenesis via PPARγ (Peroxisome Proliferator-Activated Receptor gamma) activation, providing insight and potential treatment for PolyQ diseases.
“Our work illustrates the capabilities of drug repurposing to find potential new uses for old drugs. It is however important to bear in mind that ours is a preclinical work, and that the drug that we found has some hurdles that need to be overcome for it to be useful in mammals,” says KI professor and SciLifeLab researcher Óscar Fernández-Capetillo.
While further research is needed to confirm clofazimine’s efficacy and safety in clinical settings, this discovery represents significant progress toward overcoming these diseases.
The authors want to acknowledge the collaborating laboratories of José Lucas, Karolina Pircs and David Vilchez, the zebrafish facility led by Lars Brautigam. And the funding agencies that supported the work.
