Huntington's disease is an inherited illness that causes profound cognitive and movement problems. It affects 1 in 10,000 people.
Researchers from the Riken Brain Science Institute in Saitama, Japan, used in vitro screening studies that showed a number of different disaccharides, simple sugars, can inhibit aggregation of a brain protein and increased survival in a cellular model of Huntington disease.
They then tested oral administration of trehalose, the most effective of these disaccharides, on a mouse model genetically altered to develop Huntington disease. It decreased polyglutamine aggregates in the brain and liver by about 40 per cent, improved motor dysfunction and extended lifespan by 10 per cent.
"We suggest that these beneficial effects are the result of trehalose binding to expanded polyglutamines and stabilizing the partially unfolded polyglutamine-containing protein," reports the team.
Trehalose is a naturally occurring sugar used as a sweetener in chewing gum and other foods. Generally recognized as safe (GRAS) in the US, it was approved for use in Europe in 2001 with agri-giant Cargill now a major marketer of the product in the region. But the doses consumed by people are likely to differ from those in the study.
"Lack of toxicity and high solubility, coupled with efficacy upon oral administration, make trehalose promising as a therapeutic drug or lead compound for the treatment of polyglutamine diseases," write the authors in an early online study in Nature Medicine (doi:10.1038/nm985).
But they warn that further research is needed to understand how the sugar could be used to benefit humans.