New approach for the battle against neurodegenerative diseases
10 March 2011
Research carried out by the Medical Research Council (MRC) and published this week in Science has found a new way of helping human cells deal with damaged proteins in the body, which in turn could open the door for further research that could help prevent neurodegenerative diseases such as Alzheimer’s, Parkinsons’ Huntington’s and amyotrophic lateral sclerosis (ALS).
Accumulation of damaged or ‘misfolded’ proteins is associated with ageing, and impairs the function of our organs, including the brain. This process is central to the development of neurodegenerative diseases and this research could be the first step towards developing drugs that could stop this damage from taking place.
The team, based at the MRC Laboratory of Molecular Biology (LMB), has discovered that the molecule guanabenz, currently used to treat high blood pressure, boosts the body’s natural cellular defence systems against damaged proteins and prevents them from accumulating and becoming harmful. Guanabenz works by prolonging a process known as the Unfolded Protein Response (UPR) and enhances the protective effect of the UPR in cells.
Dr Anne Bertolotti who led the study, says:
“We are delighted by this discovery. The way guanabenz acts is unique and very promising: it selectively targets cells containing damaged proteins, without affecting healthy cells. This is an entirely new and selective approach to correct protein folding defects, which could pave the way for the development of safe drugs, potentially useful for a broad range of diseases. This work shows that wide-ranging fundamental research is crucial in the hunt for new treatments.”
