New treatment in mice could hold potential for Alzheimer’s
Scientists in Leicester test compound designed to slow neurodegeneration.
Posted on 9th October 2013
Researchers at the Medical Research Council (MRC) Toxicology Unit at the University of Leicester have shown success with a potential new treatment for prion disease in mice, suggesting it could also hold benefits for the treatment of other neurodegenerative diseases like Alzheimer’s. The research, funded by the Medical Research Council, is published on Wednesday 9 October in the journal Science Translational Medicine.
Neurodegenerative diseases like Alzheimer’s, Parkinson’s and prion diseases such as Creutzfeldt-Jakob disease (CJD) are all characterised by the build-up of abnormally folded proteins in the brain and the subsequent death of nerve cells. In addition, these diseases also show activation of a cellular defence mechanism known as the unfolded protein response (UPR). The UPR aims to protect cells from the damage caused by wrongly folded proteins, and does this by temporarily shutting down the protein production system in cells.
The researchers had previously shown that prion disease in mice triggered an over-activation of the UPR, causing a long-term shut down in the production of proteins critical for the survival of brain cells. To test this further, they re-activated the UPR in mice with prion disease.
The team treated the mice with a compound designed to target the UPR and prevent protein production from being switched off. They found that the experimental drug prevented the loss of brain cells in mice affected by prion disease and slowed the rate of memory and thinking problems.
The authors suggest that these proteins could be used to aid drug development to help people affected by prion disease and potentially other neurodegenerative diseases such as Alzheimer’s.
Dr Eric Karran, Director of Research at Alzheimer’s Research UK, the UK’s leading dementia research charity:
“Targeting a mechanism relevant to a number of neurodegenerative diseases could yield a single drug with wide-reaching benefits, but this compound is still at an early stage. It will be important for these findings to be repeated and tested in models of other neurodegenerative diseases, including Alzheimer’s disease.
“While Alzheimer’s is the most common form of dementia, other diseases that cause dementia are also characterised by the abnormal build-up of proteins in the brain. If this process is also working overtime in these conditions too, targeting it could be a promising avenue for investigation. However, what is true in animals does not always hold true in people and the ultimate test for this compound will be to see whether it is safe and effective in people with these diseases.”
Read our blog post: Neurodegeneration – a common cure?
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