A research breakthrough offers new hope for people who develop a range of incurable brain diseases, it was announced last night.
British scientists have successfully found a chemical that can halt the effects of prion diseases, such as Creutzfeldt-Jakob disease, it was revealed.
However the drug has serious side-effects in laboratory mice – and is unlikely to be suitable for human patients yet, researchers warned.
The research, conducted at Leicester University, was reported in Science Translational Medicine.
The mice in the study lost about a fifth of their weight and started developing diabetes.
Researcher Professor Giovanna Mallucci said: "We’re still a long way from a usable drug for humans – this compound had serious side effects.
"But the fact that we have established that this pathway can be manipulated to protect against brain cell loss first with genetic tools and now with a compound, means that developing drug treatments targeting this pathway for prion and other neurodegenerative diseases is now a real possibility."
Professor Hugh Perry, of the Medical Research Council, which backed the research, said: "Despite the toxicity of the compound used, this study indicates that, in mice at least, we now have proof-of-principle of a therapeutic pathway that can be targeted.
"This might eventually aid the development of drugs to treat people suffering from dementias and other devastating neurodegenerative diseases."
An oral treatment targeting the unfolded protein response prevents prion neurodegeneration and clinical disease in mice. Science Translational Medicine 9 October 2013
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