Wednesday, October 9, 2013
Via the BBC:
The discovery of the first chemical to prevent the death of brain tissue in a neurodegenerative disease has been hailed as an exciting and historic moment in medical research. More work is needed to develop a drug that could be taken by patients. But scientists say a resulting medicine could treat Alzheimer's, Parkinson's, Huntington's and other diseases. The University of Leicester discovery showed all brain cell death from prion disease in mice could be prevented.
The research team at the university's Medical Research Council Toxicology Unit focused on the natural defence mechanisms built into brain cells. When a virus hijacks a brain cell it leads to a build-up of viral proteins. Cells respond by shutting down nearly all protein production in order to halt the virus's spread. However, many neurodegenerative diseases
involve the production of faulty or "misfolded" proteins. These activate the same defences, but with more severe consequences.
The misfolded proteins linger and the brain cells shut down protein production for so long that they eventually starve themselves to death.
It is rare to get cautious scientists keen to describe a study in mice as a turning point in treating Alzheimer's. It is early science, a lot can go wrong between a drug for mice and a drug for humans and the only published data is for prion disease, not even Alzheimer's. So why the excitement?
It is the first time that any form of neurodegeneration has been completely halted, so it is a significant landmark. It shows that the process being targeted has serious potential. If this can be successfully developed, which is not guaranteed, the prize would be huge. In Parkinson's the alpha-synuclein protein goes wrong, in Alzheimer's it's amyloid and tau, in Huntingdon's it's the Huntingtin protein. But the errant protein is irrelevant here as the researchers are targeting the way a cell deals with any misfolded protein. It means one drug could cure many diseases and that really would be something to get excited about.