ritish scientists may have discovered a new way to counteract the life-crippling effects of degenerative brain diseases, according to a new study published in Nature. The scientists inhibited a gene that automatically stops the production of all brain proteins when it detects the buildup of unwanted proteins. These unwanted proteins, which accumulate in the brains of patients suffering from neurodegenerative diseases like Alzheimer's, kill brain cells — but the brain also can't survive without the necessary proteins. The discovery ensures that important proteins continue to be produced, and it could one day lead to a catch-all treatment for a number of brain diseases. Here, a brief overview of the breakthrough:
Why does the brain sometimes stop protein production?
To protect itself. Extensive research has found that a specific type of misfolded protein builds up in the brains of those suffering from Alzheimer's, Parkinson's, or Creutzfeldt-Jacob Disease (CJD), which is a variant of mad cow disease. These abnormal proteins cluster together, "forming dense plaques that clog up the brain and kill brain cells in the process," says Andy Coghlan at New Scientist. When too many of these misfolded proteins are produced, the brain's "protein factories" shut down as a defense mechanism.
Does the brain ever restart production?
In healthy people, when the brain sorts out the mess, the protein factories go back to work. But in those who suffer from CJD (and quite possibly other forms of dementia), production of vital proteins stops forever.
So what did the new study look at?
A team of neurologists from the Medical Research Council laboratory in Leicester, England, genetically engineered mice to have prion's disease, the mouse equivalent of CJD and mad cow disease. Then they isolated a gene responsible for turning these protein factories on and off when there was too much plaque build-up, and injected a protein that essentially blocks the "off" switch. This allowed protein production to carry on normally. "When they prevented cells from shutting down, they prevented the brain [from] dying," says James Gallagher at BBC News. "The mice then lived significantly longer," and neurodegeneration was halted.
Why is this significant?
Alzheimer's, for example, affects more than 18 million people worldwide and is currently incurable. This discovery is appealing, says lead researcher Giovanna Mallucci, because it "could have benefits across a range of disorders." Though the concept is still in its infancy, scientists have "broadly welcomed" the breakthrough, calling it a "fascinating piece of work." The hope is that one day a treatment will allow patients to maintain a high quality of life even if they suffer from a neurodegenerative disease.
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