I wonder what will come of this discovery as reported by the BBC
9 October 2013
Last updated at 16:15 ET
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.
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.
The researchers used a compound which prevented those defence mechanisms kicking in and in turn halted neurodegeneration.
The study, published in Science Translational Medicine, showed mice with prion disease developed severe memory and movement problems. They died within 12 weeks.
However, those given the compound showed no sign of brain tissue wasting away.
Lead researcher Prof Giovanna Mallucci told the BBC news website: "They were absolutely fine, it was extraordinary.
"What's really exciting is a compound has completely prevented neurodegeneration and that's a first.
"This isn't the compound you would use in people, but it means we can do it and it's a start."
Her lab is also testing the compound on other forms of neurodegeneration in mice but the results have not yet been published.
A 'landmark' study Commenting on the research Prof Roger Morris, from King's College London, said: "This finding, I suspect, will be judged by history as a turning point in the search for medicines to control and prevent Alzheimer's Disease."
He told the BBC a cure for Alzheimer's was not imminent but: "I'm very excited, it's the first proof in any living animal that you can delay neurodegeneration.
"The world won't change tomorrow, but this is a landmark study."
David Allsop, professor of neuroscience at Lancaster University described the results as "very dramatic and highly encouraging" but cautioned that more research was needed to see how the findings would apply to diseases such as Alzheimer's and Parkinson's.
Dr Eric Karran, the director of research at the charity Alzheimer's Research UK, said: "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."
Discovery 'could hold key to Alzheimer's treatment'
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.
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.
Continue reading the main story
Prof Giovanna Mallucci Lead researcher"What's really exciting is a compound has completely prevented neurodegeneration and that's a first”
The research team at the
university's Medical Research Council Toxicology Unit focused on the
natural defence mechanisms built into brain cells.
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.
Continue reading the main story
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.
Analysis
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.
This process, repeated in neurons throughout the brain, can destroy movement or memory or even kill, depending on the disease.
'Extraordinary'
This process is thought to take place in many forms of
neurodegeneration, so safely disrupting it could treat a wide range of
diseases. The researchers used a compound which prevented those defence mechanisms kicking in and in turn halted neurodegeneration.
The study, published in Science Translational Medicine, showed mice with prion disease developed severe memory and movement problems. They died within 12 weeks.
However, those given the compound showed no sign of brain tissue wasting away.
Lead researcher Prof Giovanna Mallucci told the BBC news website: "They were absolutely fine, it was extraordinary.
"What's really exciting is a compound has completely prevented neurodegeneration and that's a first.
"This isn't the compound you would use in people, but it means we can do it and it's a start."
Her lab is also testing the compound on other forms of neurodegeneration in mice but the results have not yet been published.
Continue reading the main story
“Start Quote
Prof Roger Morris King's College LondonThis finding, I suspect, will be judged by history as a turning point in the search for medicines to control and prevent Alzheimer's disease.”
Side effects are an issue. The
compound also acted on the pancreas, meaning the mice developed a mild
form of diabetes and lost weight.
Any human drug would need to act only on the brain. However, this gives scientists and drug companies a starting point.A 'landmark' study Commenting on the research Prof Roger Morris, from King's College London, said: "This finding, I suspect, will be judged by history as a turning point in the search for medicines to control and prevent Alzheimer's Disease."
He told the BBC a cure for Alzheimer's was not imminent but: "I'm very excited, it's the first proof in any living animal that you can delay neurodegeneration.
"The world won't change tomorrow, but this is a landmark study."
David Allsop, professor of neuroscience at Lancaster University described the results as "very dramatic and highly encouraging" but cautioned that more research was needed to see how the findings would apply to diseases such as Alzheimer's and Parkinson's.
Dr Eric Karran, the director of research at the charity Alzheimer's Research UK, said: "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."
No comments:
Post a Comment
Please leave a comment-- or suggestions, particularly of topics and places you'd like to see covered