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A team of scientists in the UK and Italy has completed new research into motor neuron disease (MND) that could spark the development of new treatments. By comparing mice with fast progressing ALS (amyotrophic lateral sclerosis, the most common form of MND) to mice with the slow progressing form, they found some clues that could help develop new drugs to slow the disease.
ALS accounts for between 60-70% of all cases of motor neuron disease (MND), a serious and incurable disease where nerves in the spine and brain that control movement (motor function) gradually stop working and die. Patients in the later stages of disease may become totally paralyzed and completely lose their ability to walk, talk, eat and breathe.
The team, from the UK's University of Sheffield's Institute for Translational Neuroscience (SITraN) and the Mario Negri Institute for Pharmacological Research in Milan, Italy, report their findings in the journal Brain.
Professor Pamela Shaw, Director of SITraN, says their state-of-the-art Functional Genomics lab allowed the team to use a cutting edge technique called gene expression profiling to examine why the progression of ALS/MND varies in speed, even in the presence of a known genetic cause.
Prof. Shaw says the technology allows researchers to "get inside" the motor neurons and better understand what is happening to cause injury in the nerve cells.
For their study, the team investigated two mouse models of ALS caused by a change in the SOD1 gene, which is also known to cause the disease in humans.
One strain of mice had the rapidly progressing form of ALS, and the other had a form that progresses much more slowly.
Using gene expression profiling, they examined molecular pathways at the point of disease onset in the two types of mice.
They discovered factors that might explain differences in the speed and severity of the two forms of the disease.
The motor neurons in the mice with rapidly progressing ALS showed reduced functioning in the neuroprotection, energy-producing and waste-disposal systems in the cells.
The motor neurons of the mice with the form of ALS that progresses slowly, on the other hand, showed increased protective inflammation and immune responses, and higher activity in mechanisms that protect motor neurons from damage.
Prof. Shaw says:
"We are very excited about the results which have given us some new ideas for treatment strategies which may help to slow disease progression in human MND."
Brian Dickie from MND Association, which supported the study, says:
"These new and important findings in mice open up the possibility for new treatment approaches in man. It is heartening to see such a productive collaboration between two of the leading MND research labs in Europe, combining their unique specialist knowledge and technical expertise in the fight against this devastating disease."
MND affects more than 6,000 people in the UK, and around 30,000 in the US. Most cases are sporadic, but around 1 in 20 has an identified genetic cause.
Earlier this year, researchers in the US writing in Nature Neuroscience suggested that ALS involves cells other than motor neurons.
Written by Catharine Paddock PhD
Copyright: Medical News Today
Not to be reproduced without the permission of Medical News Today.
Transcriptomic indices of fast and slow disease progression in two mouse models of amyotrophic lateral sclerosis; Giovanni Nardo, Raffaele Iennaco, Nicolò Fusi, Paul R. Heath, Marianna Marino, Maria C. Trolese, Laura Ferraiuolo, Neil Lawrence, Pamela J. Shaw, and Caterina Bendotti; Brain first published online 24 September 2013; DOI: 10.1093/brain/awt250; Abstract.
Additional source: University of Sheffield press release 27 September 2013.
Visit our Muscular Dystrophy / ALS category page for the latest news on this subject.
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