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Stem cell research for Motor Neurone Disease advancing

1 June 2010
Appeared in BioNews 560

A new research project, which will use human stem cells to artificially create the diseased brain cells affected in Motor Neurone Disease (MND) could pave the way for a cure for sufferers.

The British led team of researchers hopes to gain its insight into MND by recreating the devastating disease in the laboratory.

The team has already taken skin cells from patients with a genetic mutation known to cause MND and chemically re-programmed them into a more versatile stem cell-like state (so called iPS cells, induced pluripotent stem cells). They then grew these iPS cells in Petri dishes into two kinds of adult nerve cells. The same was done with skin cells from healthy adults. By closely studying and comparing these nerve cells over time, the researchers hope to understand how and why cells carrying the specific genetic mutation die off.

The £800,000 study will see teams from Edinburgh, London and New York work together to try to understand how brain cells grow and why specific cells die in MND. The project will be led by Professor Sir Ian Wilmot (the pioneer who cloned Dolly the Sheep) from the University of Edinburgh.

Professor Siddharthan Chandran from the Edinburgh team said: 'This will enable us to ask questions about why the motor nerve cells die, and we can use them to develop novel platforms for the development and testing of new drugs'. He added: 'Slowing down the disease is our first aim, stopping the disease is the second, and the home run would be to repair and restore lost function'.

Dr Brian Dickie, from the Motor Neurone Disease Association which is funding the research, said: 'This international MND Association research programme will allow scientists to perform detailed studies on human motor neurones. As a result, we will be able to home in on the pivotal biochemical pathways that are altered in MND, opening up promising new treatment strategies'.

Dr Dickie concluded: 'This is a highly promising field of research to help increase our understanding of this disease. The outcomes from our programme will have a powerful impact in shaping the future of motor neuron disease research and enhancing future international research collaboration'.

Motor neurone disease is characterised by a steady loss of brain and spinal cord cells that control muscle function. It is incurable with limited treatment options. Severe muscle wasting affects breathing, speaking and swallowing and leads to progressive paralysis and death. In the UK five people die from the disease every day and around half die within three years of diagnosis. Renowned astrophysicist Professor Stephen Hawking is one of the longest surviving individuals with MND.

£800,000 grant to British team in motor neuron disease breakthrough
The Times |  24 May 2010
British scientists launch first stem cell project recreating brain disease
The Guardian |  24 May 2010
MND breakthrough 'could come without clones or human-animal hybrids'
The Telegraph |  24 May 2010
The Press Association |  24 May 2010
28 November 2011 - by Dr Marianne Kennedy 
On 18 November, Richard Grosjean became the first patient to receive a pioneering stem cell treatment in the upper part of the spinal cord. His procedure is part of an ongoing US-based clinical trial aimed at assessing the safety of injecting neural stem cells taken from eight-week-old fetuses into the spinal cords of patients with amyotrophic lateral sclerosis (ALS)...
26 September 2011 - by Dr Lux Fatimathas 
An international team of scientists has identified a genetic defect responsible for familial motor neurone disease (MND). A region on chromosome 9 was found to be expanded in 40 percent of people with familial motor neurone disease. It is hoped that a blood test for this disease will be available on the NHS in the near future...
15 May 2009 - by Heidi Colleran 
By Heidi Colleran: An international team lead by UK researchers at the Centre for Neurodegenerative Research (CNR) at Kings College, London, have discovered a genetic variation that prolongs survival in people affected with motor neuron disease (MND). In a study of over 5,000 people in six countries - almost half of...
3 March 2008 - by Dr Charlotte Maden 
New findings in the research of motor neuron disease (MND) have been published in the journal Science which have greatly furthered understanding of the disease. MND is a currently incurable degenerative disorder in which motor neurons are killed so that muscles in the body are unable to...
16 October 2006 - by Dr Jess Buxton 
US researchers have used human stem cells to lessen the symptoms of motor neurone disease in rats bred to have symptoms of the condition. The scientists, based at Johns Hopkins University, Baltimore, showed that injecting fetal nerve stem cells into the spines of rats with amytrophic...
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