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.