Researchers generate motor neurones from people with MND
Research carried out by American scientists who have generated ‘patient-specific’ stem cells from people living with Motor Neurone Disease (MND) holds great promise for advancing MND research.
What does this research mean for people with MND?
These results show that it is possible to generate human motor neurones from someone with MND in the laboratory. Now the feasibility of such studies has been proven, other researchers can use the same techniques.
These human motor neurones can be used to understand the underlying causes of MND and as a screen for selecting drugs that may be beneficial in the treatment of the disease.
Scientists working in MND research need to be able to understand the underlying causes of the disease, before they can develop new treatments to slow it down or stop it. There are many different ways that researchers can study MND in the laboratory based on animal models. However, the best way to understand MND is to understand how human motor neurones affected by MND are different from those from unaffected people. We hope that this new model will greatly increase our knowledge of how motor neurones are affected in MND. This knowledge will be used to develop ways of stopping the neurones from dying.
However, cautioned Dr Henderson "therapeutic use of these cells is probably a long way off. Right now there are safety issues with iPS" he continued "these hurdles need to be overcome before we can think about using the cells to treat disease, but we can start immediately to evaluate them as a tool for drug discovery".
How did the researchers generate these motor neurones?
Two sisters who have a rare, inherited form of MND donated some skin cells for this research. In the laboratory the skin cells were treated with a number of specific chemicals that triggered them to reprogramme into very basic cells that have many of the properties of embryonic stem cells, known as induced pluripotent cells (iPS).
Pluripotent cells have the potential to specialise into any cell type in the body. As Dr Eggan and Prof Henderson are interested in motor neurone disease, they were able to convert the pluripotent cells into motor neurones. Thus the net result was converting a skin cell from someone with MND into a motor neurone from someone with MND.
As well as the motor neurones themselves, scientists are also interest in the support cells around them. The authors of this research paper have also shown that these skin cells can be converted into support cells too. Again, this increases the potential of this model to understand MND and slow its progression.
Do researchers still need to use embryonic stem cells?
Yes, embryonic stem cell research is still needed to understand MND. Although this research has demonstrated that an alternative to embryonic stem cells can be used to generate human motor neurones, Dr Eggan one of the scientists who performed this research emphasized the need to keep all avenues of research open saying "It's essential to note that we couldn't possibly be where we are now without first doing extensive work with human embryonic stem cells (hESC). Further, it will be essential to continue to do work with hESC as they remain the stem cell gold standard"