Work Experience with the Research Development team

My name is Kiera Belson and I have just completed three days of work experience at the Motor Neurone Disease (MND) Association for an award called the Youth STEMM Award. This consists of doing different activities and experiences linked to the different STEMM sectors: Science, Technology, Engineering, Maths and Medicine. The work I have done at the MND Association has been linked to the Science and Medicine sectors.

During the time I spent here, I have learnt things about MND as well as researching a technique called induced Pluripotent Stem Cell (iPSC) technology (see below), which has been my main task over the three days. I have also learnt about the Research Development team and what they do at the Association, including management of the ‘UK MND Collections’, a resource of biological samples from people with MND, and the different categories within this: the DNA bank, the cell lines collection and the epidemiology collection.

It has been interesting to discover some of the different things that the Research team do such as deciding which projects to fund, helping researchers with various resources, and informing the public about the different things happening in MND research through media such as blog posts, magazine articles, or posters.

The research I have done here at the MND Association has been very interesting and has given me lots of information about MND and scientific research that I didn’t know before.

My task

My focus has been on iPSCs and how they are being used to research MND and look for potential treatments from the obtained results. I summarised my research below to share with you a bit about what iPSCs are, their history and how they are used in MND research.

Induced pluripotent stem cell (iPSC) technology

iPSCs are cells that are capable of turning into many different cell types. Researchers can use them by reversing adult human skin or blood cells into stem cells which have the incredible ability to become any type of cell in the body. iPSCs are increasingly a more favoured technique used in MND research as once they are made, they offer the chance to create an inexhaustible supply to model human disease which can then be used in research.

This technique was first developed by Shinya Yamanaka who used a virus to deliver four stem cell factors into skin cells, causing them to go into a stem cell state. This discovery that mature adult cells can be ‘reprogrammed’ to potentially give rise to any cell type was science fiction-turned-reality, and could be one of the most significant discoveries of the 21^st century.  In 2012, Shinya Yamanaka was awarded the Nobel Prize for his discovery.

MND researchers use human cells to make iPSCs which are then turned into motor neurones or glial cells (the cells that surround neurones providing support and insulation between them). These stem cells are made from skin cells of people with MND and are then combined with special chemicals that turn them into stem cells. These are then reprogrammed into nerve cells that contain the genetic variations involved in the development of MND, meaning the new cells provide a real model of MND that can be studied in a lab. Researchers can use the new cells to screen potential drugs before they are tested in clinical trials, potentially increasing their chance of success.

How are iPSCs made?

iPSCs are created by adding extra copies of pluripotency genes (genes that are capable of giving rise to several different cell types) to adult skin cells in a culture, by infecting the cells with laboratory viruses that contain the pluripotency gene. After a few weeks, some of the cells revert back to pluripotent cells. While iPSCs cannot currently be used for therapeutic purposes as there is still a lack of evidence that would support stem cell treatment, they could be used to study the mutations of cells with MND to find out more about the disease.

What are the advantages of iPSC technology?

There are many advantages of iPSC technology. Firstly, it can be used to research MND without having to make multiple copies of the gene with a mutation that causes the disease. Secondly, gene editing could be used to correct mutations that may cause MND in the iPSC cells, giving us the opportunity to learn more about the use of this technique for MND. Furthermore, it creates the possibility to turn the cells into many different types such as motor neurones, or astrocytes (a type of glial cell), which allows researchers to study the different cell types in the process of the disease.

We are currently funding the creation of iPSCs from our cell lines and these will be made available to researchers in a few years’ time.

Acknowledgments

Image of human iPS cell colony by Karen Burr. Image of iPSC-derived astrocyte by Nina Rzechorzek.