Stem cells are special cells that occur naturally throughout our body and have the amazing ability to turn into other types of cells, such as liver cells, heart cells or in our case, brain cells.

Why do we need a disease model?

A disease model is an experimental system that mimics some or all of a disease’s characteristics. We study the human brain – not only the most complex organ in the body but also the most inaccessible. We can’t easily obtain brain tissue from living patients, and with post-mortem tissue we can study only the end stage of the disease rather than its causes and progression.

So we harness the amazing power of stem cells to enable us to grow living human brain cells in the lab! These disease models allow our scientists to study and understand how a disease works. They also allow testing, identification and design of new treatments.  

Of course, several hundred stem cells growing in a dish is very different from the billions of cells and untold complexity that makes up a brain. That is why it’s important to remember that any findings we make using stem cell models are just a first step, and we need to test findings rigorously in human volunteers too.

How do we turn stem cells into brain cells?

Cells, such as skin or blood cells, are donated by healthy volunteers or people with the neurological conditions we’re investigating, such as MND.  Our lab researchers then use a process called “reprogramming” to generate human stem cells. This process takes the cells back to being ‘pluripotent’ which means a state where they can produce any cell type in the human body.

By applying a precise series of chemical recipes to the stem cells over several months, the researchers can then generate different cell types from the central nervous system, including brain cells. The cells live and grow in plastic dishes covered with a nutrient-rich liquid, and are kept in incubators at 37°C to mimic body temperature. The cells are then ready to be used in experiments.

The experiments investigate how the diseases work, find clues on how to tailor drugs and chemicals for better treatments, and test drugs. We can compare brain cells from a person with say, MND side by side against brain cells from a healthy person. If we see a change in the appearance or function of the brain cells from the person with MS, these direct comparisons can help us decide whether the change is happening because of the disease.

Read more about one of the people doing this research. Meet Dr Bhuvaneish T Selvaraj

Now read about our drug screening research.