Brain health

MS Stem

Multiple sclerosis (MS) is a complex neurological disease with an unknown cause. For each person, their genetics, lifestyle and environment each play a part in starting the disease, but we do not know how these interact to initiate disease in the brain.

To answer this question, we have established MS Stem, assisted by the MS community. MS Stem is a repository of stem cells. Participants consent to provide blood cells, which we reprogram into stem cells that carry the same genetic code as the blood donor. Stem cells are immature cells that can be pushed to produce any cell type in the body. We are using them to generate brain cells from people with MS, to study and learn which genes trigger MS and how this occurs.

Finding the cause and a treatment for motor neuron disease

Motor neuron disease (MND) is a rapidly progressing neurodegenerative disease that affects the central nervous system. There is no cure or effective treatment, and people diagnosed survive, on average, 3 years.

Our team is at the forefront of international efforts to identify why the disease begins and how it progresses. Our breakthrough research found that MND affects two sets of inhibitory neurons in the brain, so that activity in the brain’s motor cortex is altered, even before symptoms are seen.

Now, our goal is to use this knowledge to develop a treatment for MND. Using stem cells and ‘lab on a chip’ microfluidic devices, we can now grow a brain in a dish, modelling the complex circuitry of the central nervous system. Our research pipeline enables MND trials targeting inhibitory dysfunction, providing a way to identify which patient group is most likely to benefit and deliver a high throughput cellular platform for drug screening.

How does gene regulation affect Alzheimer’s disease?

Alzheimer’s disease has no effective treatment or cure. Our team has been studying brain cells under early threat of disease, to determine how their DNA is packaged and which genes are accessed. We developed and use ‘Targeted DamID’ (or ‘TaDa’), a versatile, cutting-edge DNA profiling tool to study a vinegar fly model of Alzheimer’s disease.

The vinegar fly is a short-lived model organism ideal for genetic manipulations. Using TaDa, we have been able to learn how disease affects the brain cells involved in learning and memory, which are some of the first affected in Alzheimer’s.

By studying brain cells before and after disease symptoms begin, we have found that brain cells rapidly repackage their DNA to switch on protective genes well before symptoms appear.

Our goal is to understand how this early response affects disease progression, and whether it is affected by diet, particularly a healthy diet, which is one of the best-known interventions to delay Alzheimer’s onset.

Stimulating brain repair in clinical trials

Our multiple sclerosis (MS) research aims to take discoveries from the laboratory to the clinic, to improve the lives of people with MS. This includes developing and testing brain repair treatments. In the brain, cells called oligodendrocytes play a critical role, wrapping up and insulating (myelinating) our electrically active nerve cells.

However, for people living with MS, these oligodendrocytes die, and there is currently no treatment to support their replacement.

Our laboratory research found that non-invasive magnetic brain stimulation increases the number of new oligodendrocytes added to the brain.

Our clinical trial is now delivering non-invasive magnetic brain stimulation to people with MS to determine (phase 1) whether it is safe, and (phase 2) whether it can be delivered alongside other MS treatments to promote brain repair and prevent disability.

Multiple Sclerosis Research Flagship

Our vision is to reduce the impact of MS on individuals and the community.

Our mission is to perform high-quality, interdisciplinary, and consumer driven research on the causes, treatment and prevention of MS through an integrated pipeline of research. Conducting high impact, translational research to improve health and benefit to society.

Find out more about the Multiple Sclerosis Research Flagship