A new study, led by Prof Siddharthan Chandran and Prof Josef Priller (UK DRI at Edinburgh), has revealed that the essential waste clearance role within microglia, the brain’s resident immune cells, is impaired in motor neuron disease (MND). The study, published in Science Advances, provides a potential new target for future drug discovery.
MND, also known as amyotrophic lateral sclerosis or ALS, is an incurable disease that causes progressive weakness of the muscles due to the degeneration of motor neurons in the brain and spinal cord. More than 1500 people are diagnosed with MND in the UK each year, and tragically the majority pass away within five years. The absence of effective treatments to slow the course of the disease highlights the pressing need to identify new drugs.
Accumulating evidence indicates microglia play an important role in maintaining the health of motor neurons. In this study, researchers from the UK DRI at Edinburgh, the Euan MacDonald Centre for MND Research at the University of Edinburgh, and the MRC Protein Phosphorylation Unit at the University of Dundee, used state of the art stem cell techniques to generate microglia from people with the most common genetic cause of MND (the C9orf72 mutation).