In an exciting new project, Dr Bhuvaneish T Selvaraj, Emerging Leader at the UK DRI at Edinburgh, will investigate the mechanisms underlying motor neuron disease (MND) using cutting-edge human stem cell technology.
The project will develop the use of so-called ‘nerve-muscle assembloids’, which are three-dimensional structures comprising nerve cells and muscle cells, all of which have been grown in the laboratory from stem cells derived from a human blood or skin sample. The nerve cells and muscle cells grow together to form functioning structures called ‘neuromuscular junctions’, just as they do in the human body. Researchers believe that the degeneration of neuromuscular junctions is a key step in the development of MND.
As the brain is so complex and inaccessible, to be able to re-create neuromuscular junctions in a laboratory is a major step forward for human brain research. Using this new technology, the researchers will be able to compare assembloids generated from healthy people against those from people with MND, to search for differences in how their neuromuscular junctions look and function.
If differences are observed, the researchers will test drugs on the assembloids to try to correct any defects. Later, these drugs might become candidates for clinical trials in people with MND, testing their ability to slow down the progression of the condition.
The researchers will validate their findings in human post-mortem muscle samples generously donated to the Edinburgh Brain and Tissue Bank by people with MND.
This research is made possible by a Diamond Project Award which Dr Selvaraj and the cross-disciplinary project team received from The Humane Research Trust – an organisation whose mission is to advance the diagnosis and treatment of disease in humans by supporting new medical research which does not include the use of animals.