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The Humane Research Trust is funding a research project to advance treatment discovery in motor neurone disease research. Scientists at the University of Edinburgh are using innovative cell culture models to test degeneration reversal techniques. This project will advance understanding of the condition and identify potential treatments, without the use of animals.
Motor neurone disease (MND) is a fatal, rapidly progressive neurological disorder. Unfortunately, there is currently no cure for MND, and 50% of patients die within 3 years of diagnosis. Additionally, despite decades of research, there is only 1 globally licensed drug for MND. This drug only extends lifespan by 2 to 3 months.
MND causes sufferers to lose nerve cells, or 'motor neurons'. These motor neurons are responsible for sending electrical signals to the muscles, to tell them to move. When this neuron-muscle connection is broken, messages stop reaching the muscles. Consequently, people affected by MND gradually lose the ability to move, speak, swallow and ultimately, breathe.
The motor neurons in the spinal cord connect with muscles to form intricate structures called neuromuscular junctions. Scientists think that the degeneration of these junctions could cause MND. By better understanding what causes this degeneration, scientists hope to find out how to slow down disease progression.
Until recently, most studies on neuromuscular degeneration have used animal models due to the lack of valid alternatives. Dr Bhuvaneish Selvaraj and his team of researchers believe that using human models will help them understand degeneration better. “It has recently been shown that using animal tissues might not be relevant. This is because there are notable differences between the neuromuscular junctions of humans and rodents,” said Dr Selvaraj.
The Humane Research Trust is funding Dr Selvaraj to oversee a research project at the University of Edinburgh. He is leading a team of multidisciplinary researchers and clinicians to investigate the causes of MND. The team are seeking out potential treatments using a cutting-edge human stem cell model and patient autopsy samples.
Dr Selvaraj and his research group have developed an exciting new way to grow human neuromuscular junctions in the lab. Researchers convert a donated blood or skin sample into spinal organoids containing motor neurons, or 'mini-spinal cords'. Similarly, they also make muscle organoids or 'mini-muscle' in the lab. Then, they fuse the two components together, which forms a full 'neuro-muscular assembloid'. During this process, neurons from the spinal organoid grow into the muscle organoid and form neuromuscular junctions.
Using this experimental model, the researchers perform experiments to investigate and try to reverse the degeneration of neuromuscular junctions. They compare assembloids generated from cells donated by people with MND against those made from cells donated by healthy people. This work will elucidate differences in the neuromuscular junctions’ structure and function, and in their molecular composition. After, the researchers can test drugs on the assembloids, looking for changes to the neuromuscular junctions on a molecular level.
Additionally, the research group plan to validate their findings from the laboratory on real human tissue. The University of Edinburgh is linked with the Anne Rowling Regenerative Neurology Clinic, granting them access to the Scottish MND Register. This means they can use muscle autopsy samples from people who have agreed to provide samples for research after their death.
It is hoped that this research will enable us to develop a better understanding of neuro degeneration. This important work could help identify therapies which could slow disease progression in patients with MND.
