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Amyotrophic Lateral Sclerosis (ALS), a Motor Neuron Disease (MND) subtype, is a debilitating neurodegenerative disorder affecting the upper and lower motor neurons (UMNs/LMNs), brain stem and spinal cord. This leads to progressive muscular weakness and atrophy, paralysis, and eventually death, usually within three to five years after the onset of symptoms. Decades of failed drug development mean that MND/ALS is still incurable; only two FDA-approved drugs exist (riluzole, and more recently, edaravone), but these only slow down disease progression.

Despite intensive research, there is still no known cure or standard treatment for Amyotrophic Lateral Sclerosis (ALS), a Motor Neuron Disease (MND) subtype. Researchers have traditionally used animal models (usually mice) to screen candidate compounds, but these models are now known to lack physiological relevance to the human pathology, which could limit translational drug development.

Amyotrophic Lateral Sclerosis (ALS), a Motor Neurone Disease (MND) subtype is characterised by the degeneration and death of nerves (motor neurons) in the brain and the spinal cord that control essential voluntary muscle activity. Affecting over 400,000 patients worldwide each year, MND/ALS progressively causes difficulties in speaking, walking, breathing, and swallowing, with the disease eventually being fatal in about a quarter of all patients affected each year.