Preclinical studies have demonstrated the drug's effectiveness in improving movement and nerve function in mice, prompting researchers to prepare for the next phase of trials to test it on humans.
Motor neurone disease is a rare neurological disease that causes progressive damage to the motor neurons responsible for transmitting signals from the brain and spinal cord to the muscles, leading to muscle weakness and stiffness, and affecting the ability of patients to walk, talk, eat, and breathe.
Scientists at the Institute of Translational Neuroscience at the University of Sheffield (SITraN), in collaboration with the US biotechnology company Aclipse Therapeutics, have developed the new drug which works by activating two protective systems within cells known as NRF2 and HSF1, which help reduce inflammation, combat stress, and eliminate damaged proteins.
The study revealed that the drug slows the progression of the disease and preserves muscle function in mice, and also protects laboratory-grown motor neurons from disease-related damage.
Professor Dame Pamela Shaw, director of the SITraN Institute and lead researcher on the study, said: "Motor neurodegenerative disease is one of the most devastating diseases, robbing sufferers of their mobility and independence at an alarming rate. But we are finally beginning to see real results from scientific progress in understanding this disease. The discovery of M102 offers new hope in significantly slowing its progression
She added that the team is currently planning to begin clinical trials on humans following the
encouraging results shown in the initial studies.Dr. Richard Mead, Senior Lecturer in Translational Neuroscience at the SITraN Institute, said: "Our collaboration with Aclipse Therapeutics has enabled us to build a vital bridge between the laboratory and the clinic, and we are now ready for the next crucial step towards effective treatmentfor motor neuron disease."
The study was published in the journal Molecular Neurodegeneration.