Sheffield Researchers Discover Potential Breakthrough Treatment for Motor Neurone Disease

In a significant breakthrough, researchers at the University of Sheffield's Institute for Translational Neuroscience (SITraN) have discovered a new drug called M102 that protects nerve cells damaged by motor neurone disease (MND). Preclinical studies of M102 demonstrated improved movement and nerve function in mice, leading the researchers to express optimism that the drug can now be tested on people with this neurological condition, for which there is currently no cure.

MND causes the gradual deterioration of motor neurones, the nerve cells that transmit signals from the brain and spinal cord to the muscles. This leads to weakening and stiffening of the muscles, affecting patients' ability to walk, talk, eat, drink, and breathe. Around 5,000 people in the UK are living with MND, and many diagnosed with the condition usually die within two to five years of the onset of symptoms.

The team at SITraN, in collaboration with US biotech company Aclipse Therapeutics, developed M102 to activate two protective systems inside cells known as NRF2 and HSF1. These systems help nerves combat stress, reduce inflammation, and clear away damaged proteins. The preclinical study, published in the journal Molecular Neurodegeneration, showed that M102 slowed the progression of MND and preserved muscle function in mice. It also protected motor neurones grown in a lab from damage caused by MND cells.