Drug breakthrough could ‘substantially slow progression’ of MND, scientists say

Scientists have discovered a new medicine that protects nerve cells damaged by motor neurone disease (MND), offering hope of substantially slowing the progression of “one of the cruellest diseases”.

Preclinical studies of M102 showed improvement in movement and nerve function in mice.

Researchers are now hopeful the drug can be tested on people with the neurological condition, for which there is currently no cure.

MND causes the messages from motor neurones – the nerve cells that transmit signals from the brain and spinal cord to muscle – to gradually stop.

This leads to muscles becoming weak and stiff, affecting how patients walk, talk, eat, drink and breathe.

There are around 5,000 people in the UK with MND, and many people diagnosed with the condition usually die within two to five years of the onset of symptoms.

M102 was developed by scientists at the University of Sheffield’s Institute for Translational Neuroscience (SITraN) in collaboration with US biotech company Aclipse Therapeutics.

It works by activating two protective systems inside cells known as NRF2 and HSF1.

These systems help nerves combat stress, reduce inflammation and clear away damaged proteins.

A preclinical study by SITraN, published in the journal Molecular Neurodegeneration, showed 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.

Professor Dame Pamela Shaw, director of SITraN and lead investigator of the study, said: “MND is one of the cruellest diseases, robbing people of their mobility and independence often at an alarming speed.

“We are now at a point where scientific understanding is finally catching up.

“Our discovery of M102 gives real hope that we can substantially slow the progression of this disease.

“The preclinical studies not only showed improvement in movement, gait, and nerve function in mouse models, but it also protected motor neurones grown in the laboratory from damage caused by MND patient cells.”

The team is now hopeful that human trials of the medicine can go ahead.

Dr Richard Mead, senior lecturer in translational neuroscience at SITraN, said: “Turning scientific discovery into real treatments takes teamwork bringing together researchers, clinicians, industry partners, and investors.

“Our collaboration with Aclipse Therapeutics has built that essential bridge between the research laboratory and the clinic. We’re now ready to take the next crucial step: testing M102 in people with MND and moving closer to a treatment that can truly make a difference.”