A “remarkable” new drug that slows and even reverses symptoms of motor neurone disease (MND) in a year has been hailed as a “treatment milestone” for those with the progressive condition.
Patients who took monthly injections of tofersen for an international study reported better mobility and lung function. One who was in a wheelchair at the start of the clinical trials is now able to walk without sticks.
Scientists said the findings, published in the New England Journal of Medicine, were “remarkable” for a disease characterised by “relentless decline”.
Dame Pamela Shaw, professor of neurology and director of Sheffield Institute for Translational Neuroscience, at the University of Sheffield, said: “I have conducted more than 25 MND clinical trials, and the tofersen trial is the first trial in which patients have reported an improvement in their motor function.
“Never before have I heard patients say ‘I am doing things today that I couldn’t do a few months ago – walking in the house without my sticks, walking up the garden steps, writing Christmas cards’. For me, this is an important treatment milestone.”
MND affects around 5,000 people in the UK, affecting their ability to walk, talk, use their arms and hands, eat and breathe. Prof Stephen Hawking, the astrophysicist, had the condition, also known as amyotrophic lateral sclerosis, for many decades.