Irshadgul News report,
Scientists involved in clinical trials say they have found a “promising” new drug that may slow the progression of motor neurone disease (MND).
In global trials, some patients with the faulty SOD1 gene reported that their lungs and mobility were better after a year of taking the Topherson drug.
Topherson is an investigational drug, which means scientists are analyzing its efficacy and safety during trials.
The findings were published in the New England Journal of Medicine, and the scientists said it was “remarkable” for a disease characterized by “relentless decline”.
Dame Pamela Shaw, Professor of Neurology and Director of the Sheffield Institute for Translational Neuroscience (SITRN), at the University of Sheffield, said: “I have conducted more than 25 MND clinical trials and the Tofferson trial is the first trial in which patients reported have improved their motor function.
“I’ve never before heard patients say, ‘I’m doing things today that I couldn’t do a few months ago – walking around the house without sticks, walking up the stairs in the garden, writing Christmas cards’.
“It’s an important healing milestone for me.”
Around 5,000 people in the UK have MND, of whom 2% develop the condition due to a faulty SOD1 gene.
Chris McDermott, Professor of Translational Neurology at SITRAN and study co-author, said: “Although topherson is a treatment for only 2% of those living with MND, we learned a lot in doing this clinical trial. Which will help us do better and faster clinical trials in the future.”
Two-thirds of the participants were randomly assigned to receive eight doses of Tofersan over a 24-week period.
They were assessed to assess motor function in swallowing and speaking, breathing, fine motor skills and gross motor skills.
They also gave samples of spinal fluid so that researchers could measure the levels of proteins associated with MND.
Results showed that the drug did not improve motor control and muscle strength after six months, but patients reported better patient mobility and lung function after 12 months.
Improvements in MND biomarkers were observed in patients’ cerebrospinal fluid at six months.
What is motor neuron disease?
On the NHS website it states: “Motor neuron disease (MND) is an uncommon condition that affects the brain and nerves. It causes weakness that gets worse over time.”
It can affect a person’s ability to walk, talk, use their arms and hands, or breathe.