A “promising” new drug has been shown to slow down or even reverse the progression of motor neuron disease (MND).
Findings of a new study into tofersen showed that some participants reported better mobility and lung function after a year of treatment.
Dame Pamela Shaw, a professor of neurology at the University of Sheffield who led the UK part of the international study, says the trial is like nothing she’s come across before.
Having taken part in 25 clinical MND trials, she says this was the first in which patients have reported an improvement in their motor functions.
“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,” she said.
“For me, this is an important treatment milestone.”
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Scientists say the findings, published in the New England Journal of Medicine, are “remarkable” for a disease characterized by “relentless decline”.
Around 5,000 people in the UK have MND, with 2% developing it due to a faulty SOD1 gene, which sees a “misfolded” version of the protein giving rise to the condition.
While the tofersen trial only applies to this small portion of patients, the researchers said their work will “change the future of MND trials for patients”.
The latest study is a global phase III trial, meaning researchers have been examining the drug’s safety and efficacy.
It was funded by biotechnology company Biogen, which involved 108 patients with faulty SOD1 genes from 32 sites in ten countries.
What is motor neuron disease and what are the warning signs?
MND, also known as amyotrophic lateral sclerosis (ALS), is a condition that affects the brain and nerves.
It is a progressive disease, meaning it gets worse over time, and can eventually impair a patient’s ability to walk, talk, eat, breathe, and use their arms and hands.
There’s no cure for MND, and it can significantly shorten life expectancy, but there are treatments to help reduce the impact it has on a person’s daily life.
Adults of all ages can have MND, but it mainly affects people in their 60s and 70s.
Early symptoms can include
– Weakness in your ankle or leg – you might trip, or find it harder to climb stairs
– Slurred speech, which may develop into difficulty swallowing some foods
– A weak grip – you might drop things, or find it hard to open jars or do up button
– Muscle cramps and twitches
– Weight loss – your arms or leg muscles may have become thinner over time
– Difficulty stopping yourself from crying or laughing in inappropriate situations
What is motor neuron disease (MND)?
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