‘Truly remarkable’ new drug slows motor neuron disease

A “promising” new drug which has been shown to slow down progression of motor neurone disease (MND) could be a turning point for patient care, according to scientists involved in clinical trials.

Some patients with a faulty SOD1 gene, who took part in the global phase III trial, reported better mobility and lung function a year after taking the drug tofersen.

It is an investigational drug, which means researchers have been examining its safety and efficacy closely during the clinical trials.

Scientists said the findings, published in the New England Journal of Medicine, are “remarkable” for a disease characterised by “relentless decline”.

Dame Pamela Shaw, professor of neurology and director of Sheffield Institute for Translational Neuroscience (SITraN) 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, also known as amyotrophic lateral sclerosis (ALS), is a condition that affects the brain and nerves.

The progressive disease affects a patient’s ability to walk, talk, use their arms and hands, eat and breathe.

Around 5,000 people in the UK have MND, with 2% developing the condition due to a faulty SOD1 gene.

The phase III trial, funded by biotechnology company Biogen, involved 108 patients from 32 sites in 10 countries.

All patients had the defective SOD1 gene, where a “misfolded” version of the protein is created, giving rise to the condition.

While MND patients with SOD1 mutations are relatively rare, the researchers said their work will “change the future of MND trials for patients”.

Chris McDermott, professor of translational neurology at SITraN and co-author of the study, said: “Although tofersen is a treatment for only 2% of those living with MND, we have learned much in doing this clinical trial that will help us do smarter and faster clinical trials in the future.”

In the trial, two-thirds (72) of the participants were randomly assigned to receive eight doses of tofersen over a 24-week period, while the remaining 36 people received eight doses of a placebo.

All participants were assessed at 28 weeks to measure motor function across four areas: swallowing and speaking, breathing, fine motor skills, and gross motor skills.

They also gave samples of spinal fluid so the researchers could measure 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 were seem in the MND biomarkers in patients’ cerebrospinal fluid at six months.

These latest results provide mounting confidence that tofersen is having both a biological and a beneficial clinical effect in people living with SOD1 MND

Dr Brian Dickie

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