‘Truly remarkable’ new drug slows motor neuron disease

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A “promising” new drug that has been shown to slow the progression of motor neuron 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 participated in a global phase III trial, reported improved mobility and lung function after one year of taking the drug tofersan.

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

The scientists said the findings, published in the New England Journal of Medicine, are “remarkable” for a disease characterized by “continuous decline.”



I’ve never heard patients say, ‘I’m doing things today that I couldn’t do a few months ago’

Dame Pamela Shaw

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.”

MND, also known as amyotrophic lateral sclerosis (ALS), is a condition that affects the brain and nerves.

Progressive disease affects the patient’s ability to walk, talk, use their arms and hands, eat and breathe.

Around 5,000 people in the UK have MND, of whom 2% develop 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 made, 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 tests for patients”.

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.”

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



These latest results provide growing confidence that Topherson has both biological and beneficial clinical implications in people living with SOD1 MND.

Dr. Brian Dickey

All participants were assessed at 28 weeks to measure motor function in four areas: 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.

Patients’ cerebrospinal fluid showed improvement in MND biomarkers at six months.

Tofferson side effects include temporary light-headedness, and some discomfort from having a lumbar puncture – a procedure in which a needle is inserted into the lower part of the spine to administer a medication.

Merritt Kudkowicz, director of the Sean M Healy and AMG Center for ALS at Massachusetts General Hospital in the US, and one of the co-investigators in the trial, described the findings as a “promising development”, adding that the drug has “potential”. To improve the quality of life of people living with MND.

Les Wood, 68, of Thorne, South Yorkshire, was diagnosed with MND 10 years ago and first participated in a Phase III trial in 2016.

He said: “After 12 months of taking the medicine I could actually walk in the house without sticks, I was able to come off some of my pain relievers and I felt great on my own.

“MND is a progressive disease, although my symptoms continue to worsen, I will not live without medication and I know it has made a difference in my quality of life.

“It not only gives us hope, it gives you hope for the future for a lot of people, for my own family too, because motor neurone disease is familial in my case, I feel good, maybe my own family.” It will be beneficial to come on time.”

Dr Brian Dickey, Director of Research at the MND Association, said: “These latest results provide growing confidence that Topherson has both biological and beneficial clinical effects in people living with SOD1 MND.

“They also provide important ‘proof of concept’ that similar gene therapy-based approaches may be helpful for other forms of the disease.

“We are closely following the recent news that Tofersan will be reviewed by US drug regulatory authorities and are in…

Credit: www.independent.co.uk /

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