In a significant stride toward enhancing patient care, researchers from the Sheffield Institute for Translational Neuroscience (SITraN) have conducted a Phase 3 clinical trial demonstrating remarkable physical improvements in motor neurone disease (MND) patients. The trial involved an investigational drug called tofersen, specifically targeting patients with a faulty SOD1 gene, responsible for 2% of MND cases. While initial findings at 28 weeks did not show significant clinical improvement, extending the trial to 52 weeks revealed notable changes in motor and lung function. Biomarkers in patients' spinal fluid indicated a reduction in SOD1 and neurofilament protein levels after six months of tofersen treatment, suggesting a successful therapeutic impact on motor neurones. Although physical improvements took time, this milestone signifies a potential turning point in MND treatment and opens doors for more efficient clinical trials.
Professor Dame Pamela Shaw, Professor of Neurology and Director of 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.”
“Patients with SOD1 mutations are relatively rare, but this trial is going to change the future of MND trials for patients. Not only can we look at other genes which also cause MND, but we now have a biomarker which we can measure to see if a treatment is working. This is going to make trials much more efficient. In future we may be able to tell in three to six months if an experimental therapy is having a positive effect.”
Professor Chris McDermott, Professor of Translational Neurology at SITraN University of Sheffield and Co-Author of the study, said: “This is the first time I have been involved in a clinical trial for people living with MND where I have seen real benefits to participants. Although tofersen is a treatment for only two per cent 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. The approach used, of reducing proteins harmful in MND, is likely to have wider applications for more common types of MND.”
By successfully slowing the progression of MND and demonstrating the ability to measure treatment effectiveness through biomarkers, this achievement promises more streamlined and faster clinical trials. It not only benefits the relatively rare group of MND patients with SOD1 mutations but also lays the groundwork for similar gene therapy-based approaches for more common types of the disease.
Source: University of Sheffield. "Promising MND drug helps slow disease progression and benefits patients physically". https://www.sheffield.ac.uk/neuroscience-institute/news/promising-mnd-drug-helps-slow-disease-progression-and-benefits-patients-physically. 21/09/22. [Date accessed: 21/09/22].
Edited by: Pulkit Pincha
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