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Promising MND drug helps slow disease progression and benefits patients physically

Patients on the trial reported better patient mobility and lung function after 12 months

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A phase-III clinical trial of the investigational drug Tofersen in patients with MND caused by the faulty SOD1 gene, has shown it can slow and reduce progression of the disease, according to a statement from the University of Sheffield.

Patients on the trial reported better patient mobility and lung function after 12 months, said the statement.

Researchers from the University of Sheffield Institute for Translational Neuroscience (SITraN) and the NIHR Sheffield Biomedical Research Centre found that, though biomarkers in patients’ cerebrospinal fluid showed improvement at six months, it took 12 months for identification of physical benefits, as per the statement.

It also said that 108 patients took part in the clinical trial, funded by the biotechnology company Biogen, and Sheffield was the major trial site in the UK.

Scientists believe a new genetically-targetted therapy to treat Motor Neurone Disease (MND) could be a turning point for patient care, after the results of a phase-III clinical trial showed significant physical benefits for patients after 12 months.

Researchers from the Sheffield Institute for Translational Neuroscience (SITraN) found that patients with a faulty SOD1 gene – responsible for two per cent of MND cases – noticed that the progression of their symptoms slowed down 12 months after taking the investigational drug Tofersen.

Results of the trial, published in the New England Journal of Medicine, show that biomarkers in patients’ spinal fluid showed a reduction in the SOD1 and neurofilament protein levels after taking Tofersen for six months, suggesting that the treatment successfully hits the therapeutic target and reduces loss of motor neurones which may allow them to start regenerating connections with muscles in the body. However, it took longer for patients to experience reported physical improvements.

Clinicians and scientists hope that this is a first step towards a licensed therapy for MND patients, the statement concluded.

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