Sheffield Institute for
Translational Neuroscience


Promising MND drug helps slow disease progression and benefits patients physically

A phase 3 clinical genetic therapy trial led by Prof Dame Pamela Shaw and Prof Chris McDermott in the UK to treat the SOD1-linked subtype of motor neurone disease (MND) could be a turning point for patient care, after the results showed significant physical benefits for patients after 12 months.

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Cerebrospinal fluid samples showed that tofersen lowered levels of SOD1 protein and neurofilament markers of neuronal damage in patients compared to placebo. 

This first demonstration of biomarkers of efficacy in a clinical trial for MND are expected to have a great impact on clinical trials in future. 

The results of the study published in the New England Journal of Medicine (linked here) attracted a large amount of media interest:

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