Home » Neurology » New drug potential game-changer for MND treatment

Dame Pamela Shaw

Head of Neurology and Director of the Neuroscience Institute

A leading neuroscientist highlights how scientists are making significant steps forward in discovering treatments for neurological disorders — but more can be done.

Professor Dame Pam Shaw discusses the impact of devastating neurological conditions such as Motor Neurone Disease (MND), Parkinson’s disease, Alzheimer’s and Multiple Sclerosis, on patients and their families and carers. 

“I want to see the same momentum and urgency for the treatment of neurological disorders as there is for cancer,” says Shaw, Professor of Neurology at the University of Sheffield and honorary consultant neurologist at Sheffield Teaching Hospitals. 

Flagship research 

However, she believes scientists are on the brink of developing effective treatments for neurological disorders that are becoming more common as people live longer. 

Shaw is Director of the Neuroscience Institute, one of the University of Sheffield’s flagship research centres, which combines expertise in medicine, science and engineering to improve prevention, diagnosis and treatment of neurological, sensory and development disorders. 

Improving outcomes 

Researchers at the Neuroscience Institute recently completed a Phase 3 clinical trial, investigating a rare type of MND (responsible for 2% of MND cases) caused by a faulty SOD1 gene. Following the trial, patients reported symptom progression had slowed down 12 months after taking an investigational drug. 

Shaw describes the trial results as “a game-changer for MND.” 

We can now investigate other genes that cause MND and measure the effects of treatment much quicker.

“Never before have I seen patients say, “I’m getting better; I’m doing things today that I couldn’t do a few months ago.” 

“Patients with SOD1 mutations are relatively rare, but this trial will change the future of MND trials. We can now investigate other genes that cause MND and measure the effects of treatment much quicker,” she adds. 

Promising future 

The Neuroscience Institute is constantly enhancing its capacity to develop better devices for neurological disability and continues to form industry partnerships to develop treatments. 

The University of Sheffield is a leader in gene therapies; a promising approach to treating neurological disorders. A new gene therapy centre (GTIMC) dramatically broadens the scope of gene therapy research in the UK and will translate scientific discoveries into new treatments.  

“We want to strengthen the power of neurosciences to find better solutions for people with neurological disorders,” Shaw concludes. 

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