Advanced therapies
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We’re not just treating multiple sclerosis.
We’re reversing it.
Our gene therapies reboot cells to fight previously untreatable neurological conditions.
Patients regained their ability to walk, run and even dance.
This is the future. And it will be Sheffield made.
Researchers at the University of Sheffield are advancing genetic therapies to give hope to patients and their families.
Advanced therapies signal a new era of medicine. For the first time we have the potential to offer much-needed treatments for devastating neurological diseases that cannot be treated by conventional drug compounds.
Advanced therapies harness the power of stem cells to enable the body to repair itself or use targeted gene therapies to replace or silence faulty genetic sequences in DNA.
Several of my symptoms have now disappeared. I now have the possibility of living a life without MS and contemplating a future without disability.
Colette Beecher
MS patient, 50
Scientists at the Neuroscience Institute at The University of Sheffield have already broken new ground in this field, being the first site in the UK to:
- Demonstrate the use of a genetic therapy to silence the SOD1 gene that can lead to motor neuron disease thus becoming the flagship UK site to deliver the first genetic therapy trial for motor neurone disease.
- Develop a stem cell treatment for multiple sclerosis that has been proven to stabilise the disease and reduce disability to levels never previously seen before in research trials. Many patients in this trial regained their ability to walk, run and even dance as a result.
Colette Beecher, 50, from Wickersley, Rotherham, was diagnosed with MS in January 2011, and has been relapse free for five years after having the MS stem cell transplantation at Sheffield's Royal Hallamshire Hospital in April 2016.
Colette said: “Several of my symptoms have now disappeared – I no longer get spasms that go down my spine when I flex my head forward, and my right leg hasn't given way for three years. I now have the possibility of living a life without MS and contemplating a future without disability."
Together we can pioneer new treatments that radically improve the lives of patients and their families.
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Download a two page summary of our pioneering work in advanced therapies (PDF, 1.3MB)
Our world-leading facilities and networks
Improving patients’ lives
Finding treatments for spinal muscular atrophy (SMA)
SMA is a devastating motor neuron disease where approximately 50 percent of children affected die before the age of two. Our pioneering preclinical work led to the FDA approval of Zolgensma®. A single dose results in babies with type 1 SMA achieving milestones where they previously would have declined.
Treating MND in children
Gene-based therapies paving the way for the treatment of Alzheimer’s Disease
Neurological researchers are investigating the benefits of using advanced gene-therapies to treat people with Alzheimer’s Disease. A new worldwide clinical research trial is currently underway.
Gene therapies for other rare diseases
The University of Sheffield research team is leading on the development of gene therapeutics of other diseases such as hereditary spastic paraplegia (SPG15, SPG47, SPG52), Syngap 1 and Hearing loss.