Sheffield is a leading international centre for the diagnosis, treatment and research of motor neuron disease (MND) and related conditions. The Sheffield Institute for Translational Neuroscience (SITraN) was established with the support of patients and donors to accelerate the pace of therapy development and improve the lives of people affected by MND and related neurodegenerative conditions such as Spinal Muscular Atrophy (SMA), Parkinson’s, Alzheimer’s disease, and stroke.
Since its opening by Her Majesty The Queen in 2010, SITraN has developed into a leading global facility which is at the forefront of research and expertise for neurodegenerative diseases. SITraN researchers are pioneering new treatments and technologies bringing new hope to patients, carers and families worldwide. In recognition of her services to neuroscience, SITraN Director Professor Pamela Shaw was awarded a damehood by HM The Queen in 2014.
SITraN has an impressive research portfolio spanning from basic laboratory research to pre-clinical drug development and clinical trials. Our team of clinical neurologists, neuropathologists and neuropsychologists work at both SITraN and the Royal Hallamshire Hospital which hosts a major UK MND Care and Research Centre, the Sheffield brain and tissue bank, memory clinic, and stroke in order to link research discoveries directly to improvements in patient care.
Our teams have already made some exciting discoveries that will hopefully result in better diagnosis and treatments for MND, Alzheimer’s, and Parkinson’s disease. We are currently establishing a Centre for Genome Translation to lead the transformation of data from the 100K genomes project into valuable insights into causes and potential new effective treatments for these diseases.
Translational Therapy Pipeline
We have received orphan drug designation for one drug (apomorphine-S) emerging from our MND drug screening programme and for the use of gene therapy in the childhood form of MND known as Spinal Muscular Atrophy (SMA). These therapies are now being developed towards clinical application. The Parkinson’s team has also discovered a promising drug which is already in use to treat liver disease in humans and has the potential to be fast tracked to clinical trials in Parkinson’s patients. Our dementia researchers have uncovered novel pathways that can lead to dementia and have shown that cognitive stimulation can maintain brain connectivity in the elderly.
Clinical Research Programmes
The MND research team led by Prof Dame Pamela Shaw was instrumental in providing evidence for MND treatments to be taken up in the NICE guidelines (riluzole and non-invasive ventilation). They have ensured that neurodegenerative diseases are included in the national portfolio of research and are delivered successfully in the NHS through the Dementia and Neurodegenerative Diseases Clinical Research Network, DeNDroN.
Our clinical teams are conducting clinical trials to provide evidence for new treatments and interventions including cough assist, diaphragm pacing and gastrostomy feeding. Working with patients and carers, they are developing assistive technologies to treat troublesome symptoms boosting the quality of life of people affected by MND including an innovative telehealth system (TiM) and a customisable neck support collar engineered under the lead of the Sheffield MND clinical team in partnership with the NIHR, the MND Association and Sheffield Hallam University.
Recent Impact Stories
Promising drug to slow Parkinson’s disease: Study supports fast track to clinical trials
A new study led by Professor Oliver Bandmann and Dr Heather Mortiboys at SITraN shows promising results for a drug to slow down the progression of Parkinson’s disease. In a collaboration with scientists from the University of York, the team could now confirm the beneficial effects of ursodeoxycholic acid (UDCA) in an animal model. The drug is already approved to treat liver disease in humans and could therefore be repurposed and fast-tracked to clinical trials in Parkinson’s patients saving years of research and hundreds of millions of pounds.
Providing an evidence base for new treatments in MND
A team of MND specialists led by Dr Chris McDermott at the Sheffield Institute for Translational Neuroscience (SITraN), conducted the first randomised controlled UK multi-centre clinical trial to assess the risks and benefits of diaphragm pacing in patients with motor neuron disease (MND). The intervention to alleviate breathing difficulties is widely offered to MND patients around the world on humanitarian grounds since 2011. The DiPALS study has now revealed that diaphragm pacing was not beneficial when used in addition to non-invasive ventilation (NIV). In fact, patients who used diaphragm pacing lived on average 11 months shorter than those who used NIV alone.
Investigating novel pathways that contribute to dementia
Scientists from the SITraN Neuropathology Group have discovered a novel pathway that contributes to dementia in individuals that lack the typical signs of Alzheimer’s disease in the brain. The scientists found that one in five of the elderly with documented dementia lack significant amounts of the classical hallmarks of Alzheimer’s disease in the brain, namely ß-amyloid plaques and neurofibrillary tangles. They found that in these individuals higher levels of DNA damage in nerve cells correlate with cognitive decline. These findings could lead to a novel therapeutic approach for dementia.
Uncovering risk factors for cognitive decline and dementia
A new study by the SITraN Translational Neuropsychology Group has revealed that blood types play a role in the development of the nervous system and may cause a higher risk of developing cognitive decline. The research, carried out in collaboration with the IRCCS San Camillo Hospital Foundation in Venice, shows that people with an ‘O’ blood type have more grey matter in their brain, which helps to protect against diseases such as Alzheimer’s, than those with ‘A’, ‘B’ or ‘AB’ blood types.
Developing new technologies to boost quality of life
Working together with patients and in response to their needs, our clinical team have developed an innovative telehealth system that could revolutionise access to care and support for people with motor neuron disease (MND). The aim of the bespoke monitoring system, provided in form of an app on a tablet computer, is to ensure that aids and assistance can get to patients in time. Weekly updates on mobility and general well-being are sent to the patient’s specialist MND care team to swiftly identify problems and points for action. The telehealth system is currently being trialled in a pilot study.
Read more about “Telehealth in Motor Neurone Disease” (TiM)
New web resource to help with non-invasive ventilation in MND
With the input of patients and carers, the SITraN MND clinical team have developed a new web resource for non-invasive ventilation in MND called myNIV. The resource contains step-by-step guides, videos and practical tips from users to get started, troubleshooting and persevering with NIV. Using NIV has been shown to be life-prolonging in MND, however many patients give up on it early.
Visit the web resource for non-invasive ventilation in MND www.mymnd.org.uk
Training the next generation of scientists
The training of future researchers is one of our highest priorities at SITraN to ensure a long-term focus on research into neurodegenerative diseases. We offer four taught masters courses as a springboard for a career in neurosciences: MSc in Translational Neuroscience, MSc in Clinical Neurology, MSc in Translational Pathology [Neuroscience] and MSc in Genomic Medicine. We also offer PhD studentships which give postgraduate students an opportunity to train in a multi-disciplinary, highly collaborative environment using cutting edge technologies and equipment.
Find out more about our postgraduate opportunities.
Inspiring young minds and engaging the public
SITraN researchers are keen to engage with the public and are doing so on a regular basis. Our scientists are getting involved in British Science Week and Festivals organised by The University of Sheffield such as the Life Festival, Festival of Science and Engineering, and Festival of the Mind. We are working with charities to promote research into neurodegenerative diseases and welcome visitors to tour the labs at specially arranged visits or our public annual Open Day. Our outreach activities include visits from SITraN researchers to schools and colleges, workshops for primary school children as well as tours and A-level workshops for school groups at SITraN. Our new research blog SITraNsmissions gives a behind-the-scenes view into our research directly from our students and scientists.
Patient and Public Involvement (PPI)
SITraN hosts public and patient involvement groups which are actively involved in our research. The Sheffield Motor Neuron Disorders Research Advisory Groups (SMNDRAG) meets on a quarterly basis; its members have been involved in a number of research projects including the new telehealth system, customisable neck collar and the myNIV web resource for people living with MND. SITraN is also host to the South Yorkshire Dementia RAG (SYDEMRAG) and is currently establishing a new PPI group run by Parkinson’s Research UK.