Sheffield Institute for
Translational Neuroscience

Clinical Research

1. Therapeutic trials of novel neuroprotective agents in MND patients

The Sheffield MND Care and Research Centre is the host centre leading the UK Dementia and Neurodegenerative Diseases (DeNDRoN) Clinical Studies Group for MND clinical trials.

Professor Pam Shaw and her clinical team have already played a key role in multiple national, European and international trials of potential neuroprotective agents. So far, riluzole is the only therapy shown to have a neuroprotective effect. The Sheffield team acted as ‘champions for patients’ by negotiating with UK Health Care providers to include this therapy in the NICE treatment guidelines and make it available to MND patients in the UK.

Find out about our latest collaboration with UK and European partners to investigate the potential of Interleukin-2 in Motor Neuron Disease. News: Ambitious Research project trials promising new therapy for MND.

2. Evaluation of technologies to improve symptomatic care
and outcomes for MND patients

In the absence of better neuroprotective therapies, the SITraN research team recognises the importance of improving symptomatic care and quality of life for MND patients. Professor Shaw’s team was fundamental in providing the evidence for the effectiveness of non-invasive ventilation (NIV) to support breathing in MND patients with respiratory muscle weakness. This has resulted in a new NICE treatment guideline to make NIV generally available for MND patients in the UK, with significant impact on survival and quality of life of
patients with MND.

There are further aspects of symptomatic treatment for which high quality clinical research is needed to provide the evidence to bring such therapies into clinical practice and make them widely available to MND patients. New multicentre trials led from SITraN include the evaluation of new technologies such as diaphragm muscle pacing, cough assist devices, new methods for nutritional management and other assistive technologies.

We are also currently assessing the potential value of measuring changes in muscle volume by whole-body magnetic resonance imaging (MRI) in diagnosing MND and monitoring clinical progression.

3. Developing assistive technologies to improve patient care

Working together with our Sheffield MND Research Advisory Group, the Sheffield team is developing new assistive technologies to meet the specific needs of MND patients. Most recently, Dr Chris McDermott has led the development of a customisable head support collar for individuals with neck muscle weakness (Head-Up), a telehealth system (TiM) to revolutionise access to specialist care for MND patients, as well as a web resource for the use of non-invasive ventilation (NIV). These technologies are all currently being trialled by MND patients.

4. Epidemiological studies to determine risk factors in MND

As yet, no definite risk factors have emerged for MND. Strikingly, well known sportsmen have developed MND implying that a high level of physical activity could play a role in MND, probably coupled with a genetic predisposition. SITraN has recently undertaken a study in collaboration with Professor Carol Brayne, Director of Public Health at the University of Cambridge, and Professor Nick Wareham of the MRC Epidemiology Unit in Cambridge to establish whether a high level of physical exercise is a definite environmental risk factor for MND.

5. Sheffield MND clinical database and biobank

The Sheffield MND Care and Research Centre importantly provides the crucial resources that underpin all our research programmes
and put the Sheffield team in a unique position to make a major
contribution to the search for causes and curative treatments for MND:

  • An extensive clinical database of > 1,500 MND patients with high
    quality clinical patient details collected at regular intervals during the disease course.
  • Biosamples donated by the majority of these patients including DNA, RNA, blood, cerebrospinal fluid (CSF) and skin cells (fibroblasts).
  • Brain and spinal cord material donated by > 220 MND patients at the end of their illness, in the Sheffield Brain Tissue Bank (SBTB).
  • As one of the three hub sites for the Wellcome Trust/MND Association funded national MND biobank, we have access to 1,700 DNA samples and lymphoblastoid cell lines from MND patients and a similar number of controls, as well as family members of patients with familial MND.

The Sheffield MND resources will be invaluable for defining further genetic factors predisposing to MND and for elucidating the interaction between genes and environmental factors. These resources are made available to our collaborators worldwide and have contributed to major discoveries such as the recent discovery of a new gene, TUB4A, associated with MND.