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Research

Motor Neurone Disease Research & Clinical Trials in Sheffield

The Sheffield Team offers people affected by Motor Neurone Disease (MND) a clinical service, access to clinical trials and research projects.

There are a large number of research projects taking place, this page summarises clinical projects and the scientific projects that patients, carers and family members can get involved in.  For more information on the full scope of our research please visit the Sheffield Institute for Translational Research (SITraN) website: www.sitran.dept.shef.ac.uk

CLINICAL PROJECTS

 

CLINICAL DRUG PROJECTS

 

LEVALS

LEVALS is a clinical drug trial, studying Levosimendan (also known as ODM-109) developed by Orion Pharma.  There is both experimental and clinical evidence that Levosimendan improves diaphragm function thereby supporting respiratory function in people with MND. (Fully recruited)

 

EQUIPMENT DEVELOPMENT

 

Head Up Project

Some individuals with MND develop weak neck muscles, leading to pain and restricted movement. The Sheffield Support Snood project is an evaluation of a collar specifically designed to support the needs of people with MND who experience neck weakness.  The project will run for a year. (Fully recruited)

 

Telemedicine Project

Following successful work in collaboration with patients, the MND care centre and Mylan, UK we have developed the TiM system (Telehealth in Motor Neurone Disease) designed to allow patients to communicate with the MND team from their own homes. Dr Esther Hobson, Dr Christopher McDermott, Professor Shaw and the SITraN team, together with the Sheffield University School of Health and Related Research completed a trial involving 40 patients and their carers funded by the National Institute for Health Research.  The trial has finished and the results are being prepared and will be ready by the end of 2016.  More information is available on this website: http://sitran.org/clinical-studies/telemedicine  (Fully recruited - results pending)

 

RESPIRATORY MANAGEMENT

 

Weakness in the muscles that control breathing is a common problem in MND as the disease progresses and an important area of clinical research in Sheffield.

 

Encore Anywhere -

Helping patients to use non-invasive ventilation

The study involves patients who have just started to use breathing machines (NIV). Half  of the patients will be monitored using a modem attached to their NIV machine.  It collects information about how someone is using the machine (when they used it, how long for and whether there are any problems).  The information is automatically sent by the mobile phone network to the team in Sheffield.  We hope this will help patients use NIV for longer and gain more benefit. The study has already started and will recruit up to 40 patients over 2016.  The study has received support from Philips, who make the breathing support machines.

You can find out more about breathing support machines (NIV) on this website:www.mymnd.org.uk.(Recruiting)

 

SYMPTOM MANAGEMENT

 

Breath Stacking

For some people with MND respiratory muscle weakness makes it difficult to cough and remove the secretions from their lungs, affecting their quality of life.  Breath stacking is a well-established technique that can improve lung capacity and help stimulate an effective cough.  It is simple to perform using a lung volume recruitment bag.

The purpose of the project is to evaluate how easy it is to teach someone to use the device and evaluate how effect it is.  Initially we are recruiting patients from Rotherham and Sheffield (20 in total).

 

A prospective evaluation of secretion burden in patients with MND (Prosec 2)

Poorly controlled oral secretions adversely affect the quality of life of people with MND.  The primary outcome of this study is to determine the reliability of responsiveness of the CSS-MND scale to detect clinically meaningful change in secretion burden over time or in response to treatments given.  If proven to be reliable then it will be used in future studies to determine the best treatments for this problem.

 

NUTRITIONAL MANAGEMENT

 

Difficulties in chewing and swallowing can occur as MND progresses making research into nutritional management valuable. 

 

Gastrostomy

Gastrostomy involves the insertion of a small feeding tube allowing all nutrition requirements to be obtained from liquid foods fed directly into the stomach. There are multiple gastrostomy techniques, each with their own advantages and disadvantages, but no national guidelines for which is the most appropriate for MND. This study aims to assess current practice across the UK and investigate the optimal timing and technique for gastrostomy insertion in MND patients. (Fully recruited - results published)

 

 

 

POPULATION STUDIES

 

Trajectories of Outcome in Neurological Conditions (TONiC) 

Quality of Life (QoL) for people with disabling neurological conditions is affected by a range of factors, which have received considerable previous research attention. TONiC is a national study examining the factors that influence quality of life in patients with neurological conditions. It is one of the largest studies on quality of life in neurological conditions ever performed in the UK. This study aims to have a significant and positive impact on the lives of all patients that suffer from neurological diseases such as Multiple Sclerosis and Motor Neurone disease, regardless of symptoms, stage of illness, age or social status. 

 

Physical Activity in Development of MND

There is evidence to suggest that excessive physical activity in early life can predispose some individuals to MND later on. This study aims to explore the impact of physical activity on the future development of MND. Researchers will use specially designed questionnaires to collect details about physical activity throughout adult life.  Data from patients and individuals who do not have a neurological condition can then be compared to identify any differences between people with MND and without. We hope to improve our understanding of the disease and why it develops, which may ultimately assist in the search for new treatments. (Fully recruited - results published)

 

Epidemiological Investigation of MND

MND does not affect other family members in the majority (approximately 95%) of cases and there are likely to be multiple environmental risk factors which predispose an individual to get MND. This project investigates environmental exposure via a detailed questionnaire and, in conjunction with DNA bank samples, will study the combined effects of lifestyle and genetics that influence the risk of individuals developing MND. (Fully recruited - results published)

 

DEVELOPING NEW TREATMENTS

 

The Sheffield Team are carrying out basic research to develop new treatments for MND and have been involved in a number of major clinical trials.  We are always happy to keep people informed about possible new trials and do our best to recruit as many patients as possible.  You can gain further information about the trials from the clinical staff.

 

ASSESSMENT OF TREATMENTS

 

Methodology Study of Novel Outcome Measures to Assess Progression of ALS

This study’s primary aim is to identify assessment measures that are more sensitive to disease progression in participants with ALS type Motor Neurone Disease and put them in rank order over 6 and 12 months.  The secondary objectives are: To repeat each of these measures to demonstrate they are accurate and reliable; To determine correlations between these measures and changes at 6 and 12 months and standard measures at 18 and 24 months; to obtain biological samples in order to identify if molecular changes are reflected in clinical measures and to further understand previously identified molecular biomarkers of disease progression for incorporation into future clinical studies. A biomarker is a biological indicator of disease, a measurable characteristic which may be used as an indicator of a condition for example: DNA extracted from blood samples.  This study runs over two years and includes seven study visits and four telephone calls in the first year and three telephone calls in the second year.

This study is sponsored by Biogen and further information can be found via: https://clinicaltrials.gov/ct2/show/NCT02611674

If you think you might be interested and would like further information, please contact Consultant Neurologist Dr Tom Jenkins via the study nurse on 0114 2222264 or h.wollff@sheffield.ac.uk  (Recruiting)

 

DIAGNOSTIC STUDIES

 

Is ALS/FTD Neuropsychologically distinct from FTD?

Amyotrophic Lateral Sclerosis (ALS) also known as Motor Neurone Disease and Frontotemporal dementia (FTD) are two brain diseases.  ALS is a physically disabling neurodegenerative condition due to physical weakening of the muscles, whereas FTD affects behavioural, reasoning and judgement of an individual.  It is known that some people with ALS develop FTD and vice versa, indicating a link between the two conditions.

The link, as to why some people with ALS develop FTD and vice versa, whereas others do not, is not fully understood.  The above study therefore aims to improve the understanding of the relationship between patients with FTD/ALS and those with FTD  by measuring changes in their behaviour, reasoning and judgement.

 

MUSCLE ENERGY project-

The aim of the MUSCLE ENERGY project is to develop imaging biomarkers in MND. A biomarker is a measurement used to help diagnosis, monitor disease progress or predict outcome. Biomarkers are very important in conditions like MND in order to help test new medicines over affordable timescales, and can also help us understand how a disease develops. At present, no useful biomarker exists in MND. In this project, patients and healthy volunteers have a magnetic resonance imaging (MRI) scan either of their whole body to look at the muscles, or a specialised scan of the brain called spectroscopy to measure energy levels. This is compared with measures of muscle strength and electrical tests of the power of the muscles. We are currently recruiting people to help with this research. If you think you might be interested and would like further information, please contact Dr Tom Jenkins, Senior Lecturer in Neurology to Professor Pamela Shaw on 0114 215 9101 or email Thomas.Jenkins@sth.nhs.uk  (Recruiting)

 

NiSALS

People who are diagnosed with MND often have an MRI scan of their brain and cervical cord (the part in the neck area) as part of their diagnostic work-up, to exclude other causes of their symptoms.  NiSALS is an international collaboration of neuroscience centres involved in research in MND.  People with MND are asked for their consent to use the MRI scans obtained at the time of diagnosis for research purposes.  If they agree, the anonymised scans are sent together with standardised clinical data to an international data repository based at the University of Jena, Germany.  Researchers can apply for permission to use this large data resource obtained from people with MND from many different countries to test ideas about the way the disease works.  The opportunity to study large numbers of scans results in a higher chance of being able to detect subtle changes of potential importance to further our understanding of how the disease works. (Recruiting)

 

LABORATORY PROJECTS

 

GENETICS: DNA BANK

Genetic research is vitally important in understanding the causes of MND and in Sheffield we collect DNA samples to allow this crucial work to go ahead. The aim of the DNA Bank is to collect clinical histories and blood samples from 100’s of patients, their partners and family members. This vital resource is then used to gain a deeper understanding of the disease process and enhance the study of genetic factors.

You can aid this project by donating a blood sample from which we can extract your DNA for addition to the Bank and use to further our understanding of what causes MND.

 

 

 

 

THE MOLECULAR BASIS OF MND

In order to develop better treatments for MND we need to understand the molecular basis of the disease and what causes motor neurones to deteriorate and die.

 

Brain and Spinal Cord Donation

Projects using human brain and spinal cord tissue are essential to understanding the biochemical changes that cause damage and thus the symptoms of MND.  Tissue donation is a similar process to that of organ donation. We welcome donations from people who do not have MND as well as people with the disease. By comparing these tissues researchers are able to study the damage caused by MND. Such information could lead to new diagnostic tests and strategies for treatment.

 

 

 

Skin Cell Donation

We cannot directly study living motor neurones from humans so we grow human skin cells from MND patients and healthy people (for comparison) in the laboratory. Skin cells have the same genetic make-up as the motor neurones from skin cell donors and we can use them to study the molecular basis of the disease and for testing potential new therapies. Participating in this study involves providing a small skin sample, please ask the clinical team to find out more.

 

 

 

BIOMARKER DISCOVERY (Recruiting)

We are studying biological fluid samples from MND patients and healthy controls to identify markers that could be used to aid diagnosis. We hope that particular changes in fluid composition may help us to understand how MND arises, how to diagnose the condition at an earlier stage and to understand responses to treatment.  We currently study two fluids as detailed below:

 

Cerebrospinal Fluid (CSF)

Cerebrospinal fluid is the liquid that surrounds the brain and spinal cord. We are studying changes in the composition to help understand the biochemical changes happening in MND.

 

Blood

Blood has the advantage of being an easily accessible fluid to examine. The aim of this project is to identify MND specific, blood-based biological ‘fingerprints’ that can be useful in diagnosis, prognosis prediction and disease monitoring.

 

 

Patients can now sign up to the UK clinical trials gateway to register their interest in clinical trials by clicking on the below link.  Registration can be specific to a condition and researchers can then contact registered individuals to participate in future studies.

 

https://www.ukctg.nihr.ac.uk/sign-up/

 

 

We are funded by the Sheffield Institute Foundation (www.sifoundation.com), the MND Association and many other organisations/charities.  The Sheffield Team are committed to discovering more about MND and looking for new treatments.

  

CAN I HELP with research into the cause and cure of motor neurone disease?

If you are interested in participating in any of these research projects, your clinical care team and Sister Theresa Walsh can provide more details (telephone: 0114 222 2266, email: Theresa.Walsh@sheffield.ac.uk).

 

No-one who visits clinic should feel under any pressure to take part in research projects.

 

You can also get involved with the research process by joining the Sheffield MND Research Advisory Group, a committee of patients, carers and family members who inform and advise researchers at SITraN. The group aims to get your views included in our research. For more information click here.

 

Further information on research being carried at Sheffield is available on the SITraN website: www.sitran.dept.shef.ac.uk

 

The MND Association also provides a good source of information on research: MNDA association research pages

and clinical trials: MNDA research pages - treatment trials

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