DeNDRoN

Section title:

Motor neurone disease CSG

Introdution:

Chair: Professor Pam Shaw, University of Sheffield

Vice Chair: Professor Nigel Leigh, King's College London

The strategy for developing our clinical research portfolio has been informed by key clinical features of MND including:

Lower prevalence compared to some other neurodegenerative diseases
Average survival of only 2.5 to 3 years
Death in most patients resulting from neuromuscular respiratory failure

In addition, MND represents an ‘orphan’ disease in terms of neuroprotective drug development, but is a good ‘model’ of neurodegeneration, with clear outcome measures. Presently, there is one neuroprotective drug, Riluzole, which has a modest effect in slowing disease progression. Several other therapeutic interventions may ameliorate symptoms during the disease course, including noninvasive ventilation (NIV) which also has the potential to improve survival and quality of life.

Initial priority areas for the MND Clinical Research portfolio are:

Development of new potential neuroprotective agents to slow disease progression is the main priority for MND patients and researchers. The EMINALS study to investigate the effect of Minocycline in slowing disease progression has already been adopted and trials of several other compounds are currently under discussion. We aim to engage the interest of industrial partners in MND as a model of neurodegenerative disease and promote the translation of potential therapies from evaluation in cellular models in academic laboratories.
Development of a standardised national UK database of MND patients: We have procured pump prime funding for this project and plan to develop a web-based, clinical studies database. We aim to facilitate rapid identification and recruitment of eligible patients for trials and to provide valid power calculations in the face of the changing natural history of MND.
The MND DNA Biobank project is jointly funded by the MNDA and the Wellcome Trust. Its key aim is to create a resource which enables identification of the genetic risk factors for MND. Over a 5 year period, it will collect 6,000 DNA samples, high quality clinical information and establish cell lines from cases including familial and sporadic MND, controls and family members. Shortly, a linked epidemiological study will start, as will genetic association and genome wide linkage studies utilising this resource. These studies should identify new genetic susceptibility factors, gene-environment interactions and subtypes of MND.
Brain tissue banking: Studying CNS tissue from MND patients compared to controls is vital in understanding disease pathogenesis and dissecting out subtypes of disease, which in turn may impact upon therapeutics. We plan to develop more effective recruitment processes into tissue donation schemes and address the current difficulties in obtaining tissue from neurologically normal control cases. In collaboration with the Neuropathology and Brain Banking CSG, we will work with the MRC committee to develop improved systems and funding streams for this activity.
Research to continue to improve standards of care will initially focus on the management of respiratory failure. We wish to develop clinical studies to address several issues including: 1. The value of airway clearance devices in improving survival and QOL; 2.The natural history of the disease course following intervention with NIV, including complications that arise and methods to ameliorate these, the impact on carers, and provision of optimal end of life care.

The opportunity to include patients with MND within the activities and supporting resources of DeNDRoN will allow us to apply the clinical research principles successfully used by the cancer clinical studies groups to achieve improved clinical outcomes for MND patients.

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