Using neurophysiology to guide the development of new treatments for motor system disorders (TRANSLATIONAL EPHYS)

Motor system disorders cause muscle weakness and can be difficult to treat. A potential barrier to testing new treatments can be found in the bridge between measurements made in patients and those performed in research laboratories. For example, different measures of disease are used in those two settings. This increases the uncertainty around the potential of emerging treatments. Neurophysiological measurements record the electrical activity from the brain and nerves in patients, and also in cells in the laboratory. Thus, these measurements may have the potential to provide a solution. To test this, we will undertake sophisticated measurements of brain and nerve function in patients being investigated for muscle weakness. We will also take a blood and skin samples and use stem cell technology to create patient specific brain cells that we can study in the laboratory. Potential patient participants will be identified at the time of referral to the Department of Clinical Neurophysiology. A study invitation letter and participant information sheet will be included with the department appointment ‘ring in’ letter. Participants will then be recruited in clinic. Our aim is to see if the measurements we take in patients and their cells relate to one another. If they do, this approach may provide a unique platform for testing new treatments for a range of different conditions. This study is funded by the Medical Research Council. While there is no anticipated direct benefit of this study to participants, it is hoped that the project will develop an improved platform for testing potential new treatments for motor system disorders.