EP.93Electrical impedance myography goes global: collaborative efforts to advance a promising preclinical and clinical tool for the development of future DMD therapies

Abstract

Improved tools to non-invasively and conveniently evaluate muscle condition and response to therapy in Duchenne muscular dystrophy (DMD) are needed. A tool with analogous application and relatable results in both preclinical and clinical contexts would be of particular interest for identifying translatable therapeutic outcomes. One approach with potential on both fronts is electrical impedance myography (EIM). EIM has been studied successfully in boys with DMD and shown correlation to functional measures, change over time, and response to corticosteroid initiation. It also can be used effectively in animal models. However, its application in mice has only been pursued by a single lab to date, impeding broader adoption and ability to relate preclinical and clinical results. In an example of cross-collaboration among industry, academic, and nonprofit partners, 3 universities, a medical device company, and patient-founded nonprofit Charley's Fund identified the opportunity to explore EIM's potential as a translatable preclinical-to-clinical tool. As an immediate next step, they developed a plan to integrate it into a 2-site, longitudinal study to establish assay selection best practices and comparative natural history of DMD animal models (part of a broader multi-stakeholder research collaboration known as 'Of Mice and Measures'). A 2-day training and planning session involving all parties was hosted at Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, USA. Researchers became versed and competent in current EIM techniques. They also identified technique refinements to enable improved testing and application in DMD models of interest. Next steps will be additional data collection and analysis, including through the 'Of Mice and Measures' study. The goal for this collaboration is to help identify and advance opportunities for expanded EIM application in preclinical DMD drug studies, and ultimately connect to and inform human clinical application.