Electrical impedance myography technology for quantitative, at-home muscle assessment in amyotrophic lateral sclerosis and other neuromuscular disorders.

Project SummaryTo improve the efficiencyconvenience and quality of ALS clinical trialswe need better biomarkers for assessing amyotrophic lateral sclerosisALSprogression and response to therapyAll recent ALS trials require patients to visit an ALS clinical trials center everymonths at which time assessments are obtainedincluding limb strengthvital capacityand the ALS functional rating scale revisedALSFRS RscoreHoweversuch infrequent assessments mean that the estimation of the rate of decline for a given patientthe major outcome of interest in clinical trialshas great uncertaintyMoreovervisits to such centers are a major burden for the patients and caregiversmany patients must withdraw as they become increasingly debilitated during the studyIt also means that many potentially eligible people cannot participate because they lack convenient transportation or simply live too far away from a participating tertiary care centerOne approach for both improving our assessments and increasing convenience and encouraging broader enrollment is to perform biomarker assessment on a more frequent basis at homeGiven the advent of the InternetSkypeand wireless technologiesthere has been interest in collecting data at home by caregivers or patientsWhile safety assessmentsmedication reconciliationand blood draws will still be neededthese can be performed by local trained health professionalse gnurses and medical assistantsand consent can be obtained electronicallymaking it possible for the next generation of clinical trials in ALS and other neuromuscular disorders to take placeto a great extentremotelyMoreoversuch proceduresonce in placecould extend beyond clinical trialsto assist in the routine management of patientsto monitor therapy effectivenessincluding the need for dose adjustment or therapy discontinuationMyolexInchas as its major focusthe development and refinement of electrical impedance myographyEIMtechnology for clinical useBuilding on a successful PhaseSBIR study that demonstrated the power of EIM to assess ALS progressionhere we propose to design and develop a device for at home medical monitoringOur focus is on ALSbut this device could be readily used in other disordersincluding muscular dystrophymyositisand deconditioningOur specific aims of this proposed PhaseB study includeTo develop a user friendly at home devicethe mSelfTMfor highly reliableclinical trial quality muscle data collectionTo develop dedicated software and backend information technologyITinfrastructure to ensure ease of useongoing feedback to the userand secure data transfer and storageTo complete a clinical study to assess disease progression with the mSelfTM and its ability to track disease versus standard measuresWith the completion of this workwe will be well positioned to seek FDA approval and will seek to establish this system as a biomarker in ALS and other neuromuscular conditions Project NarrativeTo improve the efficiencyconvenience and quality of ALS clinical trialswe need better biomarkers for assessing amyotrophic lateral sclerosisALSprogression and response to therapyIn this studywe propose the development of the MyolexmSelfTMa muscle measurement tool for home usethat will serve to improve the ability to detect a therapeutic effect of medication and to increase the diversity of participants in clinical trialsUltimatelythe tool may be used as an at home monitoring for individual patient care in ALS and other neuromuscular disordersincluding muscular dystrophy