Agency: GTR | Branch: Innovate UK | Program: | Phase: Collaborative Research & Development | Award Amount: 849.60K | Year: 2015
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with no blood-based diagnostic or prognostic test available. The current management of ALS is supportive, palliative, and multidisciplinary with the only approved therapy, riluzole, having a modest efficacy. Recent late stage clinical failures are thought in part to be due to the existence of disease subtypes in ALS. Biomarkers, especially those connected to different disease processes, would be valuable for stratifying individuals in trial recruitment and to measure treatment effect within each group. OBD and partners are seeking to validate a panel of epigenetic markers, obtained through earlier research, to enable the development of a diagnostic and prognostic test for ALS. This test will enable the stratification of ALS patients with fast vs. slow progressing disease for use as a companion test for novel targeted therapeutic approaches, as a trial selection tool and indicator of treatment efficacy. Additionally the test will have value as a standalone prognostic to inform the wider clinical management of patients.