Can we use blood to determine whether potential FA treatments are working?

This proposal aims at identifying blood-based biomarkers of FA. The development and inclusion of different types of biomarkers can advance the process of drug development and clinical trials by determining the target engagement of a drug, predicting disease progression rate, drug responsiveness, and/or occurrence of adverse events. An ideal biomarker for FA should, among other characteristics, show a correlation with disease processes in the nervous system or track disease progression and is conveniently obtained in peripheral tissue such as blood. Emerging evidence reveals the presence of inflammation in various tissues in FA, but little is known about its significance and extent. This proposal is aimed at elucidating the role of the peripheral inflammatory process in FA and its cross-talk with inflammation in the central nervous system. The goal is to identify blood-based, non-invasive biomarkers to monitor disease progression. In combination with imaging techniques and other assays of neuronal degeneration, a noninvasive blood-based prognostic biomarker would provide a better sense of the disease progression rate and will open a new window of therapeutic opportunity. The study will define and optimize an inflammatory biomarker panel using gene expression profiles of peripheral blood from 100 FA patients and 50 control subjects.