Biomarkers in type 1 diabetes: application to the clinical trial setting

Biomarkers of type 1 diabetes (T1D) are important for assessing risk of developing disease, monitoring disease progression, and determining responses to clinical treatments. Here we review recent advances in the development of biomarkers of T1D with a focus on their utility in clinical trials.

Measurements of autoantibodies and metabolic outcomes have been the foundation of monitoring T1D for the past 20 years. Recent advancements have led to improvements in T-cell-specific assays that have been used in large-scale clinical trials to measure antigen-specific T cell responses. Additionally, new tools are being developed for the measurement of β cell mass and death that will allow for more direct measurement of disease activity. Lastly, recent studies have used both immunologic and nonimmunologic biomarkers to identify responders to treatments in clinical trials.

Use of biomarkers in the study of T1D has largely not changed over the past 20 years; however, recent advancements in the field are establishing new techniques that allow for more precise monitoring of disease progression. These new tools will ultimately lead to an improvement in understanding of disease and will be utilized in clinical trials.