Title : Challenges in drug development for T1D: Model systems and biomarkers
Abstract:
Drug development is often challenged by the available human disease-relevant models that are available to understand targetable mechanisms and biomarkers of disease progression or response. Type 1 diabetes (T1D) is an autoimmune disease that leads to loss of beta cells and eventual dependence on exogenous insulin. The cause of T1D is unknown though there is clear evidence that T-cells mediate much of the beta cell loss associated with the disease. Targeting T-cells or associated immune mechanisms is an obvious path of development, however, the risk of immune suppression, understanding how much or little immune changes are meaningful, and identifying the relevant pathway(s) to target remain unclear. Further, with the approval of teplizumab for the delay of T1D, activities related to curing, reversing, or preventing T1D are increasing, but without biomarkers to track disease progression and regression, the field of drug development is challenged with how to progress.
For drug development to succeed for T1D prevention or delay, access to biomimetic model systems and robust methods to assess response are critical needs for drug development in diabetes. We have developed a novel islet-immune assay in collaboration with Insphero AG to begin to understand and model the immune mechanisms leading to beta cell loss and are continuing to refine this system for drug development to meet one key need. This model has utility in understanding immune-mediated beta cell loss, screening for relevant targetable biology, and testing predictive biomarkers.
Audience take-away:
- Understanding in vitro model challenges and needs in biomarker development will help the audience apply concepts to their own work and be aware of the model availability through InSphero for their own use.
- Designing or building better models and better biomarker assays will improve the long-term success and clinical translatability of their work.
