HHS SBIR RFA-DK-11-024

Type 1 diabetes (T1D) results from the autoimmune destruction of the insulin-producing cells of the pancreatic islets of Langerhans and affects more than one million Americans, usually with onset in childhood or young adulthood. The disease markedly impairs quality of life and shortens lifespan as is associated with numerous complications including blindness, renal failure, painful nerve disorders, cardiovascular disease and amputation.  In addition to its devastating toll in human suffering, T1D and its complications result in significant health care expenditures for families and constitute a major societal economic burden.

 Early identification of T1D risk and the onset of autoimmunity provides the basis for a variety of major ongoing studies seeking to prevent or delay the disease.   Already research on the natural history of the development of T1D in at risk neonates has shown that early identification of those at risk can foster early diagnosis of T1D and avoid life-threatening diabetic ketoacidosis DKA.  Also, clinical trials are currently in progress to identify ways to prevent or reverse the autoimmunity of T1D.  Investigators have used a combination of islet autoantibody positivity, autoantibody seroconversion, biomarkers of genetic susceptibility and beta cell functional assays as criteria to select individuals at high risk of developing T1D.  However, current technology for identification of at risk individuals is costly, requires participation of research laboratories, and may not be suitable for public health screening that would ensue should effective preventative interventions be established.  Methods for more efficient identification of individuals at risk of T1D who may be eligible for preventative intervention would include low cost, high-throughput, accurate and predictive assays/devices that could be used at the point of care level. Application of such technologies could facilitate and expedite testing when effective ways to prevent or delay T1D become available and would be essential for identifying individuals who can benefit from such treatments. Population based screening of individuals would be required as the majority of new cases of T1D (~70-80%) have no affected relatives.

This initiative intends to stimulate and support innovative research at small businesses for the development of low cost novel assays and devices for predictive screening for T1D risk and autoimmunity using genetic, immune and metabolics markers.  The development of these technologies would facilitate recruitment for clinical research focused on identifying environmental triggers of T1D, T1D natural history, and interventions to prevent T1D.  It will also facilitate clinical implementation of measures subsequently proven effective in delaying or preventing T1D in those at risk.  Use of such assays in organ donors could also facilitate provision of autoimmune pancreas to researchers.

Examples of topics relevant to this announcement include but are not limited to:

• Development of techniques or products useful for predicting, preventing or delaying progression of diabetes, including tests for identifying patients at risk, and methods of monitoring disease progression.
• High throughput - Point of care assays (reliable, accurate, cost-effective, highly sensitive/specific standardized having rapid turnaround time) for autoantibody detection and other immune parameters for autoimmune diabetes diagnosis and follow-up. This could also include newborn screening methods.
• Point of care low cost /portable devices for pre-diabetes and diabetes diagnosis.
• Development of methods to measure changes in the immune status that may be used as markers to follow the immune-modulatory activity and beneficial effect (beta cell mass preservation, reduction of inflammation at the target organ, etc) of agents tested in clinical trials for the prevention and/or treatment of T1D.
• Development of non-invasive imaging as well as other methods/biomarkers for the in vivo measurement/ evaluation of pancreatic beta cell mass, function or inflammation for the in vivo diagnosis and prognosis of a pre-diabetic stage and subsequent follow-up.
• Development of high throughput assays based on biologic pathways likely involved in the pathogenesis of diabetes that could be used to develop novel diagnostic systems/platforms.