Background & Purpose

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PURPOSE

JDRF aims to catalyze and support innovative studies for the selective delivery of therapeutics (bioactive molecules) to human beta cells, or other major pancreatic cell types, to promote the survival and recover functional beta-cell mass in type 1 diabetes (T1D). Expressions of Interest (EOI) from investigators who would be interested in collaborating with a cross-functional team to help achieve this goal are being solicited. Both validation of existing drug-delivery systems and the discovery of novel drug-delivery systems are of interest.

BACKGROUND

The JDRF Beta Cell Regeneration Program aims to halt the progression of T1D and ultimately achieve a cure through the development of disease-modifying therapies (small molecules and/or biologics) that promote the survival, function, and regeneration of endogenous insulin-producing beta cells (Specific program details can be found here).

Novel targets and pathways have recently been described that are able to promote beta-cell regeneration and survival. However, it has been empirically determined that many of these targets and pathways are not sufficiently specific or unique to the islet/beta cell and may present safety concerns if delivered systemically, when non-pancreatic cellular targets could become deleteriously affected. Targeted drug delivery presents an opportunity to restrict a regenerative or survival therapy to the islet/beta cell and avoid or reduce on target but deleterious effects on tissues or cells outside the pancreas.