Unmet Need
Type 1 diabetes (T1D) affects 1.25 million Americans currently and by 2050, this number is expected to reach 5 million. Though a currently untreatable autoimmune disease, one of the more promising approaches has been a costimulatory blockade of malfunctioning immune cells via administration of CTLA4-Ig. However, its efficacy is impeded by inflammatory factors such as type 1 interferons. Tofacitinib (Tofa) is a known inhibitor of such inflammatory factors. The controlled and localized delivery of this small molecule compound is thus of particular clinical significance.
Technology Overview
A nanostructured lipid carrier (NLC) with low toxicity and favorable uptake of Tofa was developed for drug delivery to antigen presenting cells. Due to the lipid composition, they can be taken up by multiple immune cells, giving them the unique property of accumulating in lymphoid tissues. During in-vitro experiments, NLCs delivered Tofa to mouse antigen presenting cells, preventing maturation and release of inflammatory cytokines. Ongoing in-vivo experiments have shown that short-term administration of NLCs carrying Tofa reduces T1D onset. Overall, Tofa carrying NLCs represent a promising strategy to complement CTLA4-Ig (currently investigated) and enhance the efficacy of antigen specific immunotherapy.
Stage of Development
Long term in-vivo experimentation is ongoing. Future studies will characterize a combination therapy approach with CTLA4-Ig
