A novel, lymphocyte-based treatment for autoimmune and inflammatory diseases.
Inflammation-based disorders include a wide range of diseases such as autoimmune diseases, graft rejection, graft versus host syndromes and inflammation induced malignancies. Despite formidable efforts to develop newer and better agents for treating these conditions, frustratingly few novel drugs have passed muster in clinical trials and reached the market in the last decade. The current treatments for such diseases are symptomatic, non-specific and of a short range. The market continues to eagerly await safe and effective alternatives to existing therapies. Consequently, there is a significant unmet need for a disease-specific and durable therapy. The present technology offers a method to genetically manipulate Regulatory T cells (Tregs) such that their suppressive effect on acute inflammation states is greatly enhanced.
The level of Tregs in the human body is very low and although some suppressive effect of normal Tregs could be shown in animal models, it is rather impractical to obtain an effective therapeutic dose of such cells for clinical treatments. In contrary, treatment with redirected genetically engineered Tregs show more than 10 fold suppressive, therapeutic effect in mice suffering from acute colitis. The outlined technology which was tested in animal models relies on ex vivo modification of the patient’s own Tregs, using DNA encoding chimeric receptor whose recognition unit is engineered to bind the desired antigen. The resulting cells are selected and propagated in vitro and can be then administered to the patient as well be stored frozen for further treatments.