An Ultra filtrate perfusion bio artificial pancreas for high-density islet replacement without immunosuppression

DESCRIPTIONprovided by applicantThe Cell SafeTM is a fundamentally new approach in replacement of dysfunctional insulin producing cells in patients with diabetesby transplantation of new islets in a three dimensional scaffold embedded in a highly compatible bioengineered perfusion system that continuously supplies tissue fluidblood ultrafiltrateso that the cells ae protected from immune rejection but have optimal exchange of oxygen and nutrientsThis compact device is totally implanted in the subcutaneous space and the unique design facilitates loadingreloadingand biopsy of cell products using simple needle access through the skinThe pump controlled ultrafiltrate perfusion system supplies oxygennutrientsand flushes metabolic wasteThanks to the immunoisolation environment survival of transplanted islets can be achieved in complete absence of immunosuppressionThis approach could solve the issues in islet transplantationnamely the complications of immunosuppressive medication and scarcity of human donor pancreaseswhich issues have severely limited islet cell transplantation to a select group of patients who suffer from insufficient control of their blood sugar level and hence are at risk for developing diabetes complicationsControl of blood sugar levels is restored in most patients who receive an allograft transplantbut unfortunately long term allograft survival rates are low and the most patients will eventually require retransplant to continue benefiting from the therapySuccess of the Cell SafeTM device has an immediate implication for human islet allotransplantationbut also would facilitate `unlimitedandaposcell sources like xenogeneic porcie islets or stem cell derived insulin producing cellsTaken together this technology presents a major paradigm shift incell replacement therapyThe Phaseproject will evaluate the capacity of the deviceislet reloading schemesand characterize factors in blood ultrafiltrate tht support islet function and survival that was demonstrated in preliminary studiesAt the same timein vitro stress testing of the device will be performed to prepare for clinical manufacturing The Phasewill focus on preclinical pivotal trials addressing safety tolerability and efficacyintended to support regulatory approval of this device with the final aim of meeting the tremendous clinical need for safeeffectiveand durable islet replacement that also enables expanded accessibility of this therapy in diabetes patients PUBLIC HEALTH RELEVANCEThe goal of this project is to develop an immunosuppression free therapy to replace defective insulin production using a highly compatible bioartificial pancreas implanted just under the skin that supports transplanted pancreatic islets using the patientandapos s own tissue fluid for active perfusion to provide oxygen and nutrients and bring secreted insulin in the bodyThis is a fundamentally new approach to replace insulinproducing cells with the potential to reverse diabetes and prevent diabetes complicationsto answer the unmet medical need in typediabetes patients