IMMUNOISOLATION BIOCAPSULES FOR PANCREATIC ISLET CELLS

描述(申请人提供)：这项第二阶段研究资助申请的目标是开发一种含有产生胰岛素的胰岛细胞的生物胶囊植入物，然后在体外和体内模型中测试植入物，为与药物合作伙伴进行产品的临床开发奠定基础。第一阶段计划的结果表明，纳米孔膜可以通过允许葡萄糖、营养素和胰岛素通过，同时限制免疫球蛋白和其他可能攻击和破坏胰岛素产生细胞的成分进入，从而有效地免疫保护悬浮在基质中的异种胰岛素产生细胞。第二阶段计划将开发一种适用于在小动物和大动物模型上进行临床前测试的原型植入物，如果成功，该植入物有望用含有胰岛细胞的植入物取代胰岛素注射，该植入物可以根据患者的需求产生胰岛素，保持正常的血糖水平。与其他胰岛细胞移植不同，这种方法可以利用来自猪或人类干细胞的潜在大量胰岛细胞，而不需要任何免疫抑制治疗。植入物将保持移植的胰岛细胞与接受者的免疫系统隔离。它将是耐用的、可充电的，如果出现问题，还可以拆卸。

DESCRIPTION (provided by applicant): The objective of this Phase II research grant application is to develop a biocapsule implant containing insulin producing islet cells and then test the implant in vitro and in vivo models to set the stage for clinical development of the product with a pharmaceutical partner. Results from the Phase I program demonstrated that nanopore membranes can effectively immunologically protect xenograft insulin producing cells suspended in a matrix by allowing passage of glucose, nutrients and insulin while restricting entry of immunoglobulins and other components that may attack and destroy insulin producing cells. The Phase II program will develop a prototype implant suitable for preclinical testing in small and large animal models, if successful, this implant promises to replace insulin injections with an implant containing islet cells that produce insulin in response to the patients demands, maintaining normal levels of blood glucose. Unlike other islet cell transplantation, this approach can make use of a potentially large supply of islet cells from pigs or human stem cells without the need for any immunosuppressive therapy. The implant will keep transplanted islet cells sequestered from the recipient's immune system. It will be durable, rechargeable and, if problems develop, removable.