Type I diabetes is a widespread disease in which insulin producing pancreatic islets are lost, leading to unstable glucose levels. If the islets can be replaced, as with transplantation, then diabetes can be reversed. Human islets have limited utility because of a severe shortage of donors and the need for constant immune suppression to prevent rejection. Pigs could provide an unlimited supply of islets but the rejection would be even more vigorous, necessitating greater immune suppression. Ximerex, Inc. has developed an innovative and proprietary technology known as surrogate tolerogenesis (ST). With ST pig islets can be transplanted without rejection or immune suppression. ST is supported by extensive proof of principle, including pig islets to diabetic monkey transplants with prolonged survival and function without immune suppression. The program was recently reviewed by the NIH commercialization assistance program (CAP). A large panel of experts in business development concluded that the technology does provide a strong competitive advantage and is protected by a robust patent portfolio. However, the regulatory hurdles are substantial, requiring FDA approval for commercialization. Ximerex, Inc. is unlikely to attract private investments until the FDA allows phase I/II trails. Many specific suggestions were made. RFA-OD-09008 (an RC3 BRDG-SPAN pilot program) targets small companies such as Ximerex, Inc. in the """"""""Valley of Death"""""""", i.e. with extensive proof of principle but unable to attrac investors. This proposal takes the steps necessary for initial clinical trials and subsequent investment based development. Specifically, it includes a large definitive pig to nonhuman primate transplant study to establish efficacy of ST to the FDA;recruitment of a chief medical officer for planning clinical trials and interacting with the FDA;and development of facilities and labs for the FDA certified swine source herd and tissue processing. Furthermore, this proposal transforms the program from one based on the founder to a multi-institutional partnership, including the University of Miami-Diabetes Research Institute with their wealth of knowledge and facilities.
While developing our technologies in the field of transplantation, we have measured our progress with both success and failure. Through these trials we have developed a simple idea into what may change our ability to treat disease, putting transplantation of porcine tissues into the limelight. This project fully develops transplantation f pig islets into diabetic patients without rejection of the tissue and without the need for immune suppression. The aims of this project will demonstrate the recent refinements to the technology and lead to initial clinical trials.