Portable Gas Perfusion System for Pancreas Preservation

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DESCRIPTION (provided by applicant): Pancreatic islet transplantation (ITx) is an emerging treatment for a subset of Type 1 diabetics for which standard insulin therapies are insufficient. I is a promising alternative to pancreas transplantation, since ITx has significantly less surgical risk and a less toxic immunosuppression regimen. Enhanced organ preservation via the proposed technology has the potential to increase the number of lifesaving ITx while improving outcomes. There have been 840 infusions in 340 recipients performed in North America (most of them in Canada) and 1380 in 775 recipients in Europe. ITx is an approved standard of care in Canada, the UK and a number of European countries. In the US, islet centers are preparing to file for FDA biologics licenses based on the favorable outcomes in the North American clinical trials; once those are granted, the numbers of US ITx are expected to increase substantially. The Giner Portable Pancreas Preservation System (P3S) will be the first complete, portable commercial system utilizing the persufflation organ preservation technique. The patent pending technology produces oxygen by electrochemical generation. The system supplies oxygen via gaseous perfusion ("persufflation") in which humid gas containing oxygen is flowed through the vasculature of the cold stored organ to minimize oxygen deprivation time. Persufflation enhances preservation of the pancreas as well as other organs. In the Phase I/II program, after development and fabrication, 8 portable persufflation units have been in preclinical and clinical use at seven facilities. The units have been utilized in over 55 procedures and over 1000 hours of operation including during seven airplane flights. The P3S has been approved for routine use on Air Canada. In preclinical testing for 24 hour preservation, the human pancreatic islet yield was 76% higher (p<0.003) relative to the current state-of-the-art preservation method at 10 hours; this higher yield would: (1) theoretically translate to 100% of current ITx recipients being insulin-free with a single ITx; and (2) increase the fraction of transplantable islet preparations from 42% to 81%. There is also evidence of enhanced islet quality based on insulin secretion assays. A Canadian clinical trial at University of Alberta, Edmonton started in October 2013 using the P3S. Four of the first four (100%) pancreatic persufflated in the pilot trial resulted in transplantable islet preparations compared to their historical standard of 42% transplantable preparations; also there was a significant increase in viability (as measured by OCR/DNA) at the day of transplant compared to historical control. Two of the three transplanted preparations have resulted in insulin independence for patients at ~ 2 months post-transplant. These early results are extremely encouraging and confirm our preclinical Phase II results. The goal of the Phase IIB program is to advance the technology to clinical implementation. At the end of Ph IIB, we expect to have a medical device that is ready: 1) for filing for 510K clearance in US and US clinical investigational use; and 2) to be sold commercially internationally in Canada, Europe and Australia with a CE Mark. The Ph IIB has 3 aims: 1) design and production of 12 pilot devices meeting international medical device standards; 2) preclinical testing with evaluation of islets from research-grade human pancreatic; and 3) support of clinical testing (study already approved and pilot study initiated) with ITx in Canada.