Human induced pluripotent stem cell-derived beta-cells for drug and toxicity testing

DESCRIPTION provided by applicant Diabetes represents a global health crisis In the United States alone approximately million people of the population suffer from diabetes resulting in direct medical costs of $ billion National Diabetes Statistics Report New medicines that can restore cell function in these patients thereby preventing or alleviating the debilitating consequences of the disease are urgently needed Although immortalized human cell lines have been aggressively sought by pharmaceutical companies as tools to discover such treatments cell lines that accurately model the complex physiology of primary cells have not yet been developed In response to this unmet market need Regenerative Medical Solutions RMS has developed a proprietary protocol to differentiate induced pluripotent stem cells iPSC into pancreatic islet like clusters ILC containing abundant glucose responsive like cells These ILC represent a potentially unlimited source of human like cells for use in drug discovery and testing Using Phase I funding RMS successfully adapted a panel of iPSC lines for use in its protocol and demonstrated that ILC made from these cells closely resemble primary human pancreatic islets in terms of both gene expression and function In the Phase II project proposed here RMS will move four of these iPSC lines forward into product development Each cell line will be thoroughly characterized and banked for large scale production ILC derived from each cell line will be extensively characterized and this characterization will serve as the basis for the establishment of stringent product release criteria Production scale culture protocols will be developed based on RMSandapos s highly successful research scale protocols enabling production of batch sizes that will meet customer needs A first in class cryopreserved cell product will also be developed Finally a suite of highly customizable live cell products geared towards high throughput and high content compound screening will be generated This suite of new products will meet the strong demand among RMSandapos s customers for physiologically accurate cell based tools for the discovery of new medicines to mitigate the diabetes epidemic PUBLIC HEALTH RELEVANCE Regenerative Medical Solutions RMS has developed a new method to convert stem cells into insulin producing cells and has shown that these cells function very similarly to cells isolated from the human body Using this funding RMS will adapt its methods so that it can produce large quantities of stem cell derived insulin producing cells that its customers can use to find new medicines to treat diabetes