Tissue Collection for Disease Modeling
ICOC Funds Committed:
Reprogramming of patient derived somatic cells into iPSCs opens the possibility for personalized medicine and drug testing. Genetic diseases responsible for the manifestation of disease can be “modeled” by re-differentiation of iPSCs into the cell lineages of interest, primarily those affected by each particular disease. As such, patient-derived iPSCs hold promise not only for regenerative medicine but also for the elucidation of complex molecular mechanisms ultimately responsible for pathological manifestation. Disease modeling subsequently presents the possibility for in vitro drug discovery, testing and development of personalized therapies. Many efforts aim to identify the earliest signs of Diabetic Kidney Disease (DKD) so that treatments can be instituted before irreversible renal injury occurs. Considering that Diabetes Mellitus (DM) now accounts for more cases of end-stage renal disease (ESRD) than any other cause of chronic kidney disease (CKD) and that no treatment apart from renal replacement is available, modeling disease with patient-derived iPSCs represent the most innovative approach for the development of novel therapeutics targeting the kidney. We will obtain up to DKD1000 + 100 control human samples suitable for reprogramming into iPSCs. The generated iPSCs will represent an invaluable platform for the study, and treatment of renal pathologies as well as shed new light on our understanding of these complex diseases and the causative role that Diabetes plays.
Statement of Benefit to California:
The California Institute for Regenerative Medicine (CIRM) was established in 2004 with the passage of Proposition 71, which provided $3 billion in funding for stem cell research at California universities and research institutions to advance stem cell research and develop therapies to relieve human suffering. Eight years later, the creation of a culture for the Medicine of the 21st century, as reflected by the many similar initiatives initiated world-wide, is already a clear indication of success. Our position is that curing diseases and perfecting technologies that allow for the development of novel therapies will boost CIRM’s success. The present RFA on Tissue Collection for disease modeling is an example of the far-reaching objectives of CIRM’s initiative, a way of embracing life science disciplines into a multidisciplinary program with the ultimate goal of developing novel therapies. We see three direct, major positive effects for Californians: (1) Patients in California will be privileged once actual therapies are developed and ready to move to the bedside. (2) California will witness the growth of its technological/industrial infrastructure to develop new forms of treatment. (3) California will establish itself as a world-wide reference for the banking and generation of stem cell models of disease. The combination of the above-mentioned factors is powerful and dividends will be generated in the form of revenues from health care delivery and intellectual property.