Recent advances in research conducted at Harvard Stem Cell and Regenerative Biology, Harvard Stem Cell Institute and later further developed at Semma Therapeutics, demonstrated that large quantities of insulin-producing cells can be generated from human iPSC. Since human iPSC can be derived from a simple blood sample from any patient, this technology now allows for generation of unlimited number of insulin-producing cells in a personalized manner. Other clinical research has shown that normal glycemic control can be restored in type 1 diabetes patients when they are transplanted with enough insulin-producing cells. The Boston Autologous Beta Cell Therapy [BAIRT] Program was formed between Brigham and Women Hospital (BWH), Dana Farber Cancer Institute (DFCI), Harvard Stem Cell Institute (HSCI), Joslin Diabetes Center (Joslin), and Semma Therapeutics; this group aims to use these approaches to derive patient-specific iPSC lines, manufacture clinical grade insulin-producing cell products, and perform autologous cell transplants to treat diabetes patients. As part of the BAIRT program, the goal of the proposed project is to establish a cGMP standard operating protocol to derive iPSC lines from freshly collected human blood. The protocol has been developed by HSCI in collaboration with DFCI as well as input from other partners in the Boston Autologous Beta Cell Therapy Program. It is expected during the project period DFCI will establish the capacity and know-how to begin deriving and banking cGMP-grade patient-specific iPSC lines from blood samples collected from diabetes patients. Joslin and BWH have begun the recruitment of suitable candidates with diabetes resulted from pancreatectomies, so that research subjects will be available to provide their cells for iPSC derivation. The project will facilitate the testing and development of assays to characterize and qualify iPSC products intended for autologous cell therapies. This valuable information will prove crucial toward developing personalized cell therapies, a long-sought goal in regenerative medicine.

Public Health Relevance

While tools for insulin therapies for diabetes management continue to improve, the development of beta cell replacement therapies for diabetes is expected to improve the quality of life and more precise blood glucose control. The use of patient- specific stem cells and personalized therapy may pave the way toward a practical cure for insulin-dependent diabetes.

National Institute of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
High Impact Research and Research Infrastructure Cooperative Agreement Programs—Multi-Yr Funding (UC4)
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Special Emphasis Panel (ZHL1)
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Arreaza-Rubin, Guillermo
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Harvard University
Anatomy/Cell Biology
Schools of Arts and Sciences
United States
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Pope, Benjamin D; Warren, Curtis R; Parker, Kevin Kit et al. (2016) Microenvironmental Control of Adipocyte Fate and Function. Trends Cell Biol 26:745-755