Replacement Research Strategy
Replacement of functional beta cells by either whole pancreas or islet transplantation from another source has established proof of concept that restoration of functional beta cells along with immunosuppression therapy has the potential to restore insulin independence. This success, however, is hampered by the lack of sufficient human donor cadaver pancreatic organs and the requirement for chronic immunosuppression, which has significant side effects. The Replacement Research portfolio is designed to address the limited source issue by focusing on generating a replenishable source of islets/beta cells or beta cell precursors and obviating the need for immunosuppression by creating a physical barrier through encapsulation to thwart their rejection. Potential solutions to the limited cell source that have been prioritized include use of foreign species sources of islets and the generation of glucose-responsive, insulin-secreting cells or their precursors from alternative sources such as human embryonic stem cells or reprogrammed human non-beta cells. For suppression of rejection of transplanted islets, encapsulation is a solution that could more quickly revolutionize the way people live with type 1 diabetes. We believe that the area of encapsulation is a limitation for many approaches in this area and is therefore the current strategic focus of our efforts in replacement research.
The goal for JDRF in the area of imaging and measurement tools is the clinical translation of technology to reliably assess progression of disease and the effectiveness of new therapies. Thus, JDRF will work to establish technologies to monitor beta cell mass, function, survival, and stress in patients to support beta cell replacement (as well as regeneration) efforts. JDRF is funding basic studies to discover and validate novel tools for beta-cell function and mass, as well as programs to develop imaging probes and tools in preclinical studies. By fostering collaborations between scientists with different expertise directly and through partnerships with various consortia and societies we hope to attract new talent and accelerate progress in this field.
Replacement Research: Top Priority Areas for Fiscal Year 2013
- Encapsulation strategies to allow long-term survival and function of transplanted beta cells/progenitors/islets in the absence of chronic immunosuppression.