Our goal is to cure diabetes. To do that, we have successfully developed ways to create new insulin-producing beta cells. This critical advance has enabled the first beta cell replacement clinical trial for type 1 diabetes. We are now focused on devising strategies to effectively protect beta cells from attack by the immune system.
What we are investigating
Our researchers have discovered how to reprogram adult and embryonic stem cells into new beta cells. Now, we are exploring how these beta cells can be effectively transplanted into patients, without being rejected. This will require protecting transplanted cells against immune attack, but also determining the best way to transplant them to retain their full function. HSCI researchers are also investigating:
- How and why the immune attack on beta cells in type 1 diabetes begins.
- How beta cell function is affected.
- How the immune attack can be prevented or stopped.
- How beta cells could be replaced or regenerated.
This work dovetails with the goal of many HSCI researchers, who are seeking to identify “universal donor” cells that can be transplanted without triggering an immune reaction.
What we have achieved so far
Researchers in the HSCI Diabetes Program have:
- Coaxed human embryonic and adult stem cells into becoming pancreatic beta cells.
- Mapped out the full set of proteins at play in mouse and human pancreatic islets.
- Pinpointed the genes involved in partitioning parts of the embryo into organs.
- Clarified how genes are controlling the late stages of beta cell development.
- Identified genes that may help protect beta cells from the immune system attack.
The BAIRT collaboration
We are central players in the Boston Autologous Islet Replacement Program (BAIRT), a unique collaboration that seeks to accelerate a cure for diabetes. HSCI, Brigham and Women’s Hospital, the Joslin Diabetes Center, and the Dana-Farber Cancer Institute are working together in BAIRT to generate clinical-grade, pluripotent stem cells that are suitable for use in patients. This work will enable us to show that beta cells derived from patients themselves can be a safe and effective therapy in regulating blood sugar. BAIRT aims to advance key concepts including:
- Preparing clinical-grade patient-derived beta cells.
- Identifying the optimal site of implantation in the body.
- Testing the best delivery method.
The JDRF New England Center of Excellence
We are also integral members of the JDRF New England Center of Excellence. This cross-institutional collaboration involves diabetes experts from HSCI, Joslin Diabetes Center, UMass Chan Medical School, Dana-Farber Cancer Institute, and the Jackson Laboratory. The group is working towards genetic engineering of beta cells created from pluripotent stem cells to enable their transplantation without the need for immunosuppression. The goal of the JDRF New England Center of Excellence is to deliver a genome modification strategy that will protect beta cells against both autoimmune destruction and transplant rejection.