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Genetic Engineering of Immune Cells to Advance Cell Therapies for T1D

Researchers from Stanford University School of Medicine have discovered in mouse models that genetically engineering an immune cell can support islet transplantation, a therapy for type 1 diabetes (T1D). Helmsley is supporting efforts to translate this work into human cells.


Funding of this research aims to advance the use of immune cells as targeted therapeutic agents to combat T1D. Such research may eventually allow individuals to forgo receiving insulin through injections or an insulin pump, a process which can be difficult, time-consuming, and can lead to various complications.


Islet transplantation involves the removal of islet cells from a donor pancreas and the transfer of these cells into an individual who has T1D. Islets contain beta cells that produce insulin, which regulates blood sugar levels, and are lost during disease progression. The goal of this procedure is to replace enough islets to produce sufficient insulin for the person to maintain normal blood sugar levels.


The strategy used by the researchers showed that genetic engineering of an immune cell was effective in reducing inflammation and protecting transplanted tissues, including islets. This highlights the adaptability and power of a specific type of immune cell, called T-regs, to suppress the body’s immune response in a highly-targeted way. Additionally, this approach may improve patient tolerance to islet transplantation and be safer than the immunosuppression currently used that broadly suppresses the immune system.


Dr. Seung Kim, Professor of Developmental Biology, Director of the Stanford Diabetes Research Center, and co-author on this study, thinks this approach could be useful for multiple types of cell-based therapies for T1D. “In addition to islet transplantation, we envision that genetic modification and targeting of immune cells to a patient’s own islets before they are lost could protect them — delaying or preventing T1D, or promoting regeneration.”


This method, termed antigen-specific Treg therapy, is derived from CAR-T cancer therapy, a rapidly emerging strategy in which immune cells can be genetically modified to cause an immune response that results in tumor regression and elimination.


Read the full paper at insight.jci.org.