Researchers at the Medical University of South Carolina (MUSC) are developing a two-part cellular therapy designed to restore insulin production in people with type 1 diabetes, without the use of immunosuppressive drugs. The project, led by immunologist Leonardo Ferreira, Ph.D., has received $1 million in funding from Breakthrough T1D, a global diabetes research and advocacy organization.
Type 1 diabetes affects approximately 1.5 million Americans. The condition causes the immune system to destroy the pancreatic beta cells responsible for producing insulin, forcing patients into a lifetime of glucose monitoring and daily injections. Existing treatments manage the disease; they do not eliminate it.
The Two-Part Strategy
The MUSC-led approach combines two distinct technologies. The first involves generating insulin-producing islet cells from stem cells, creating a lab-based supply that bypasses the chronic shortage of donor tissue. The second involves engineering immune cells called regulatory T cells, or Tregs, to act as protectors for the transplanted beta cells.
Tregs naturally suppress excessive immune responses, preventing the body from attacking its own tissue. Ferreira’s team modifies these cells using chimeric antigen receptors, known as CARs, which direct the Tregs specifically toward transplanted beta cells. The goal is to stop the autoimmune attack that has historically made islet transplants unreliable.
“These awards support the most promising work that can significantly advance the path to cures for type 1 diabetes,” said Ferreira. “This is what Breakthrough T1D believes is the next wave in type 1 diabetes therapy.”
The Problem With Current Transplants
Islet cell transplantation already exists as a treatment option for patients with severe, difficult-to-control diabetes. It works, but it carries two significant obstacles. Donor tissue is scarce, and recipients must take immunosuppressive drugs indefinitely to prevent rejection. Those drugs carry serious side effects and introduce long-term health risks of their own.
By producing stem cell-derived islet cells in the lab, the team addresses the supply problem. By engineering Treg bodyguards to shield the transplanted cells, the team aims to eliminate the need for immunosuppression entirely. If successful, the therapy could be manufactured in advance and made available as a ready-to-use treatment, potentially applicable even to patients who have lived with the disease for years.
The Research Team
Ferreira is working with two collaborators who bring complementary expertise. Holger Russ, Ph.D., associate professor of Pharmacology and Therapeutics at the University of Florida, leads stem cell research focused on generating islet cells for transplantation. Michael Brehm, Ph.D., of the University of Massachusetts Medical School, develops humanized mouse models that allow researchers to study human immune and metabolic responses in a controlled setting.
The current project builds on earlier work funded by a 2021 Discovery Pilot grant from the South Carolina Clinical & Translational Research Institute, which first connected Ferreira and Russ. That foundational collaboration produced the scientific basis for this larger, more ambitious phase of research.
The combination of stem cell biology, immune engineering, and transplant science in a single therapeutic framework is technically demanding. Each component has been explored individually before, but integrating them into a single, drug-free treatment represents a meaningful step beyond where the field currently stands.
Photo by Andrey Matveev on Unsplash
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