A team led by Leonardo Ferreira at the Medical University of South Carolina is developing a novel therapy combining lab-made insulin-producing cells with engineered immune cells to protect them. Funded by $1 million from Breakthrough T1D, the approach aims to restore beta cell function without immunosuppressive drugs. This strategy builds on prior research and targets all stages of the disease.
At the Medical University of South Carolina (MUSC), Leonardo Ferreira, Ph.D., is spearheading an effort to transform type 1 diabetes (T1D) treatment. T1D is an autoimmune condition where the immune system attacks the pancreas's insulin-producing beta cells, leaving about 1.5 million Americans reliant on insulin injections, according to the Centers for Disease Control and Prevention. Complications can include nerve damage, blindness, and coma.
The new project, supported by $1 million from Breakthrough T1D, integrates stem cell science, immunology, and transplantation. Collaborators include Holger Russ, Ph.D., from the University of Florida, who specializes in stem cell-derived islet cells, and Michael Brehm, Ph.D., from the University of Massachusetts Medical School, known for humanized mouse models.
The therapy addresses two key challenges in islet cell transplants: donor shortages and immune rejection. Researchers produce beta cells from stem cells in the lab, providing a scalable supply that can be frozen and stored. To prevent rejection, Ferreira engineers regulatory T cells (Tregs) with chimeric antigen receptors (CARs) that target a specific protein on the beta cells, acting as protectors.
"These awards support the most promising work that can significantly advance the path to cures for type 1 diabetes," Ferreira said. The approach avoids immunosuppressive drugs, which pose long-term risks, especially for children.
This builds on a 2021 Discovery Pilot grant from the South Carolina Clinical & Translational Research Institute, which initially connected Ferreira and Russ. In preclinical studies with humanized mice, the protection lasted up to one month, the longest tested so far. Future work will explore extending this duration and improving delivery.
"We're trying to develop a therapy that would work for all people with type 1 diabetes at every stage, even people who have had the disease for many years and have no beta cells left," Ferreira stated. If successful, it could shift T1D from management to cure, with broader implications for regenerative medicine.
"I think this can change how medicine is done," Ferreira added. "Instead of treating symptoms, we can actually replace the missing cells."
