New research has found an experimental cure for Type 1 diabetes that has cured almost 80% diabetic mice included in the study. The findings provide possible hope of curing a condition that affects 3 million American.
The study was presented at The Endocrine Society’s 93rd Annual Meeting in Boston. Principal investigator of the study, Vijay Yechoor, MD, an assistant professor at Baylor College of Medicine in Houston, said, “With just one injection of this gene therapy, the mice remain diabetes-free long term and have a return of normal insulin levels in the body.”
The researchers used their new gene therapy in a non-obese mouse model of Type 1 diabetes. The therapy is done for the purpose of countering the two defects cause this autoimmune form of diabetes: autoimmune attack and destruction of the insulin-producing beta cells by T cells. The first part of the therapy is to genetically engineer the formation of new beta cells in the liver using neurogenin3, a gene that defines the development of pancreatic islets, which are clusters of beta cells and other cells. Along with neurogenin3, they give an islet growth factor gene known as betacellulin for stimulating growth of these new islets.
The second part aims at preventing the rodent’s immune system from killing the newly formed islets and beta cells.
The researchers combined neurogin3 with a gene called CD274 or PD-L1 (programmed cell death 1 ligand-1). This gene inhibits activity of the T cells only around the new islets in the liver and not in the rest of the body, Yechoor explained.
“We want the gene to inactivate T cells only when they come to the new islet cells. Otherwise, the whole body would become immunocompromised,” he said.
The therapy was able to reversed diabetes in 17 of the 22 mice. Diabetic mice that otherwise have a life span of only six to eight weeks were experiencing normal growth and were free of diabetes as long as 18 weeks after injection of the gene therapy, Yechoor explained.
This method of treatment “has the potential to be a curative therapy for Type 1 diabetes,” according to him.