They’ve Done it in Mice… Now They Plan to Cure Type 1 in Humans

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Researchers at Children’s Hospital in Pittsburgh have started an FDA-approved phase 1 test in humans of a lab procedure that successfully reversed type 1 diabetes in mice.

In the original procedure, scientists extracted dendritic cells from the blood of  a diabetic mouse. Dendritic cells are immune cells that identify foreign substances and spur infection-fighting T cells to defend the body. Recent research has shown that in mice with diabetes, dendritic cells mistake islet cells in the pancreas for foreign invaders, then wrongly incite attacks upon them—proof of the autoimmune nature of type 1 diabetes.

The scientists “enhanced” the extracted cells with “blockers” that inhibit T cell response, then injected the treated cells back into the mouse. Over several weeks, the beefed-up dendritic cells were able to completely shut off  the T cell attacks on the pancreatic beta cells. As a result, the beta cells regenerated and were able to begin producing insulin—essentially reversing the diabetes.

Insulin Will Assist Dendritic Cell “Targeting”

The phase 1 trial, which began in March, involves 15 type 1 patients over 18 years of age. The trial team is led by Dr. Massimo Trucco, the Hillman Professor of Pediatric Immunology at Children’s Hospital and a professor of Pediatrics at the University of Pittsburgh School of Medicine.

Dr. Trucco and his team want to combine the dendritic cells with small portions of insulin, in the hope that the insulin will guide the dendritic cells directly to the T cells. Adding the insulin will ensure that the dendritic cells specifically block T cell destruction of the beta cells and do not otherwise interrupt the immune system.

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