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Researchers reverse juvenile diabetes in animal model; Clinical trial FDA approved

Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center researchers Massimo Trucco, MD, and Nick Giannoukakis, PhD, observed marked amelioration of diabetes in a mouse model by a novel treatment strategy involving specific modification of the animal's own dendritic cells, thereby reversing diabetes in animal studies.

The U.S. Food and Drug Administration (FDA) approved the start of a clinical trial to evaluate the safety and feasibility of the treatment (Phase 1 trial). The trial is expected to begin sometime this spring and will include at least 15 patients over the age of 18, with type 1 diabetes.

Dr. Trucco, director of the Division of Immunogenetics at Children's, and his research team will collaborate with Theresa Whiteside, PhD, scientific director at the University of Pittsburgh Cancer Institute.

Dr. Trucco and his team found that by removing dendritic cells from the blood during a two- to four-hour procedure, some 20 million dendritic cells can be harvested.

Dendritic cells are cells found in the bloodstream and normally function as one of nature's most efficient immune function cells. The cells identify foreign substances such as cancer cells, process these foreign substances, and then jumpstart the immune response by bringing these foreign substances to the attention of T cells.

Once harvested, researchers then combine the dendritic cells with specific blockers of molecules, known as CD40, CD80 and CD86, all of which can be synthesized in a laboratory. This treatment strategy was found to inhibit the interaction and destructive effect of T cells on the insulin-producing beta cells of the pancreas, a process that is known to be a critical part of how diabetes occurs.

Subcutaneous injection of dendritic cells into the abdominal/pelvic area near the pancreas and lymph nodes, blocks the T cells as they travel to the pancreas to destroy beta cells.

"We did this in mice, giving them s
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Contact: Melanie Finnigan
Melanie.Finnigan@chp.edu
412-692-5502
Children's Hospital of Pittsburgh
27-Mar-2006


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