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Diabetes
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The Fight Against Diabetes
Researchers at Children’s, including Drs. Becker and Trucco, are leading local, regional, national and international clinical trials to study treatments that could offer better control and potentially even a cure of type 1 diabetes. During the week of Nov. 12, Drs. Becker and Trucco attended a meeting in Miami of scientists from around the world, where they provided updates on much of the cutting-edge diabetes happening in Pittsburgh. The meeting was organized by TrialNet, a global network of researchers from more than 150 medical centers dedicated to studying the prevention, early detection and treatment of type 1 diabetes.
Children’s Hospital of Pittsburgh of UPMC Studying Drug With the Potential To Prevent or Delay the Onset of Type 1 Diabetes
Researchers at Children’s Hospital of Pittsburgh of UPMC are participating in an international clinical trial to study the effectiveness of oral insulin in preventing or delaying the onset of type 1 diabetes in people at risk for the disease.
The researchers want to determine if one insulin capsule taken daily can prevent or delay the onset of type 1 diabetes in relatives of people who are found to be at risk for developing the disease, according to Dorothy Becker, MBBCh, chief of the Division of Pediatric Diabetes and Endocrinology at Children’s and principal investigator of the study.
An earlier trial called Diabetes Prevention Trial 1, conducted at Children’s and other centers around the world, suggested that oral insulin might delay type 1 diabetes by about four years in some people with auto-antibodies to insulin in their blood. Oral insulin has no known side effects.
“Type 1 diabetes is a very difficult disease to manage. Because it typically begins in childhood or young adulthood, and if not properly controlled with insulin injections and diet and exercise, diabetes can lead to a lifetime of complications that can cause chronic disability and be life-threatening,” said Dr. Becker. “If oral insulin could delay the onset or prevent the disease, we could spare these patients years of difficult management and potential complications such as heart disease and vision loss.”
Learn more about diabetes research at Children’s Hospital.
Groundbreaking Human Clinical Trial for Type 1 Diabetes to Start This Spring
in Pittsburgh
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 six injections over the course of several weeks. The injections interrupted the T cell and beta cell interaction, allowing the beta cells in the pancreas to regenerate. This enabled the pancreas of the mice to begin producing insulin again,” said Dr. Trucco, who also is the Hillman Professor of Pediatric Immunology at Children’s Hospital and a professor of Pediatrics at the University of Pittsburgh School of Medicine. “The injections proved capable of stopping this vicious cycle, and through this process curing type 1 diabetes in a mouse.”
Until now, scientists weren’t sure why the immune system attacks the beta cells, but the result is that the body stops producing insulin. When that happens, glucose builds up in the blood, but the body’s cells starve to death.
“We are very excited to begin the clinical trials and see if this process will work in patients with type 1 diabetes,” said Dr. Trucco. “A type 1 diabetes diagnosis can be devastating for children and their families. We hope this trial will have results that are life-altering for patients who suffer from this disease.”
In addition, Dr. Trucco and his team want to combine the dendritic cells with small portions (i.e. peptides) of insulin. Dr. Trucco believes that adding small doses of insulin to the dendritic cells will help guide the dendritic cells directly to the T cells. Adding the insulin ensures the dendritic cells are used specifically to block the T cell destruction of the beta cells, and not otherwise interrupt a person’s immunity. In other words, the dendritic cells are led directly to the target, the T cells that are causing the malfunction of the pancreas, and not to another part of the body.
