JDF awards $10 Million to Children's Hospital of Pittsburgh and University of Pittsburgh for new research center to help speed diabetes cure

May 10, 2000

To expedite the search for a cure to Type 1 Diabetes, the Juvenile Diabetes Foundation International (JDF) has awarded a $10 million grant to Children's Hospital of Pittsburgh and the University of Pittsburgh to establish the JDF Center for Gene Therapy Approaches to Type 1 Diabetes.

The JDF Center brings together world-renowned experts in the areas of diabetes, gene therapy, immunology and transplantation with a common goal of finding a cure for diabetes through islet cell transplantation and genetic engineering.

Seven research projects will be undertaken, most of which will focus on making islet cell transplants viable for young patients. Islet cells are located in the pancreas and produce insulin. In people with Type 1 diabetes, the body's immune system malfunctions, producing an autoimmune response that destroys islet cells. The goals of the JDF Center are to develop new gene therapy techniques that will increase the supply of islet cells available for transplantation and to protect transplanted islet cells from immune system attack.

"Islet cell transplantation is one of our most promising hopes of finding a cure," said Dr. Sandra Pucznynski, chair of research for the JDF. "The new JDF Center is an important building block in helping us put islet transplantation on a fast track." Dr. Puczynski is an assistant professor of medicine and director of research and evaluation at the Medical College of Ohio. She co-founded the Pittsburgh (now Western Pennsylvania) Chapter of JDF in 1987 and was its president from 1988-90. One of her daughters has Type 1 diabetes.

Massimo Trucco, MD, chief of the division of immunogenetics and the Hillman Professor of Immunology at Children's Hospital of Pittsburgh and the University of Pittsburgh School of Medicine, is the director of the new JDF Center. "Transplanting healthy islet cells into Type 1 diabetes patients poses many challenges," he said. "The research of my colleagues and myself will approach the problem of autoimmunity and alloimmunity from many angles. By bringing together experts in the fields of transplantation, immunology and gene therapy, we can collectively attack the barriers that are impeding islet cell transplantation and expedite the search for a cure."

The co-directors of the center are Thomas E. Starzl, M.D., Ph.D., world-renowned transplant surgeon and professor of surgery at the Thomas E. Starzl Transplantation Institute, University of Pittsburgh School of Medicine; and Joseph C. Glorioso III, Ph.D., professor and chairman of the department of molecular genetics and biochemistry at the University of Pittsburgh School of Medicine.

More than one million Americans have Type 1 (juvenile) diabetes. It can occur at any age, but most commonly is diagnosed in childhood. In Type 1 diabetes, a person's pancreas produces little or no insulin. Although the causes are not entirely known, scientists believe the body's own immune system attacks and destroys insulin-producing cells in the pancreas. Because insulin is necessary for life, people with Type 1 diabetes must take multiple insulin injections each day for the rest of their lives to survive.

JDF is the world's number one nonprofit, nongovernmental funder of diabetes research. It was founded in 1970 by parents of children with diabetes. JDF's mission is to find a cure for diabetes and its complications through the support of research, and since its inception has provided more than $326 million to diabetes research worldwide .

Children's Hospital of Pittsburgh is the only freestanding hospital in Western Pennsylvania dedicated solely to the care of infants, children and young adults. The hospital has been named consistently to several elite lists of pediatric health care facilities. This recognition includes being ranked as one of the top 10 children's hospitals in the country by U.S. News and World Report and ranking sixth in funding provided by the National Institutes of Health.

The University of Pittsburgh School of Medicine is consistently ranked among the nation's leading medical schools. It is one of the university's six Schools of the Health Sciences, which include the schools of Nursing, Dental Medicine, Pharmacy, Health and Rehabilitation Sciences and the Graduate School of Public Health. Their combined mission is to train tomorrow's health care specialists and biomedical scientists, engage in groundbreaking research that will advance the understanding of the causes and treatments of disease and to participate in the delivery of care as a partner with the UPMC Health System. Among the many areas for which its faculty and programs are internationally recognized are oncology, psychiatry, genetics, transplantation and public health.

JDF Center for Gene Therapy Approaches to Type 1 Diabetes at Children's Hospital of Pittsburgh and the University of Pittsburgh

The Juvenile Diabetes Foundation International (JDF) Center for Gene Therapy Approaches to Type 1 Diabetes at Children's Hospital of Pittsburgh and the University of Pittsburgh marks a new approach to the treatment of Type 1 diabetes, using gene therapy to improve islet cell transplantation for insulin replacement therapy and to treat neurologic complications.

The goal of the center is to advance the practice of islet transplantation en route to developing a cure for diabetes. For the first time, the new center brings together world-renowned experts in the fields of immunogenetics, transplantation and gene therapy to work toward the common goal of curing Type 1 diabetes.

Background. Islets, beta cells located in the pancreas, are the body's natural insulin-producing cells. Insulin promotes the absorption of sugar (glucose) by various organs and lowers glucose levels in the blood. In people with Type 1 diabetes, the body's immune system malfunctions, producing an "autoimmune" response that destroys the islet cells. Transplanting healthy islets from a donor is an experimental method that diabetes researchers are using to attempt to restore normal insulin production in Type 1 patients. However, transplantation of islets faces several major problems.

Because of a shortage of organ donors, the supply of pancreatic islets available for transplantation is very limited. For the transplant of one patient, three to four donor pancreases are required. In addition, the immune system of any patient who receives transplants rejects the foreign cells, unless the recipient's immune response is suppressed. Unfortunately, current therapies that suppress the immune system target not only the transplanted organ or cells but also generally reduce immune responsiveness, thereby greatly increasing the recipient's risk of infection and cancer. Also, the drugs currently available are particularly toxic to islets and can in themselves cause diabetes. Finally, the particular autoimmune response of Type 1 diabetes patients, which causes Type 1 diabetes, can also attack and destroy the transplanted islets that are meant to cure the disease.

Gene therapists treat or prevent disease by replacing, altering or supplementing a patient's genetic material. To do so, they must not only determine what genetic material a patient needs, they must also find or develop "vectors." Vectors carry genes or genetic material to a target cell and transfer the therapeutic genes to that cell. Viruses are often used because of their ability to enter cells and to introduce DNA into them.

The goal of the JDF Center is to develop new gene therapy techniques that will increase the number of viable islets for transplantation, protect these transplanted cells from immune system attack and prevent the diabetes-related complication, peripheral neuropathy, in which nerves are injured. In at least the first three years of the Pittsburgh Center, all of the projects' research and testing will be on laboratory animals. Eventually, investigators expect that findings from these projects will form the basis of clinical trials with humans.

Six of the seven projects at the JDF Center will use ex vivo gene therapy; that is, cells will be taken from a living animal, treated with genetic material, then returned to the animal. In the project to prevent diabetic nerve damage, gene therapy will be used in vivo; that is, genetic material will be introduced directly into a living animal.
Aditional contacts:Lisa Rossi
University of Pittsburgh

Julie Kimbrough

University of Pittsburgh Medical Center

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