Two patients with severe type 1 diabetes to be selected for clinical trial

August 27, 2004

WORCESTER, Mass. — Taking the next step in research aimed at a better treatment for the most severe form of diabetes, a team at the University of Massachusetts Medical School and the UMass Memorial Medical Center will soon transplant insulin-producing cells into two people who suffer from type 1 diabetes. The transplant program uses a novel protocol for therapy that may help the patients live lives free of insulin dependence.

“Our ultimate goals is to be able to transplant islet cells without the need for the patients to stay on powerful immunosuppressive drugs for the rest of their lives,” said Aldo A. Rossini, MD, the William and Doris Krupp Professor of Medicine and chief of the Division of Diabetes at UMMS, leader of the team preparing for the islet transplantation. “We hope this next round of transplants will take us one step closer to that goal.”

The new round of islet transplantation is funded by the National Institutes of Health and will build on the experience Dr. Rossini and his team gained participating in the international trial of  “Edmonton Protocol” in 2002.   At that time, UMMS was among 18 centers worldwide that participated in a clinical trial to test a transplant protocol first developed at the University of Alberta in Edmonton, Canada to reverse the insulin dependence of diabetics through islet cell transplantation.

In the summer of 2002 Dr. Rossini’s team successfully replicated the protocol, transplanting islet cells into two type 1 diabetics. Both patients improved significantly following the transplants, though they still rely on some insulin injections to manage their diabetes. “We’ve taken the lessons of Edmonton and come up with a plan to use a different combination of drugs that we hope will prove more successful long-term,” said Jeffrey S. Stoff, MD, professor of medicine and chief of the Division of Renal Medicine and Transplantation Medicine at UMMS, who works with Rossini on the islet program.

The actual infusion of the islet cells is a simple procedure. Once a donor pancreas has been received and the islet cells isolated from the organ by the Joslin Diabetes Center in Boston, they are transported to UMass Memorial Medical Center’s University campus and the waiting patient. A catheter is threaded into the patient’s portal vein in the liver, where the islet cells are infused. The patient is given a mild anesthetic, but is conscious during the procedure. The total time needed to infuse some 300,000 to 500,000 cell equivalents is approximately 30 minutes, yet the effect on the patient's blood sugar levels is almost immediate and dramatic.

The burden of immunosuppression, however, makes islet cell transplantation a consideration for only a highly select group of people with type 1 diabetes. In order to prevent rejection of the transplanted cells, patients must take powerful medications for a lifetime that have the potential for serious side effects.  The medications to be used in the coming round of transplants are designed to be potentially less damaging, but they still carry significant long-term risks.   “Many people with this disease ask me if they can have an islet cell transplantation, but I discourage them because we are not there yet—we’ve got to develop better ways to rid people of their insulin dependence, without immunosuppressive therapy,” Rossini said. “This new protocol for transplantation is an important step, but much work remains to be done.”

An estimated 1.7 million people in the United States suffer from type 1 diabetes – a disease in which the individual’s immune system attacks the insulin-producing islet cells in the pancreas. People with type 1 diabetes are unable to produce insulin, which is needed for the body to use and regulate sugar levels in the blood.  Left untreated, type 1 diabetes is fatal.

Through research centered on tolerance in autoimmunity and transplant immunology, Rossini’s team is seeking a way to allow patients to receive transplants of islet cells without the need for potentially harmful anti-rejection drugs. Rossini has made major advances in understanding the immune system’s response to pancreas cell transplantation and the immunological basis for cellular rejection.  Specifically, his lab has made discoveries that suggest the viability of an approach that establishes “tolerance” in a system that is being readied to accept transplanted islet cells.

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The University of Massachusetts Medical School is one of the fastest growing academic health centers in the country and has built a reputation as a world-class research institution, consistently producing noteworthy advances in clinical and basic research.  The Medical School attracts more than $154 million in research funding annually, 80 percent of which comes from federal funding sources.  Research funding enables UMMS scientists to explore human disease from the molecular level to large-scale clinical trials.  Basic and clinical research leads to new approaches for diagnosis, treatment and prevention of disease.


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