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Thursday May 28, 2020
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July 2004 - Volume 1 - Issue 1

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Diabetes Research Institute Committed To Curing Diabetes

There are some years that stand out in history. 1066. 1776. 1865. 1921. Why 1921?

Prior to that year, a diagnosis of type 1 diabetes meant certain death, but after 1921, thanks to the work of Drs. Frederick Banting and Charles Best who first isolated the hormone insulin and identified its role in controlling blood glucose levels, type 1 diabetes became a disease that at least could be treated and lived with, even if it couldn’t be cured.

The scientists at the Diabetes Research Institute (DRI) at the University of Miami are working diligently to change that. Their hope is, in the not too distant future, that 1921 will be relegated to a footnote in history. Based upon their recent success with a promising procedure known as islet transplantation, that future may not be too far off.

"We are really searching for a cure," says Norma Sue Kenyon, Ph.D., an immunologist who is the Associate Director of Research and Program Development and the Director of Pre-Clinical Islet Transplantation at DRI, as well as the Martin Kleiman Professor of Surgery, Medicine, Microbiology and Immunology at the University of Miami School of Medicine. "I believe it will come in our lifetime."

This hope for a cure has been the passion of the staff at DRI since it was founded in 1971. Actually, the passion for a cure existed even before there was a DRI.

"In the early 1970s, a handful of parents of children with type 1 diabetes got together and began trying to address some of the needs of their children," said Gary Kleiman, Executive Director of Medical Development at the DRI. Kleiman, who was diagnosed with the disease at age 6, knows about this first-hand because his parents became a part of that group when had begun to develop diabetes-related complications in his eyes and kidneys.

Together, the families rallied the community, and began raising money to fund research into diabetes. Kleiman says they had a very clear focus – to find a cure – but he admits that at the time they probably were unaware of the enormity of the task ahead of them.

"In many ways, the early 1970s were a very naïve time," he said. "We had just put a man on the moon a few years before, so on one level you felt anything could be done…science and technology could accomplish anything."

This commitment to a cure would eventually lead to establishing a multi-disciplinary program at the University of Miami. Under the umbrella of the DRI, cell biologists, immunologists, endocrinologists, and surgeons would come together to search for a cure for diabetes. Kleiman says that this was the first program that rallied different medical specialties under the banner of a single disease. Leading the way was Daniel Mintz, M.D., the Institute’s founder and first scientific director. Dr. Mintz was a well-respected physician scientist whose vision and concern for those families touched by diabetes created an Institute with a single purpose.

Today, under the direction of Camillo Ricordi, M.D., the Stacy Joy Goodman Professor of Surgery and Medicine and the Scientific Director and Chief Academic Officer at DRI, the program still puts patients first and they are still searching for that elusive cure. But they are much closer to their ultimate goal.

Much of the DRI’s current research is focused on islet cell transplantation, a procedure that is considered to be the most promising method to cure diabetes. The islets of Langerhans are the insulin-producing cells in the pancreas, which are mistakenly destroyed by the body’s own immune system for reasons that are still unknown. The result of this "autoimmune" attack is type 1 diabetes, and patients must take daily insulin injections to stay alive. The goal of islet transplantation is to give back these cells that have been destroyed, thereby restoring the patient’s ability to again make his or her own insulin.

In the mid-1980s, the first islet transplant was conducted with only minimal success. Since then, significant advances in transplant immunology and islet isolation have allowed some patients to become free of insulin injections. Kleiman is one of those patients. After living with diabetes for more than four decades and witnessing all of the research progress that has occurred over that time, he received an islet transplant a year and a half ago, and has not needed one drop of insulin ever since. "While I was unlucky to develop complications at such an early age, I was fortunate to be able to ride the crest of developing technologies, such as laser treatments and anti-rejection drugs that kept me going. Needless to say, the ultimate benefit was having been able to receive an islet transplant. It’s truly been remarkable," said Kleiman.

While there has been a dramatic increase in the success rate, islet transplantation is still considered an experimental procedure. Two major hurdles still need to be overcome before this therapy can be made available to many who can benefit. The first is increasing the supply of these much-needed cells and the second is overcoming the need for the use of powerful drugs to prevent transplant rejection. Dr. Kenyon’s team of researchers is focused on ways to transplant islets without the need for these drugs. She has conducted numerous studies using bone marrow to "re-educate" the recipient to become "tolerant," to donor islets. She’s also studying ways to block specific immune cells from attacking the transplanted tissue.

"One of the side effects of the anti-rejection drugs is that they turn off all of the immune cells, leaving the person prone to infections or cancer," she said. "Ideally, immunologists believe we can target only those immune cells that react with the transplanted tissue."

Dr. Kenyon has been able to build on this research to cure diabetes in mice and non-human primates.

"We have cured diabetes in mice hundreds of times, but humans are much more complex," she said. "We have been able to show that islets could work in a model very similar to humans. Typically, if it works with primates, there is a good chance that it will work in humans."

As an immunologist, Dr. Kenyon believes that these issues will be resolved, opening the door for others to benefit from islet transplantation, including children like her own daughter, Laura.

"I was trained as a transplant immunologist," Dr. Kenyon said. "I was doing diabetes research and then moved on to other types of research, but when Laura was diagnosed with diabetes, I came back to it."

Such inspiration isn’t a prerequisite for working in medical research – Dr. Kenyon says most scientists she knows truly love the quest for new discoveries in itself – but it doesn’t hurt either, and, with such passion and commitment, soon Dr. Kenyon and the other researchers at DRI will have a cure for all the Lauras everywhere.

Gary Kleiman can be reached by calling (305) 243-3899. Norma Sue Kenyon, Ph.D., can be reached by calling (305) 243-5346.
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