Cure Insight

Inside the Hope Factory

Scientists at Miami’s Diabetes Research Institute, led by Dr. Camillo Ricordi, believe they are closing in on a cure.
(Left) Today’s diabetes technology helps people to manage the disease. Dr. Camillo Ricordi (center) Scientific Director of The Diabetes Research Institute: “… the cure is always a possibility that lies just around the corner.” Advances in diabetes research aim to benefit PWDs of all ages, including children.

Gordon Moore, co-founder of Intel Corporation, is famous in technology circles for inventing Moore’s Law, which states that the number of transistors on an integrated circuit doubles every two years. The truth of his maxim is borne out every time a new, smaller, faster, smarter product enters the market. Think iPad and iPhone, and the dizzying number of innovations like them that have literally transformed millions of lives, and the way people work, in less than a decade. Dr. Camillo Ricordi, Scientific Director of Miami’s Diabetes Research Institute, has probably never met Moore, but he acts on results of his premise, orchestrating worldwide research efforts focused on a biological cure for diabetes. Ricordi said the DRI (www.diabetesresearch.org) operates in the third era of scientific research — the digital era.

“The first era,” he noted, “was a lone scientist working by himself in a laboratory away from the rest of the world. “The second was the Manhattan Project — the way the atomic bomb was developed during World War II. You take the world’s smartest people and put them together in a single facility. That was the original model for the DRI.

“My model is taking advantage of the third era, the digital era,” he said. “We can eliminate geographic barriers to scientific collaboration, we can link project teams worldwide, we can perform clinical trials where it is most cost-efficient, and we can get around some of the regulatory and legal barriers that halt research projects in some areas. Now the world is our platform for developing a cure.”

My model is taking advantage of the third era, the digital era

DRI is the world’s largest and most comprehensive research center solely dedicated to curing diabetes, particularly Type 1 where an autoimmune response destroys all or most of the insulin-producing beta cells in an otherwise normal pancreas. Ricordi understands the magnitude of that cure challenge better than anyone. He is a pioneer in cell transplantation, which is at the heart of the search. Ricordi invented the machine that makes it possible to isolate large numbers of islet cells (insulin-producing cells) from the human pancreas. He also performed the first series of clinical islet transplants that reversed diabetes after implantation of purified donor islets into the liver of recipients with diabetes. His procedures are now used by laboratories performing clinical islet transplants worldwide.

Dr Alberto Pugliese is one of those working on cutting-edge research at DRI.

When Ricordi arrived at the DRI in 1993, there were only 10 employees. He became its head in 1996. Under his leadership, the Institute’s staff has grown to 200, including many of the world’s leading diabetes researchers, in an 87,000-square-foot building that houses laboratories, offices and a patient clinic. The DRI is a National Institutes of Health-designated Center for Excellence that is affiliated with the University of Miami Miller School of Medicine. And through the DRI Federation, an international collaborative network created by Ricordi, the DRI has online, real-time connections to more than 20 like-minded research centers around the globe.

As is the case with many of his colleagues at the DRI, Ricordi’s career path to diabetes research was the result of a series of unplanned, but increasingly interesting, connections that happened during his medical training. The interest turned personal, however, when a cousin was diagnosed with diabetes. Ricordi promised then to cure her; years later, it’s a promise he still intends to keep.

The DRI’s mission statement reads: “To develop and rapidly apply the most promising research to treat and cure those now living with diabetes.” The two most important words in that statement are “cure” and “rapidly,” in that order. The DRI is focused entirely on translational (cure-focused) research. This is no ivory tower where researchers focus on grants, journal citations and professional reputations, and jealously guard the details of their work from colleagues they view as competitors. Instead, everything is open, and everything is shared. Only the most promising research avenues are explored; if they turn out to be false starts or dead ends, they are immediately abandoned.

The DRI’s “affiliation” relationship with the University of Miami is a bit unconventional, but Ricordi believes it is vital to the Institute’s ability to pursue its own mission, unimpeded by the agenda of a larger, controlling academic entity. “To me, the affiliation represents a unique strategic advantage if your mission is to get to a cure for diabetes in the fastest, most efficient way possible,” he said. “My loyalty is to the mission, not to a particular place.”

Much of Ricordi’s effort involves moving certain research projects out of the United States to escape financial or regulatory constraints, and this is one of the reasons he created the DRI Federation. “To me, it is essential that we be able to work worldwide,” said Ricordi. “For example, we just completed enrollment in an islet transplantation trial for people with the most severe cases of diabetes. The cost for 120 patients is $250,000 per patient in the United States. By contrast, the very same islet transplantation costs $35,000 per patient in Italy. It will be free in China because it will be subsidized. That’s zero in China versus $30 million in the United States.” Ricordi has no concern with international politics or job creation; his total focus is on finding a cure, and “efficient” means cost as well as speed.

When you are on a fast-track model, Ricordi believes you have to think outside the box. “If you build your strategy and your operational framework around the mission of curing diabetes in the fastest, most efficient way possible, and you put the cure and patient at the center of your efforts, then everything has to answer the question, ‘Does this decision, this strategy, this model, this alliance help us get to the cure faster, more efficiently, to benefit the patient?’”

Although the DRI’s scientists haven’t found a cure yet, they are optimistic that they are gradually closing in on one. The mechanisms involved in diabetes are fairly well known, and a cure is based on meeting two fundamental challenges:

First comes resetting the immune system. In Type 1 diabetes, there is an autoimmune attack that causes the initial onset of the disease. This occurs when the body’s immune system mistakenly destroys the insulin-producing beta cells located in the pancreas. Halting the autoimmune attack on these cells and restoring natural insulin production continues to be an area of intense focus at the DRI, and several strategies are underway to address this issue.

Second involves generating an unlimited supply of islets. To address the short supply of insulin-producing tissue available for transplant, DRI researchers are seeking ways to create an unlimited supply of insulin-producing cells and/or to regenerate the islets that have been destroyed by the immune system. The current focus is on stem cell research, a field filled with promising developments.

“We are dealing with an extremely complex problem that has many different components,” said Ricordi. “In 1985, I thought we were three to five years from a cure, but it wasn’t to be. We have to be very careful about raising hopes prematurely. At the same time, I have never seen a speedier rate of research progress as I have in the past three to five years — nanotechnology, immunology, new molecules, biological approaches, understanding of cell biology, tissue reprogramming, stem cells, transplantation, replacement, regeneration, reprogramming, tolerance induction and many other areas.”

Scientists work to carry out DRI’s mission, “To develop and rapidly apply the most promising research to treat and cure” diabetes.

“We need to maintain a sense of urgency in research,” said Ricordi. “We are on the verge of the next quantum leap. I don’t want anyone to lose the momentum, the focus and the intensity of our effort by thinking this is so far away. First, because I don’t believe it is beyond the 10-year mark. But also because it’s impossible to say when we will discover the cure. The next trial that we’re planning could be the one that changes everything. In a way, the cure is always a possibility that lies just around the next corner.”

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Bob Benchley is an experienced health care writer and editor. He has written extensively about diabetes and other health care topics for patient-focused media for pharmaceutical companies and insurance plan providers. He lives in Miami.

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