In a downtown Miami tower, 27 researchers peer into microscopes, take specimens from refrigerators, and drop liquid into test tubes in their daily search for a cure for diabetes.
In the tower’s third floor imaging lab, Dr. Midhat Abdulreda studies whether insulin-producing islets can be implanted in the eye and thrive.
Down the hall in the pre-clinical research lab, Dr. Norma Kenyon investigates how to best replace insulin-producing cells without the use of anti-rejection drugs that lower the whole immune system.
And, in the fifth floor cell therapy lab, Dr. Giacomo Lanzoni researches how certain stem cells regulate the immune system.
Their goal is focused: They want to to improve the lives of people with insulin-dependent diabetes. Already, they are making headway. More than 37 million Americans have some form of diabetes, a difficult and exhausting-to-manage disease that effects people of all ages.
At the Diabetes Research Institute in Miami, hope rolls off the tongue of scientists, patient educators, foundation fundraisers, and a new CEO.
“As a field, we are absolutely poised to make great progress and to deliver new therapies and one day a biological cure for this disease,” said Michael Burton, CEO of the Diabetes Research Institute Foundation. “That’s really what this institute is focused on.”
The institute at the University of Miami Miller School of Medicine is supported by a private foundation that funds its cure-focused research.
Experiments and discoveries
Clinical trials, experiments and patient education at the Diabetes Research Institute focus on Type 1, an autoimmune disease that often strikes without warning. Type 1 is autoimmune and previously had been known as juvenile diabetes because it usually appears during childhood or adolescence. However, it has been developing more often in adults. To stay alive, a patient needs insulin and ongoing blood sugar monitoring.
Whatever discoveries are made in Miami, the science may also apply to Type 2, the most common form of diabetes usually associated with obesity and lifestyle.
Researchers believe a cure for diabetes will involve a combination of therapies.
The only cure until now has been a pancreas transplant or a transplant of islet cells from an organ donor’s pancreas into the liver. A shortage of organ donations limits this option and because a patient has to take drugs that suppress the immune system to tolerate the transplant, there is a high risk for life-threatening infections.
A challenge for the Miami team is to find a way to create insulin-producing islets rather than relying on those from an organ donor’s pancreas, and implant the islets without having to suppress the immune system. The objective is for a diabetic to live without the daily insulin injections and be free of the risk of hypoglycemia, those big blood sugar drops
“Right now patients have had to make a decision, risk versus reward,” explains Jill Shapiro Miller, executive vice president of the Diabetes Institute Research. “That’s not the answer for the population that we’re trying to serve here, to give everybody the chance to have their own insulin production back to normal.”
Many doctors and scientists at the Diabetes Research Institute have a personal connection to the disease. For Dr. Kenyon, the connection is her daughter who fell into a diabetic coma at only 14 months old and has struggled to manage the disease for the last 30 years.
Kenyon, an immunologist, spends her days in the Miami lab testing new biological substances or compounds that attach to insulin-producing islets when they are implanted. These substances would target immune cells to control rejection, eliminating the need to use anti-rejection drugs to suppress the whole immune system.
“We’re testing a lot of devices with collaborators to see if we can get local delivery of immune intervention,” she said.
As she conducts experiments, Kenyon says she feels hopeful: “To me we are at an absolute transformational point in Type 1 diabetes cure research.”
Could the answer be the eye?
Like Kenyon, Dr. Abdulreda explores how to get transplanted islets to avoid being rejected by the body’s immune system. His work centers on where the insulin-producing islets are transplanted. A variety of sites are being tested.
Abdulreda thinks putting them into the interior chamber of the eye, rather than into the liver, may be a solution. He has seen success with mice and non-human primates. The liver has become the default because the pancreas is embedded deeply in the body and hard for surgeons to access, he said.
“The islets thrive in the eye,” he said. “They have sufficient oxygen levels from literally the first hour after transplantation, so the risk of islets dying is absent.”
The eye, he explains, is immune privileged, meaning it has limited immune and inflammatory responses so the cells are less likely to attack an invader.
“We have convinced the FDA to give us approval to test the safety of implanting pancreatic islets into the eye of human patients with Type 1 diabetes who are legally blind,” he said. “Once safety is established in a limited number of patients, the next step is to advance on a larger scale.”
What patients say
On the fifth floor, Dr. Lanzoni focuses on another approach to a cure. He’s researching everything from what triggers Type 1 diabetes to how to get more protective cells in the body. He also is working with companies that are developing cell therapies to help patients reduce or cut out insulin use entirely.
Lanzoni points to his assistant who is pouring liquid in test tubes. “He is studying what components released by stem cells are key so we can use the effective portion of the components to get the benefits we are looking for. ”
What makes the Diabetes Research Institute unique, Lanzoni explains, is that doctors in the building treat diabetic patients, and those patients participate in research.
“Patients tell us what things they don’t like in their current treatment, and from those conversations we recognize there’s so much more that needs to be solved,” he said. “When it comes to immune suppression, we know where the problems are that we want to solve in the laboratory.”
Clinical trials for diabetes
In a big advancement for diabetes, Vertex Pharmaceuticals has been cleared by the FDA to enter clinical trials in the United States for a therapy that eliminates the need for insulin. The company has developed a way to manufacture insulin-producing beta cells from stem cells grown in its lab. This replaces the need for islets from a human pancreas.
Miami is one of the clinical trial sites.
If the lab-grown cells work, the hope is that they can be encapsulated in a specially-designed device to make the therapy safer.
“We want to replace insulin-producing beta cells but also protect them from an immune effect,” Lanzoni said. “We think all of this can be done in a bioengineered environment. If we can do this, not only can we control diabetes in the short term, but in the long term because those cells are protected.”
Vertex is not the only company with trials underway.
“I think there’s about 800 companies in the world that focus on developing products for Type 1 diabetes and we at the DRI are collaborating with about 600 of them,” Lanzoni said.
Dr. Khemraj Hirani, chief operating officer at the Diabetes Research Institute, said patients are involved in 48 different human studies. Twenty of them are Phase 1, which primarily establishes a new drug’s safety and dose range. “These are being done by industry. We just collaborate with them,” he said.
At the institute, prevention trials also are ongoing for people who have antibodies and family members with the disease, and there are trials underway for newly diagnosed patients.
“Not one thing alone is going to be the answer,” said Shapiro Miller, who has been at the Diabetes Research Institute Foundation for 41 years and says advancements now are happening quickly. “There are a bunch of horses in race and some of these are going to be put together to create the therapy that’s actually going to do the trick.”
Sun Sentinel health reporter Cindy Goodman can be reached at firstname.lastname@example.org.