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Johnson & Johnson, continuing its long quest for a Type 1 diabetes cure, is joining forces with biotech company ViaCyte to speed development of the first stem cell treatment that could fix the life-threatening hormonal disorder.
The therapy involves inducing embryonic stem cells in a lab dish to turn into insulin-producing cells, then putting them inside a small capsule that is implanted under the skin. Researchers at universities and other drug companies also are working toward a diabetes cure, using various strategies.
If the project succeeds, the product could be available in several years for Type 1 diabetes patients and down the road could also treat insulin-using Type 2 diabetics. People with Type 1 diabetes no longer produce insulin, the hormone that converts sugar in the blood into energy, because their immune system has killed off the beta cells in the pancreas. Many patients can't control it well because treatment is exhausting, requiring a strict diet, frequent exercise, multiple daily insulin injections or other medicines and several finger pricks a day to test blood sugar.
ViaCyte Inc., based in San Diego, has been researching its treatment for a decade, partly with funding from the Juvenile Diabetes Research Fund and the California Institute for Regenerative Medicine.
The privately held company began the first round of patient testing a year ago, implanting its product, dubbed VC-01, in a dozen people with Type 1 diabetes, said Paul Laikind, ViaCyte's CEO and president. After 12 weeks, the device had properly attached to nearby blood vessels, their new insulin-producing cells were still multiplying and no side effects were seen. If that goes well, in the next round of testing a few dozen patients will get devices holding a full dose of the cells implanted, likely in the second half of this year.
Earlier testing in thousands of mice over years showed the lab-created insulin-producing cells matured and produced the needed hormone inside the mice for as long as they lived, about a year, noted Laikind. Because of the protective capsule, which is flattish and smaller than a business card, if something goes awry, the capsule can be removed immediately to prevent patient harm. A CURE for diabetes is within reach after scientists proved the debilitating condition can be stopped for six months. A new vanguard of researchers continues UF’s tradition of pushing the boundaries of diabetes research and care. More than 30 years ago, Mark Atkinson, PhD ’88, was given three research questions that he would spend his career trying to solve.
And in the years since, Atkinson, now director of the University of Florida Diabetes Institute, and Desmond Schatz, MD, the institute’s medical director for Type 1 diabetes, have resolutely maintained a strong team of researchers to chip away at those questions and to translate their findings to the care of patients. While other institutions often find it challenging to sustain a research endeavor that has the kind of influence and staying power UF’s diabetes program has had over the decades, UF now looks to its third generation of physicians and scientists to make their own mark on the global effort to prevent and cure diabetes.
Schatz agrees, saying the program’s legacy is its talent for attracting new team members who continue to provide insight into the mechanisms of diabetes and ways to impact care for those with it. The lineage of UF’s diabetes program dates back to 1968 with Rosenbloom, who brought Janet Silverstein, MD, now a professor in the department of pediatrics, and Noel Maclaren, MD, on board. Their proteges, including Haller and colleagues like Clayton Mathews, PhD, the Sebastian Family professor for diabetes research, and Clive Wasserfall, MS ’98, make up the third wave, and already they have helped attract the latest vanguard of researchers who appear to be following traditions by pushing the boundaries of diabetes research and care. Todd Brusko, PhD ’06, can easily say he’s studied diabetes at UF for more than 15 years — even though the researcher is only 37. Brusko, who leads a laboratory in the department of pathology, immunology and laboratory medicine, started working as an undergraduate in a Type 1 diabetes genetics lab at UF. Through his research, Brusko is trying to understand how a defect in immune regulation leads to Type 1 diabetes.
Brusko thinks some of the genes related to Type 1 diabetes cause T cells to attack beta cells.
Jing Chen, MD, PhD, started her study of Type 1 diabetes in her home country of China, when she first was faced with an overwhelming number of patients with diabetes.
After completing postdoctoral work in the United States, Chen began working with Mathews, also a professor in the UF department of pathology, immunology and laboratory medicine, but then at the University of Pittsburgh.


Chen and Mathews have shown how dysfunction in mitochondria, organelles within cells that produce energy, may contribute to the development of Type 1 diabetes. The group hopes to discover ways therapies can target T cell mitochondrial function that will prevent the immune system from attacking beta cells.
When Laura Jacobsen, MD ’12, returned to UF after a residency at the University of North Carolina in Chapel Hill, she wasted no time becoming involved in a project or three. Jacobsen’s first project uses data from nPOD, a repository of donated pancreas tissue established by UF’s Atkinson and hosted by the Juvenile Diabetes Research Foundation. Typically, researchers use the repository to study what happens in the organ of someone who has diabetes.
When pancreas tissue is donated to nPOD, medical records and history are included, providing information about what caused that person’s death. Jacobsen is also assisting Haller with clinical trials that examine if some medications can slow the rate of beta cell decline. UF has long been recognized internationally as a leading center for Type 1 diabetes research — but Type 2 diabetes and diabetes in adults is reaching a fever pitch across the United States. Enter Kenneth Cusi, MD, chief of the division of endocrinology, diabetes and metabolism in the UF College of Medicine’s department of medicine. The year before Cusi arrived, the endocrine team consulted on just 360 patients hospitalized for complications of diabetes. Cusi developed a program called the Diabetes Care Task Force, a multidisciplinary team whose goal is to make UF Health a premier institution for diabetes care. Along with developing UF Health’s capacity to care for patients and training future endocrinologists, Cusi continues to delve into the factors that contribute to the complications associated with diabetes. Undiagnosed, the disease can also lead to a type of cancer called hepatocellular carcinoma, as well as cardiovascular disease. At UF, the work of diabetes scientists and clinicians is interwoven, which Atkinson says is a must. In 2014, the university officially pulled all of that research together with the launch of the UF Diabetes Institute, with Atkinson as director and Schatz as medical director for Type 1 diabetes. From clinicians to researchers to social workers to teachers, nearly every department that can contribute to diabetes research is involved.
To learn more about how UF is impacting diabetes research, download the Florida Physician iPad app from the Apple App Store. From humble beginnings to a multibillion dollar industry, Gatorade inspired a culture of innovation that continues to help define the UF College of Medicine.
Siblings Amy and Chris Bucciarelli took to Africa’s highest peak to honor their father, Richard Bucciarelli. If it works as well in patients as it has in animals, it would amount to a cure, ending the need for frequent insulin injections and blood sugar testing.
The capsule protects the cells from the immune system, which otherwise would attack them as invaders - a roadblock that has stymied other research projects.
Tom Donner, director of the diabetes centre at Johns Hopkins University School of Medicine. Without effective treatment, diabetics suffer severe complications: blindness, kidney failure, heart disease, amputations, even premature death. They received a small dose of insulin-producing cells inside their devices and are being closely monitored for two years to see insulin production and other effects. Another dozen planned patients will soon get the same cell dose in capsules to be implanted in them. Betul Hatipoglu, an endocrinologist at Cleveland Clinic, wrote in an email that preliminary results on the device are promising.


He points to Michael Haller, MD, a 2000 graduate of the UF College of Medicine who began working under Schatz’s tutelage as a UF undergraduate. Atkinson and Schatz, associate chair of pediatrics in the UF College of Medicine who recently began his term as president of the American Diabetes Association, began researching Type 1 diabetes in the early ’80s. Highlighted here are just a few of what should be UF’s latest generation of promising researchers in diabetes. After completing a doctoral degree in the UF College of Medicine’s Interdisciplinary Program in Biomedical Sciences, Brusko studied as a post-doctoral researcher at the University of California – San Francisco before returning to UF in 2010.
During Type 1 diabetes, a person’s immune cells, specifically T cells, begin attacking beta cells in the pancreas.
But Jacobsen is working with Schatz to study how the person who donated the pancreas lived — and died. A donor may have died from complications with diabetes such as diabetic ketoacidosis — a condition in which a person doesn’t have enough insulin to convert glucose into energy and begins breaking down fat, releasing ketones.
For example, a history of drug or alcohol abuse could indicate to researchers that some patients require help in managing their stressful disease. With Janet Silverstein, MD, Jacobsen is studying a way to use a portable eye scanner to test for retinopathy in children with Type 1 diabetes. The endocrinology fellowship has likewise grown from two positions to five, and according to the numbers, the country really needs this kind of education. The program oversees care for adult patients with Type 2 and Type 1 diabetes — about 30 percent of adult patients seen at UF Health have Type 1 diabetes.
He’s an expert in nonalcoholic fatty liver disease, or NAFLD, a condition that affects about two-thirds of patients with Type 2 diabetes. The institute brings together nearly 100 faculty members from the colleges of Medicine, Engineering, Public Health and Health Professions, Nursing and Liberal Arts and Sciences as well as the Institute of Food and Agricultural Sciences and UF Health Jacksonville in the fight to cure both Type 1 and Type 2 diabetes.
The interdisciplinary approach has turned UF into a hub for diabetes work and has attracted a cadre of talented researchers and grants. On the other hand, too much insulin can cause very low blood sugar, which can kill patients, particularly young children. Meanwhile, the number with Type 2 diabetes, whose bodies make some insulin but don't use it efficiently, is increasing exponentially due to the global epidemic of obesity and sedentary lifestyles. Haller, who has led first-of-its-kind clinical trials studying the use of cord blood, as well as combination drug therapies to prevent or reverse Type 1 diabetes, was recently named chief of pediatric endocrinology at the UF College of Medicine. With Michael Clare-Salzler, MD, current chair of the UF College of Medicine’s department of pathology, immunology and laboratory medicine, they represent UF’s second wave of diabetes scientists and clinicians. A donor’s death by suicide could show physicians a need for emotional support and mental health.
At the Malcom Randall Veterans Administration Medical Center, where Cusi was chief of endocrinology from 2013-2015, the number of visits from patients with diabetes tripled to 7,000 visits per year.
These patients are also at the highest risk of the more severe form of liver inflammation known as nonalcoholic steatohepatitis, or NASH, which often leads to end-stage liver disease.
Researchers also learn about factors that could have contributed to a person’s heart attack, kidney disease or stroke. At UF Health Endocrinology – Medical Specialties – Medical Plaza’s outpatient clinic, physicians had 12,000 patient visits in just the last year.



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