The following story was written for the first edition of Advances in Endocrinology, a publication of New York-Presbyterian Hospital, Cornell Weill/ Columbia University Medical Center.
Before 1922, children diagnosed with type 1 diabetes would have died. But in that year, Dr. Frederick Banting and his assistant Charles Best discovered how to extract insulin from a dog’s pancreas, completely altering the once fatal diagnosis and changing the paradigm for care.
“Since then strides in clinical care for the treatment of diabetes have been spectacular, but it is not enough,” says Robin S. Goland, MD, a renowned endocrinologist and Co-Director of the Naomi Berrie Diabetes Center at NewYork-Presbyterian/Columbia University Medical Center. Dr. Goland, along with her Co-Director Rudolph L. Leibel, MD, and the physicians, scientists, and healthcare team at the Berrie Center are relentless in their pursuit of reducing barriers to care and to finding a cure for diabetes.
“Those two things are what the Berrie Center does best,” says Dr. Goland. “Our research labs are located next to the physicians’ offices, so advances are translated to the patient as rapidly as possible. It seems to us the fastest way to a cure is to have everyone talking to each other. And what we never imagined, and what has turned out to be so powerful, are the insights our patients provide to the scientists.”
Established in 1998, the Naomi Berrie Diabetes Center is internationally recognized for excellence and innovation in patient care and research in diabetes. One of the largest multidisciplinary diabetes centers in the world, the program provided some 14,000 patient visits and 500 screenings of patients’ relatives for antibodies indicating risk for type 1 diabetes (T1D) in 2016. Half of the adult patients at the Berrie Center have T1D and virtually all of the pediatric patients. “We’re experts in treating people with new onset type 1 diabetes and transitioning patients from pediatric to adult care,” says Dr. Goland. “Caring for teenagers and young adults with diabetes is especially rewarding and, at the same time, it is so complex for the patients, their families, and the care team.”
Dr. Goland notes that the Center’s doctors, diabetes educators, nutritionists, and mental health specialists emphasize the importance of self-care in diabetes management. “This is a very challenging disease to control. It’s a constantly changing and demanding landscape. If patients take too much insulin they can make themselves very sick. If they take too little they could develop diabetes complications. So it’s a balancing act that requires an enormous amount of effort from patients and their families.”
A Robust Research Program
While their sights are firmly set on finding a cure, Dr. Goland and her colleagues are also working to prevent the clinical onset and to delay progression of T1D. To that end, Columbia University Medical Center participates in Type 1 Diabetes TrialNet, an international network of leading academic institutions, physicians, scientists, and healthcare teams that are exploring ways to prevent, delay, and reverse the progression of T1D.
Jointly funded by the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of Allergy and Infectious Diseases, and the National Institute of Child Health and Development, TrialNet conducts clinical research and clinical trials in a number of areas. These include the preservation of insulin-generating islet cells in those newly diagnosed with T1D and in the etiology and prevention of the onset of diabetes in individuals at high risk for developing the disease. Patients with autoimmune pre-diabetes are followed to evaluate factors associated with the development of diabetes and are also enrolled in studies of agents to prevent diabetes onset. TrialNet is also conducting studies of the use of immunosuppression or immunomodulation to preserve beta cell function in individuals newly diagnosed.
Pathway to Prevention is a TrialNet research screening program that serves as the first step for all TrialNet prevention studies. Screening is offered free to relatives of patients with T1D to evaluate their personal risk of developing the disease. “TrialNet’s goal is to identify the disease at its earliest stage, delay progression, and ultimately prevent it,” says Dr. Goland, a TrialNet Principal Investigator. “At the Berrie Center, we offer screening and clinical trials for every stage of type 1 diabetes and close monitoring for disease progression.”
“We are making progress in identifying individuals at risk for developing T1D,” says Magdalena M. Bogun, MD, an endocrinologist whose clinical practice focuses on adults with type 1 and type 2 diabetes. “Earlier identification gives patients an opportunity to enter clinical trials that hopefully can delay or prevent the onset of type 1 diabetes.”
Relatives of individuals with T1D are 15 times more likely to develop the disease than the general population. The increased risk is linked to the presence of five diabetes-related autoantibodies. The Juvenile Diabetes Research Foundation, American Diabetes Association, and the Endocrine Society now classify having two or more of these autoantibodies as early stage T1D.
In 2015, Dr. Bogun was one of four physicians selected to participate in the TrialNet Emerging Leader Program designed to promote the engagement of new investigators in diabetes clinical research. “My research focuses on the natural history of type 1 diabetes drawing on TrialNet data,” says Dr. Bogun, whose project is a longitudinal study of insulin secretion in patients who have had metabolic testing both before and after diagnosis of T1D. “Understanding the patterns of insulin secretion would give us more understanding about the pathogenic evolution of the disease– which could, in turn, guide our treatments.”
According to Dr. Bogun, TrialNet is the only cohort that includes data on glucose and insulin secretion before, at diagnosis, and after diagnosis of type 1 diabetes mellitus. “We then follow them every six months, watching the glucose levels and tracking their endogenous insulin production,” she says. “The goal of new onset studies is to preserve endogenous insulin secretion as measured by C-peptide. Studies have shown that preservation of C-peptide is linked to reduced risk of progression of microvascular complications such as retinopathy, nephropathy, and neuropathy.”
The natural history of type 1 diabetes differs depending on age, adds Dr. Bogun. “For example, we know that children, when diagnosed with T1D, will start with a much lower endogenous insulin production. They also drop their endogenous insulin production much quicker than patients who are diagnosed later in life.”
“Over the last 23 years that I’ve been working in this field the life expectancy of patients with diabetes has gotten closer and closer to normal,” says Dr. Goland. “The outlook for people diagnosed today is much brighter. However, we need a new way of looking at a world without diabetes, and I believe we are on the brink of a revolution in science to achieve that goal.”