Type 1 Diabetes—Reaping the Rewards of a Targeted Research Investment

Judith E. Fradkin; Julie A. Wallace; Beena Akolkar; Griffin P. Rodgers


Diabetes. 2016;65(2):307-313. 

In This Article

Abstract and Introduction


The Diabetes Control and Complications Trial (DCCT) precipitated a major research effort to develop new approaches to achieve near-normal glycemic control in real-world settings in people with type 1 diabetes. Toward that end, a unique funding stream from the U.S. Congress—the Special Statutory Funding Program for Type 1 Diabetes Research—has provided nearly $2.5 billion for research into the prevention, cure, and treatment of type 1 diabetes since 1998. This funding generated a targeted, sustained investment in type 1 diabetes research with six specific goals: identifying new therapeutic targets through the understanding of disease etiology and pathogenesis, preventing or reversing the disease, developing cell replacement therapy, improving management and care, preventing or reducing the complications, and attracting new talent and applying new technologies to type 1 diabetes research. This Perspective describes exciting results that have emerged from the investment and further advances on the horizon, including artificial pancreas technologies, new therapies for diabetic retinopathy, and breakthroughs in laboratory production of β-cells. The recent program extension enables us to build on this foundation and pursue key new initiatives to harness emerging technologies and develop the next generation of type 1 diabetes researchers.


Two decades ago, the results of the Diabetes Control and Complications Trial (DCCT) revolutionized modern-day treatment of type 1 diabetes by demonstrating that intensive glycemic control, beginning as soon as possible after diagnosis and compared with conventional care at that time, prevented or delayed the development of complications of the eyes, kidneys, and nerves.[1] This result precipitated a major research effort to develop approaches to achieve near-normal glycemic control safely in real-world settings, an effort that became increasingly urgent with subsequent demonstration of the enduring and expanding benefits of glycemic control. Over 30 years after the DCCT began, critical insights continue to emerge from this study about the importance of intensive glycemic control. Results from the DCCT follow-on study, the Epidemiology of Diabetes Interventions and Complications (EDIC) study, demonstrated that the finite 6.5-year period of intensive glycemic control provided enduring protection from later-stage microvascular complications after 30 years,[2] as well as protection from cardiovascular disease.[3] Recently, the EDIC study found that intensive glycemic control reduced deaths by 33% in the intensive treatment group compared with the standard treatment group,[4] even though glycemic control converged to similar levels in the two groups in the two decades after the trial ended in 1993. Notably, this study found that higher average blood glucose levels and increased proteinuria were major risk factors for death, demonstrating the importance of glycemic control and reductions in diabetes complications to longer and healthier life spans for people with type 1 diabetes. These results have transformed clinical care for people with type 1 diabetes, with doctors now recommending that people with the disease practice intensive control as early in the course of the disease as safely possible. Yet despite its dramatic health benefits, early intensive glycemic control remains burdensome and elusive, making new strategies for prevention, cure, and treatment of type 1 diabetes imperative.