Promising breakthroughs in basic and translational research on the immunologic defect in type 1 diabetes have led to the development of new drugs to treat, and potentially reverse, newly diagnosed type 1 diabetes. These agents — exemplified by the anti-CD3 drugs — represent a robust pipeline for testing in clinical trials. A substantial increase in funding for this goal over the next 5 years is needed to provide the resources to test these new agents, first in phase I and II trials to determine safety and assess efficacy. Drugs showing the most potential to reverse type 1 diabetes will then need to be tested in large, phase III trials to secure regulatory approval and make effective therapies widely available.

REVERSAL OF NEW ONSET TYPE 1 DIABETES
By the time type 1 diabetes is diagnosed, patients have already suffered a devastating autoimmune attack that has destroyed the majority of the insulin-producing beta cells of the pancreas. At onset, the beta cells that remain are insufficient to maintain proper glucose control and the cells continue to die off from an ongoing immune-mediated, self-destructive reaction. Recent research has shown that a patient's level of residual beta cell activity at diagnosis correlates with the ability to more easily maintain glucose levels close to normal, which lowers the risk for disease-associated complications.

HOPE AND PROGRESS THROUGH RESEARCH
Clinical trial platforms established with federal funding provided a means to test, and to head-to-head compare many promising new treatments to halt the autoimmune attack in type 1 diabetes. Trials carried out through these and other resources have generated remarkable insights into the disease as well as potential ways to reverse it.

Researchers have recently discovered that, at the time of diagnosis, type 1 diabetes patients have far more insulin-producing islet cells remaining than previously thought. This unexpected finding bolsters the idea that the opportunities for intervening in the disease process are greater than was believed just a few years ago.

Investigators have made astounding progress toward the goal of reversing recently diagnosed type 1 diabetes with a promising new drug that can effectively alter the clinical course of the disease. A short, 1-2 week course of treatment with an antibody — named anti-CD3 — helps patients maintain or increase their ability to produce insulin for up to 18 months after diagnosis compared to a placebo treatment.

Anti-CD3 is just the leading edge of a robust pipeline of potential therapies for reversing new onset type 1 diabetes.

Drugs originally designed for use in patients with other clinical disorders, such as Byetta (for type 2 diabetes) or rituximab (for rheumatoid arthritis), are being tested in patients with recent onset type 1 diabetes. Repositioning drugs approved for cancer, transplantation, and other indications for use in type 1 diabetes has the potential to speed up the timeline and reduce the costs of developing new treatments.

Clinical researchers have launched the first stem cell based therapy for type 1 diabetes, utilizing cells from umbilical cord blood to regulate the immune system of newly-diagnosed type 1 diabetes patients.

The establishment of collaborative clinical trial networks has greatly enhanced the research community's ability to quickly and efficiently conduct large trials of potential new therapies. Importantly, newly developed "core laboratories" allow researchers to directly compare results and samples from multiple trials.

It has become clear that patients enrolled in clinical trials have better outcomes in terms of their diabetes management and glucose control over the short-to-intermediate term compared to patients who are not participating in clinical trials, even when the trial is considered "unsuccessful" at reversing disease.

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