Prevention of any disease is challenging, and this is certainly true for T1D. Preventing T1D requires retraining the immune system. Specifically, it needs to deter the autoimmune attack on the body that destroys the insulin-producing beta cells in the pancreas, which results in T1D. Identifying prevention therapies is a long-term endeavor fraught with challenges, yet ultimately with great reward – and a priority for Helmsley. We anticipate that the research efforts we support will uncover new biomarkers of disease susceptibility along the way, in addition to other discoveries.
Preventing a disease requires first understanding the mechanisms that cause it, as well as knowing who is at risk for developing it. There are currently two common ways to determine if an individual is at risk of developing T1D. One is to screen for genetic predisposition. The second is to test for autoantibodies. Other metabolic measures can also help to stratify risk.
If someone is shown to have genetic risk, they can at a minimum be monitored to determine if they are crossing the threshold to autoimmunity. For those where autoimmunity is present, they can be monitored for changes in blood sugars that signal onset of clinical disease, with a goal of minimizing diabetic ketoacidosis, a potentially life-threatening complication too often present at diagnosis. There is a fundamental public health benefit to helping more people understand their potential risk for T1D and minimizing poor outcomes.
Monitoring is an important option, but far from sufficient for such a life-changing condition. Over the past decade Helmsley has partnered with organizations around the world to carry out clinical prevention trials. These include novel efforts to delay autoimmunity and are led by investigators who have expertise in T1D pathogenesis, immunology, and pancreatic biology, and who are also committed to understanding what triggers T1D in the first place.
Our largest investment has been in Europe to support the Global Platform for the Prevention of Autoimmune Diabetes (GPPAD); it has sites in Germany, the United Kingdom, Belgium, Sweden, and Poland. The network identifies newborns with a high genetic risk for developing T1D and offers participation in clinical trials to prevent it.
Helmsley also invests in discovering and developing new drugs that can halt or delay progression of T1D, before insulin is needed. Our partners span from academic research labs to biotech companies.
An important part of discovering new drugs is understanding the development of T1D in humans, not just in laboratory models. Understanding the human disease can reveal promising drug targets that are the most likely to be successful in clinical trials. For example, we fund studies examining unique clinical cases of T1D, such as families with a high prevalence of the disease, or long-term non-progressors—individuals with autoimmunity but who take years longer than most to progress to diabetes. These special cases may shine a spotlight on a key aspect of T1D development, and lead to new generalizable therapeutic strategies.
We also fund the development of new therapies, pushing them from the laboratory to clinical trials. A promising class of drugs is targeted immunotherapies, which are designed specifically to tamp down autoimmunity and prevent further damage to pancreatic tissue while leaving other tissues untouched. These targeted immunotherapies are designed to be safer and more powerful than many existing drugs, which suppress both autoimmune and healthy immune reactions.
People living with type 1 diabetes and their loved ones know that the daily burden of this disease is significant. The Helmsley-funded INNODIA consortium is expanding our scientific knowledge of T1D to transform its trajectory.
What if we sought new discoveries by investigating the exceptions to the rule, the clinical outliers that are often ignored?
Ben Williams, Ph.D., T1D Program Officer
February 3, 2023
April 20, 2022
April 5, 2022
