WHAT IS GPPAD?
The Global Platform for the Prevention of Autoimmune Diabetes (GPPAD) was initiated in 2015. Its goal is to provide an international infrastructure that will enable type 1 diabetes primary prevention trials. These trials will be built around programs that identify infants with an elevated genetic predisposition for type 1 diabetes, and will aim to reduce the incidence of the beta-cell autoimmunity that precedes clinical diabetes in children.
The incidence of childhood type 1 diabetes continues to increase. GPPAD’s long-term vision is to stop this trend. GPPAD understands that new approaches are necessary in order to achieve its vision. There must be a global action built around a platform that coordinates efforts to launch controlled prevention trials. The effort should be focused on prevention of the autoimmunity that precedes and destroys the insulin-producing cells, eventually leading to type 1 diabetes. Treatment must commence in infancy or earlier to prevent the autoimmunity that often presents at 9 to 24 months of age.
GPPAD will facilitate this by establishing linked early genetic testing and preventive programs.
Simple genetic testing can identify newborns with 25-fold increased risk of type 1 diabetes. Families of high-risk neonates can participate in a clinical study that will test the efficacy of oral insulin to prevent autoimmunity and type 1 diabetes
Importantly, the GPPAD vision requires the participation and engagement of clinicians, scientists, government, laypersons, and the public at large. Prevention is indeed a global effort, and GPPAD’s success will rely on its ability to integrate and inform all these parties of the need and the means to bring about prevention of a lifelong disease that, in western countries, affects 4 in every 1000 children.
Early detection of the risk of developing type 1 diabetes
Neonates and infants who are at increased risk to develop type 1 diabetes can now be identified using genetic markers. Testing requires only a few drops of blood, which can be drawn with the regular newborn screening or at a pediatric visit up until the age of 4 months. Alternatively, also cord blood can be used at delivery. Families of infants with a 25-fold increased risk for type 1 diabetes will be contacted by their local GPPAD team. They are being informed about the meaning of an elevated risk for type 1 diabetes, educated about symptoms of the disease as well as asked to participate in a randomized controlled trial aiming to prevent type 1 diabetes.
In this trial called POInT (primary oral Insulin trial), children receive a small daily dose of insulin powder orally together with a meal. The goal is to introduce immune tolerance to insulin, as insulin and the cells that produce insulin in the pancreas are the primary targets of the destructive autoimmune reaction that characterizes type 1 diabetes. Previous studies have shown that oral administration of insulin is safe and does not affect plasma glucose levels.
Preliminary studies and experiences from allergy research show that this approach may be successful. However, there is currently no guarantee of this. POInT is a placebo-controlled double-blind study. This means that half of the children taking part in the study take insulin powder, while the other half receive a placebo (capsules containing an inactive substance). Neither the study participants nor the doctors know which capsules contain insulin and which contain placebo. This is important in order to be able to assess the efficacy of the treatment.
>> Take advantage of this opportunity: have your child tested for the risk of developing type 1 diabetes so that they may participate in the POInT study and receive what we hope will become preventive treatment for all babies who are at increased risk for this life-long disease.
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GPPAD is funded by The Leona M. and Harry B. Helmsley Charitable Trust. The Helmsley Charitable Trust aspires to improve lives by supporting exceptional nonprofits and other mission-aligned organizations in health and place-based initiatives.
Original publication about GPPAD
A.G. Ziegler, T. Danne, D.B. Dunger, R. Berner, R. Puff, W. Kiess, G. Agiostratidou, J.A. Todd, E. Bonifacio: Primary prevention of beta-cell autoimmunity and type 1 diabetes – The Global Platform for the Prevention of Autoimmune Diabetes (GPPAD) perspectives. Molecular Metabolism 5 (2016) 255-262
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