New York, NY — The Leona M. and Harry B. Helmsley Charitable Trust today announced five new grants that will support the European, multinational Global Platform for the Prevention of Autoimmune Diabetes (GPPAD; “gee-pad”) and its first trial – the Primary Oral Insulin Trial (POInT). Leading GPPAD will be Professor Dr. Anette Ziegler, Director of the Institute of Diabetes Research, Helmholtz Zentrum München and Technical University München.
Launched in 2015, the Helmsley Type 1 Diabetes (T1D) Prevention Initiative aims to support the discovery and development of interventions to prevent the disease. The GPPAD network is designed to advance the goals of the T1D Prevention Initiative by identifying newborns with a high genetic risk of developing T1D autoimmunity and conducting primary prevention clinical trials. Primary prevention aims to prevent the onset of autoimmunity associated with T1D.
Helmsley’s investment of over $52 million in grants has been awarded to the Helmholtz Zentrum München with its partners, including the Technical University München, Lund University, the University of Oxford, Katholieke Universiteit Leuven, Technische Universität Dresden, Hannoversche Kinderheilanstalt, Instytut Matki i Dziecka, and Warszawski Uniwersytet Medyczny.
In addition to supporting the existing GPPAD network already in place in Germany, Helmsley grants will fund expansion in Germany and start-up activities in Sweden, the United Kingdom, Belgium, and Poland to begin genetic testing of newborns. The participating institutions will identify infants with a significant genetic risk for type 1 diabetes to begin recruiting for POInT, a randomized controlled phase IIb trial to determine if daily administration of high-dose oral insulin in early life can prevent T1D-associated autoimmunity. To fully enroll POInT, over 300,000 newborns will be tested across all participating institutions. The study is also expected to provide a bioresource to share with scientists who study the causes of early autoimmunity and type 1 diabetes.
GPPAD, expected to be a major advancement in the field of T1D, provides a multicenter platform for both large and small T1D primary prevention trials and for investigating the immune system in a crucial period before autoimmunity starts. In the long term, it is hoped that the current work will lay the foundation for potential expansion to other childhood diseases, with an overarching goal of promoting health in early life.
“This international partnership is a tremendous opportunity to test strategies to prevent type 1 diabetes and advance knowledge about the development of this chronic disease,” said Gina Agiostratidou Program Director of Helmsley’s Type 1 Diabetes Program. “We are proud to invest in the future of T1D prevention, especially now as the incidence of this disease is increasing.”
Günther Wess, CEO of the Helmholtz Zentrum München that is also investing resources into this project, commented, “This is a very important step in advancing decades of hard work in basic and clinical science to make a difference for patients and stop type 1 diabetes. Prevention is the future of medicine. GPPAD is setting a new paradigm in finding novel therapeutic approaches by closely bridging science, medicine, and society. This is the way we need to go to open new avenues for promoting health, both in children and adults.”
“The trial targets the first years of life, a period when the immune system is learning what is safe versus what is dangerous. Through oral tolerance, we have a real chance to help the immune system learn that insulin is self and thereby correct the defect that leads to autoimmunity,” said trial leader Anette Ziegler from the Helmholtz Zentrum München and the Technical University München.
“This is an enormous investment. It brings together 30 years of genetic and immunology research with important findings from scientists who are now part of GPPAD, and takes it to the people in one of the largest ever efforts in early prevention of type 1 diabetes,” said Ezio Bonifacio, Director of the Center for Regenerative Therapies Dresden at the Technische Universtät Dresden.
“The progress achieved in just over three years for a program of this magnitude and complexity is truly incredible. The trial is unique and the platform will have wide-ranging implications not only for type 1 diabetes but also in the future for other childhood diseases,” said John Todd, Professor of Precision Medicine at the University of Oxford.
“The support from Helmsley will make it possible to reinstitute newborn screening in a way that’s never been done before to identify children at increased genetic risk to participate in unique prevention clinical trials,” said Åke Lernmark, Professor of Diabetes Research at Lund University in Sweden.
“Prevention of T1D-associated autoimmunity is likely to be the most effective way to reduce the incidence of this disease,” said Anne Koralova, Program Officer of Helmsley’s Type 1 Diabetes Program. “With these grants, we are excited to support such important and exciting work in the prevention space.”