As the incidence of type 1 diabetes (T1D) rises worldwide, The Leona M. and Harry B. Helmsley Charitable Trust today announced the launch of an ambitious new T1D Prevention Initiative to investigate the early stages of development of the disease and identify new pathways to prevent it. As part of this new effort, the Trust is making an initial investment of over $8.7 million in international grants to support new and existing prevention research across five organizations: Helmholtz Zentrum München, the University of Cambridge, the University of South Florida, JDRF Australia and Keck Medicine of the University of Southern California (USC).
Together, these institutions will initiate and continue observation of hundreds of thousands of newborns and children across the U.S., Europe and Australia; design a plan to analyze the massive quantity of data that chart the history and progression of T1D; and develop an infrastructure to perform intervention clinical trials with a primary focus to delay or stop the development of T1D.
The combined investment in international resources, population-based patient identification and big data technology signals an important new undertaking to understand and prevent T1D.
"While curing T1D has been a research focus for many decades, that goal is still a long way off unfortunately. The launch of this initiative represents the first steps in a long-term effort to prevent this chronic disease," said David Panzirer, a trustee of the Helmsley Charitable Trust. "We are delighted to support ambitious observational studies like TEDDY and ENDIA, and through new European partnerships we are eager to develop an infrastructure to perform intervention clinical trials, which is one of the main goals of the T1D Prevention Initiative."
The launch of the T1D Prevention Initiative corresponds with the release of a white paper by the Helmsley Charitable Trust that documents opportunities for prevention research in the current field of T1D science.
"The science, technology and patient access for studying T1D are now sufficiently advanced that we have an opportunity to begin cracking open the 'black box' of how humans develop this disease," said Gina Agiostratidou, a Senior Program Officer in the Trust's T1D Program, who will be coordinating this grantmaking strategy for the Trust. "We are optimistic that a robust investment in primary prevention could yield dramatic steps toward understanding and, ultimately, stopping the disease."
ABOUT THE GRANTS
Helmholtz Zentrum München, German Research Center for Environmental Health- Amount: $762,323; Term: Two Years
The Chancellor, Masters and Scholars of the University of Cambridge- Amount: $885,765; Term: Two Years
One of the main strategic goals of the T1D Prevention Initiative is to develop an infrastructure to perform T1D prevention clinical trials that will help understand the development of the disease and identify ways to prevent it. In search of the optimal location that has a large enough population and a cost effective health care system, the Helmsley Charitable Trust has identified that Europe has the ability to apply large scale testing for risk prior to the development of type 1 diabetes. The health care systems in Europe also have national structures that facilitate implementation of standardized procedures region-wide.
The project leads are Professor Anette-Gabriele Ziegler, director of the Institute of Diabetes Research at the Helmholtz Zentrum München and Professor of Diabetes at the Technische Universität München, Professor Ezio Bonifacio, Professor at the Center for Regenerative Therapies at the Technische Universitaet Dresden, and Professor Thomas Danne at the Hannoversche Kinderheilanstalt; and Professor John Todd, Professor of Medical Genetics, Dr. Frank Waldron-Lynch, Senior Research Associate and Honorary Consultant in Diabetes and Endocrinology, Dr. Chris Wallace, Senior Research Associate and Wellcome Trust Career Development Fellow, and Professor David Dunger, Professor of Pediatrics at the University of Cambridge.
Jointly, the two institutions will design the infrastructure required for global population-based prevention clinical trials including the development of adaptive clinical trial protocols, identification and implementation of safe and cost-effective interventions and a network to accumulate bio samples.
"We have taken a first step of population-based screening for early stages of type 1 diabetes in Bavaria, Germany within the Fr1da study. The Fr1da network will provide an essential basis for implementing primary prevention into our public health system," said Professor Dr. Ziegler.
"The first steps in primary prevention have been successfully taken with some very encouraging results from the Pre-POINT oral insulin prevention study," said Professor Bonifacio, who is the Dresden partner of the European prevention program and who has pioneered primary antigen vaccination against type 1 diabetes.
JDRF/Australia- Amount: $4,500,100; Term: Three Years
JDRF Australia will partner with the Helmsley Charitable Trust to support a globally unique birth cohort study of type 1 diabetes prevention and provide oversight and tracking of the project progress via the Australian Type 1 Diabetes Clinical Research Network (T1DRCN), a research initiative led by JDRF Australia that has received more than $26 million in funding from the Australian government.
The Helmsley grant will support the Environmental Determinants of Islet Autoimmunity (ENDIA) study, which will investigate candidate environmental exposures and gene-environment interactions that may contribute to the development of islet autoimmunity and type 1 diabetes in early life. Candidate environmental factors included in this study are the microbiome, nutrition, bodyweight/composition, lipid and metabolic biomarkers, insulin resistance, immune function and viral infections. The study will recruit 1,400 unborn infants or infants less than six months of age with a first-degree relative (i.e. mother, father or sibling) with type 1 diabetes across Australia.
ENDIA will be led by Professor Jenny Couper (University of Adelaide / Women's and Children's Hospital, South Australia) and Professor Len Harrison (Walter and Eliza Hall Institute, Victoria, Australia).
ENDIA aims to identify the environmental factors, together with a person's genes, that may influence the development of type 1 diabetes. "We believe children are exposed to the environmental triggers that lead to type 1 diabetes very early in life - perhaps even before they are born," said the study's principal investigator, Professor Jenny Couper.
University of South Florida- Amount: $2,441,656; Term: Two Years
This grant provides essential logistic support for The Environmental Determinants of Diabetes in the Young (TEDDY), which is hosted by the University of Southern Florida, the Coordinating Center for TEDDY. The NIH-funded TEDDY study began in 2002 and has screened over 400,000 newborns and recruited and enrolled over 8,000 eligible newborns into a long-term observational study. To analyze the development of islet autoantibodies, the participants are being followed with blood sampling every three months until the age of four and then at least every six months until the age of 15. Parents collect monthly stool samples in early childhood, fill out questionnaires at regular intervals in connection with study visits, and record information about diet and health in the child's TEDDY Book between visits. Continued long-term follow-up of the TEDDY participants will provide important scientific information on early childhood diet, reported and measured infections, vaccinations and psychosocial stressors.
TEDDY's leadership along with its Data Coordinating Center are under the direction of Dr. Jeffrey Krischer. Through the Helmsley grant project, Dr. Krischer will develop a competitive process to identify investigators who can analyze and integrate TEDDY data to test new hypotheses for the development of T1D. We anticipate at the end of this grant that new hypotheses will be developed, which will help understand the development of T1D and could subsequently identify pathways to prevent T1D.
"The Helmsley funding enables TEDDY to reach out to a broader community of scientists to bring new analytical skills and approaches to the integration and analysis of TEDDY 's comprehensive exposure (the microbiome, the genome, metabolomics, gene expression and proteomics) data and its association with diabetes-related autoimmunity and type 1 diabetes," said Dr. Krischer. "These initiatives represent the largest clinical study of the causes of type 1 diabetes to date."
Keck Medicine of USC- Amount: $169,806: Term: Six Months
Dr. David Agus is a Professor of Medicine and Engineering at the Keck School of Medicine of USC and USC Viterbi School of Engineering.
Through this grant, Dr. Agus and his team, led by Dr. Daniel Ruderman, will review the availability and quality of existing clinical data from completed T1D prevention clinical trials and trials of other autoimmune diseases to determine whether they are appropriate and sufficient to inform the longer-term goal of designing a prevention intervention trial. If this pilot project is successful, a second phase project may follow to guide the design and development of the prevention clinical trial that is developed in Europe.
"My team and I are excited to dive into the clinical trial data and try to learn about the onset of diabetes," said Dr. Agus. "Through the remarkable patients who donated their data, we will hopefully develop a new understanding to better prevent and treat T1D."
About the Helmsley Charitable Trust
The Leona M. and Harry B. Helmsley Charitable Trust aspires to improve lives by supporting exceptional nonprofits and other mission-aligned organizations in health, selected place-based initiatives, and education and human services. Since 2008, when the Trust began its active grantmaking, it has committed more than $1 billion. The Helmsley Type 1 Diabetes Program is the largest private foundation funder of T1D in the United States focused on understanding the disease, developing better treatments and improving care and access. For more information, visit http://www.