100 000 babies screened for increased type 1 diabetes risk in Freder1k Study
It is a major milestone for one of Europe’s biggest health research cooperations: The international platform GPPAD (“The Global Platform for the Prevention of Autoimmune Diabetes”) has successfully screened 100,000 newborns across Europe for an increased genetic risk of developing type 1 diabetes. Participant number 100,000 is a two week old baby from Dresden, Germany.
The research team in Munich celebrates the 100 000. participant @Helmholtz Munich
If an increased risk is detected, the children are offered to take part in a prevention trial with oral insulin (‘POInT’). The goal: to delay or even prevent the manifestation of the autoimmune disease type 1 diabetes. Study sites in five European countries (Germany, Belgium, Poland, Sweden and Great Britain) started working on the innovative trial in November 2017, among them the research team around Prof. Dr. Anette-Gabriele Ziegler, Director of the Institute of Diabetes Research at the Helmholtz Munich.
“We are thrilled that we have now screened 100,000 children with GPPAD and are grateful to all the hospitals and clinics involved”, Prof. Ziegler says. “This number shows us that families have great interest in learning about their children’s risk status. It also means we are right on track towards the target of screening 330,000 newborns Europe-wide by 2022. And of course we are hopeful that our treatment approach of the prevention trial ‘POInT’ works. This would mean a big step towards our vision of a world without type 1 diabetes. But already today the families benefit greatly from early detection – through counseling and the excellent medical care that all of our study sites provide. Even if some of the children should develop type 1 diabetes at some point, the families will avoid possible grave complications and therefore improve the general course of the disease.”
The Meiringer family, whose twins Ben and Daniel participate in the POINT study, adds: “When we learned about the possibility to screen for an increased genetic risk for type 1 diabetes, we were quickly convinced to have our children tested. It is reassuring to know that we’re doing everything we can to increase the chance that they will not have to live with this disease – and of course we’re happy to support the research. At the very least we’re helping other families in the future who are in the same place as us.”
Only a few drops of blood are needed for the screening as taken directly form umbilical cord at birth or later from the child’s hand or heel. Participation is free and available for newborns up to the age of 4 months. Currently there are 317 young participants enrolled in the prevention trial ‘POInT’ – that is almost half of all children for whom an increased risk has been detected.
“We’re so grateful for the investigators, nurses, and all those who are committed to shedding light on how type 1 diabetes develops and paving the pathway to life without its burdens,” said Gina Agiostratidou, PhD, Type 1 Diabetes Program Director at The Leona M. and Harry B. Helmsley Charitable Trust, the funder of GPPAD. “In 2014, the GPPAD investigators and Helmsley started to work to together to build a new kind of platform for type 1 diabetes trials focused on preventing this lifelong chronic disease from developing. We’re excited to continue our collaboration to make our shared vision of winning the battle against type 1 diabetes a reality.
More about GPPAD: www.gppad.org
Type 1 diabetes: Type 1 diabetes is an autoimmune disease which means that the immune system attacks and destroys insulin-producing “beta cells” in the pancreas. Insulin is a vital hormone that transports sugar attained from ingested food from the blood into the cells. If the body cannot produce enough insulin, sugar will accumulate in the blood after a meal. This can lead to life-threatening metabolism disruptions and people with type 1 diabetes must therefore take insulin injections for life. However, even patients receiving good treatment may suffer from various health issues related to the autoimmune disease. The average life expectancy of a child with type 1 diabetes is reduced by 14 to 18 years.
POInT: POInT (Primary Oral Insulin Trial) is a randomized, placebo-controlled double-blind study recruiting infants aged four to seven months. Children receive a small daily dose of insulin powder or placebo orally together with a meal until age 3. 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.
Die Globale Plattform zur Prävention des autoimmunen Diabetes (GPPAD) wurde 2015 gegründet. GPPAD hat sich zum Ziel gesetzt, eine internationale Infrastruktur für Studien zur Primärprävention von Typ-1-Diabetes zu etablieren. Die Studien zur primären Prävention von Typ-1-Diabetes sollen die Inzidenz der Betazell-Autoimmunität reduzieren und dadurch Typ-1-Diabetes verhindern. Voraussetzung dafür sind Screening-Programme, welche diejenigen Kinder erkennen, die auf Grund einer genetischen Prädisposition ein erhöhtes Erkrankungsrisiko haben. Zu den Mitgliedern von GPPAD zählen das Helmholtz Munich, die Technische Universität München (TUM), das DFG-Forschungszentrum für Regenerative Therapien der Technischen Universität Dresden, die Hannoversche Kinderheilanstalt (Kinder- und Jugendkrankenhaus auf der Bult), die Universitäten von Oxford (Großbritannien), Lund (Schweden), Leuven (Belgien) sowie Instytut Matki i Dziecka und Medizinische Universität Warschau (beiden Polen).
As German Research Center for Environmental Health, Helmholtz Munich pursues the goal of developing personalized medical approaches for the prevention and therapy of major common diseases such as diabetes mellitus, allergies and lung diseases. To achieve this, it investigates the interaction of genetics, environmental factors and lifestyle. The Helmholtz Munich has about 2,500 staff members and is headquartered in Neuherberg in the north of Munich. Helmholtz Munich is a member of the Helmholtz Association, a community of 19 scientific-technical and medical-biological research centers with a total of about 37,000 staff members.