Researchers from the Thames Valley contribute to international success
It is a major milestone for one of Europe’s biggest type 1 diabetes research cooperations: The international platform GPPAD (“The Global Platform for the Prevention of Autoimmune Diabetes”) has successfully screened 100,000 newborns across Europe (including over 7500 newborns in the Thames Valley) for an increased genetic risk of developing type 1 diabetes. Participant number 100,000 is two-week-old Arthur from Dresden, Germany.
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 Dr. Manu Vatish, Chief Investigator of the INGR1D study. “We are thrilled that we have now screened 100,000 children with GPPAD and are grateful to all the hospitals and clinics involved”, Dr. Vatish 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.”
Surplus blood from a baby’s newborn bloodspot screening card (taken on day five of a child’s life) is used for the screening. Participation is free and available for newborns up to the age of 3 months. Currently there are 317 young participants enrolled in the prevention trial ‘POInT’ – that is a third of all children for whom an increased risk has been detected.
“We’re so grateful for the investigators, midwives, 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 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.
You can read further information on GPPAD here.
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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.
GPPAD: The Global Platform for the Prevention of Autoimmune Diabetes (GPPAD) was initiated in 2015. It provides an international infrastructure that enables type 1 diabetes primary prevention trials. These trials are built around programs that identify infants with an elevated genetic predisposition for type 1 diabetes, and aim to reduce the incidence of the beta-cell autoimmunity that precedes clinical diabetes in children. The research is focused on newborn screening in order to identify at-risk children before an autoimmune attack sets in. GPPAD is funded by The Leona M. and Harry B. Helmsley Charitable Trust.
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.