The JAMA published Pre-POINT study tested the effects of prophylactic insulin oral immunization in young children with a family history of type 1 diabetes

New York, NY, April 21, 2015 – Children with type 1 diabetes (T1D) must inject insulin several times a day for the rest of their lives because their own immune systems have destroyed the pancreatic cells that produce insulin. The immune system normally learns to ignore its own proteins in a process known as tolerance. Early exposure to these self-proteins usually causes the body to produce protective cells that prevent the immune system from destroying its own cells. This goes wrong in children who develop T1D. Instead of protective cells, the children make an immune reaction against insulin and other proteins of the pancreas.

In the Pre-POINT study published in the April 21 issue of JAMA, Ezio Bonifacio, Ph.D., from the Center of Regenerative Therapies in Dresden, Germany, and his colleagues in Austria, the United States and the United Kingdom, have taken important steps to coax the body into making the protective responses that should prevent children from developing T1D. They gave children age 2 to 7 years old with a very strong family history of T1D increasing doses of insulin in their food every day for an average of six months, and found that the highest dose of insulin used in study could modify the immune response. Instead of making the immune response seen in T1D, the children had features of the response that have the potential to protect against the disease.

“This is the first time we are seeing any sort of response by the immune system to orally administered insulin in children,” said Bonifacio, who led to study. “We had to use relatively large amounts of insulin and we were pleased to see that there were no unwanted side effects and thus far only signs that mimic what normally happens in children who don’t get type 1 diabetes. Many people thought that the treatment would fail because the insulin would be broken up by the time it gets through the stomach. However, we believe that most of the response is happening while insulin is still in the mouth. The same seems to be true in some recent studies when increasing doses of protein were given to children with peanut allergy.”

“JDRF is very encouraged by the results of the Pre-POINT study as a first step to potentially prevent type 1 diabetes in children that are at high risk for getting T1D,” said Julia Greenstein, vice president of Discovery Research at JDRF, which helped to fund the study. “This is a significant finding, and given JDRF’s mission to achieve a world without type 1 diabetes, these study results are exciting and bring us one step closer to the potential of seeing  an oral vaccination strategy to prevent type 1 diabetes.”

Professor Anette Ziegler, M.D., from the Helmholtz Zentrum Munich comments: “Unique in our study is that we gave the insulin as a prophylactic vaccine to children before they had any signs of the destructive immune response that leads to type 1 diabetes. This is quite a big change to the way people have been thinking, but it is quite logical that if the body’s immune system doesn’t learn how to make the protective responses by itself we need to give it a little help.”

The Pre-POINT study was designed to determine the amount of insulin that could stimulate the immune system to produce a protective response. The investigators plan to continue testing this dose range of insulin in a larger number of genetically at-risk babies.  If these results continue to show impact on the immune system, they eventually hope to launch a larger and longer study to test whether the protective immune response induced by oral exposure to insulin actually prevents T1D. If they are successful, oral vaccination of at-risk children with insulin may provide a means for controlling the worldwide increasing incidence of childhood T1D.

About JDRF
JDRF is the leading global organization focused on type 1 diabetes (T1D) research. JDRF’s goal is to progressively remove the impact of T1D from people’s lives until we achieve a world without T1D. JDRF collaborates with a wide spectrum of partners and is the only organization with the scientific resources, policy influence and a working plan to bring life-changing therapies from the lab to the community. As the largest charitable supporter of T1D research, JDRF has invested nearly $2B in research over the past 45 years and is sponsoring scientific research in 17 countries worldwide. For more information, please visit jdrf.org

Media Contact:
Christopher Rucas
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Email: crucas@jdrf.org