Is the best way to beat type 1 diabetes to simply stop it before it starts? That’s a question being tackled in new clinical trial that proposes to prevent diabetes in those prone to developing it.
“Often times, the best answer is the simple answer,” says Ezio Bonifacio, Ph.D., from the Center for Regenerative Therapies, in Dresden, who led a team of researchers to develop a vaccine to prevent type 1 diabetes. The vaccine has ended up being insulin itself.
In what is called the Pre-POINT study, twenty-five children aged two to seven in four countries, who were identified as likely to develop type 1 diabetes, were given doses of powdered insulin. The children receiving the highest dose of powdered insulin (so it would be absorbed in the mucous membranes in the mouth) developed an immune response that kept their immune systems from attacking insulin-producing beta cells, a process which leads to type 1 diabetes.
“This is a revolutionary way to prevent type 1 diabetes,” says Prof. Anette-Gabriele Ziegler from the Institut für Diabetesforschung, who was part of the study, “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 next phase of the clinical trial will not merely test more children, but younger children who do not yet have fully developed immune systems. Bonifacio says he will seek to test children aged six months.
That phase calls for children at study centers in Germany, Austria, England, Canada, and the United States to receive the treatment for between three and 18 months. After completion of Pre-POINT, Bonifacio and his team will move on to the next phase of the study called, appropriately enough, POINT. That phase will seek to test the treatment in children over a longer period of time than pre-POINT. Additionally, children who participated in pre-POINT can be considered to take part in the POINT study.
Bonifacio credited recent advances in genetic testing for the likelihood that it would be much easier in the future to determine who should receive the vaccine.
In the initial study the criteria for determining which children were more prone than others to develop type 1 diabetes included having two members of one’s family who developed diabetes and testing for specific genetic markers that presage type 1 diabetes.
“In 10, 15, or 20 years from now instead of all those criteria it might be we do a genetic test at birth to determine who is most likely to develop diabetes,” says Bonifacio. “Based on that test, we can then administer the vaccine as needed.”
Writing in an editorial in the April 21, 2015 issue of the Journal of the American Medical Association (where the pre-POINT study results were also published) Dr. Jay S Skyler, of the from the Diabetes Research Institute at the University of Miami’s Miller School of Medicine in Florida, said he was “cautiously optimistic” about the pre-POINT findings.
“The promise of autoantigen-specific therapy for prevention of type 1 diabetes in humans has yet to be realized,” Skyler wrote. Despite this, “the Pre-POINT study provides additional evidence to inform trial design and increases enthusiasm for cautiously moving forward with a study of primary prevention in genetically screened children.”
The Pre-POINT study has thus far established that the treatment is safe. Bonifacio said there were no adverse side effects from children ingesting powdered insulin.
The study is moving forward with significant support from at least two established organizations dedicated to researching cures for diabetes. The Juvenile Diabetes Research Foundation, and the Helmsley Charitable Trust are supporting the follow-up to the Pre-POINT study.
“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 type 1,” Julia Greenstein, vice president of Discovery Research at JDRF said in a news release. “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.”
“It has taken almost 15 years for us to get to this point with Pre-POINT,” says Bonifacio, who is considered the preeminent researcher into developing a diabetes vaccine, “but we’re determined to see it through.”