Never before has a drug against type 1 diabetes in children been needed as urgently as it is now. In Germany alone, 3,700 minors contract diabetes every yearbecause their immune system is destroying the insulin-producing cells in their pancreas. As a rule, they have to inject the hormone that regulates blood sugar levels for the rest of their lives. And every year the rate of newly diagnosed children increases. The problem was exacerbated during the pandemic. Between the beginning of 2020 and the middle of 2121, the number of fresh diagnoses has increased again for reasons that are not yet known by 15 percent shot up.
The cause of the disease is an autoimmune reaction, in which the immune system attacks its own body. The disease affects children who are genetically particularly susceptible, for example because they have parents with type 1 diabetes. So far, all attempts to prevent the outbreak of the disease with medication have failed.
In the United States, for the first time, a drug is now available that can do this – at least temporarily. At the end of last week, the FDA approved the drug teplizumab. This is an antibody that is directed against certain immune cells, so-called T-lymphocytes, and in this way triggers a kind of restart in the immune system. When the body rebuilds the destroyed T-cells in the weeks following the administration of the drug, the number of those lymphocytes that suppress autoimmune reactions and thus the attack on the insulin-producing cells in the pancreas increases.
The approval could mark the beginning of a new era.
According to a study in the journal New England Journal of Medicinee it is not possible in this way to completely prevent the outbreak of the disease; however, the drug manages to delay the onset by an average of two years. For Anette-Gabriele Ziegler, Director of the Institute for Diabetes Research at Helmholtz Zentrum München, the approval of teplizumab is “an absolute milestone” in the treatment of type 1 diabetes. “For the first time, a drug is available that is already effective in the early stages of the disease.”
She describes the approval as the “beginning of a new era” because she assumes that this or similar approaches may be able to achieve even more. For example, attempts are currently being made to reset the immune system again later and thereby postpone the outbreak of the disease even further – with teplizumab or other drugs with a similar effect. “Another step will be to use the antibodies in even younger children,” says the diabetologist. Teplizumab is approved in the USA for children over the age of eight. However, the first signs of the autoimmune reaction can often be seen at the age of one or two years. Ziegler expects “that we will see even greater effects when treating younger children”.
According to the approval study, no serious side effects have occurred so far: the temporary dwindling of the lymphocytes had not led to an increase in infections with pathogens. Every third person treated suffered from a temporary skin rash.
Early detection programs would be necessary
The drug is not expected to be approved in Europe before the end of next year. But it can already be said that it would pose new challenges for the healthcare system: on the one hand, the drug is very expensive. The American Daily Newspaper New York Times reports that the 14-day infusion therapy will cost the equivalent of almost 190,000 euros.
Also, it necessitates early detection programs to spot the children who need treatment in time. In the UK, for example, one in four children with type 1 diabetes is not diagnosed until they are admitted to a clinic with serious complications. It’s clearly too late. For this reason, too, Anette-Gabriele Ziegler established a type 1 diabetes screening program together with other scientists in Bavaria, Lower Saxony and Saxony. This is an antibody test as part of the regular check-ups for children. So far, this has been financed from research funds. “Then,” demands the diabetes expert, “that would have to be covered by health insurance in the future and offered to all children.”