Discovery of a promising drug candidate for the prevention of type 1 diabetes


Researchers at the University of Helsinki (Finland) studied the mechanisms of action of risk genes associated with type 1 diabetes with the aim of finding new methods of preventing the disease.

According to this study, published in the scientific journal ‘Nature Communications’, type 1 diabetes could be prevented by inhibiting a gene associated with the onset of the disease.

A drug based on the same mechanism has already been approved for the treatment of another autoimmune disease, psoriasis, in the United States, which could prompt the development of drug therapies for diabetes.

The incidence of type 1 diabetes is higher in Finland than in any other country. Although hundreds of genes underlying the disease have been identified, the mechanisms of action of most of them remain unclear.

In other words, it is possible to identify individuals at high risk of developing the disease, but so far no effective way to prevent its progression without the risk of significant adverse effects has been found.

“We focused on a gene known as TYK2 because its mutations are known to protect against type 1 diabetes,” lead researcher Timo Otonkoski said.

Researchers hypothesized that inhibiting the expression of the TYK2 gene could reduce the destruction of pancreatic beta cells that leads to the onset of type 1 diabetes. Pancreatic beta cells produce vital insulin for the body, that diabetic patients must replace with insulin injections.

The hypothesis was confirmed in the study and the destruction of beta cells was effectively stopped by inhibiting the expression of TYK2. “The destruction of beta cells is the result of an autoimmune reaction in which white blood cells activated by the body’s immune system attack their own tissues. With the TYK2 inhibitor, the havoc wrought by white blood cells was markedly reduced,” Otonkoski confirmed.

Another surprising finding from the study was that removing the effect of the TYK2 gene caused a reduction in the differentiation of pancreatic islet cells, i.e. a reduction in the production of beta cells.

“However, this adverse effect was only observed in the early stages of beta cell development. Inhibition of TYK2 expression did not affect the functioning of mature beta cells,” said Dr. Vikash Chandra, first author of the paper.

Previously, Otonkoski’s research group successfully developed fully functional pancreatic beta cells from stem cells that can be used to restore the body’s insulin production.

Based on the results, a TYK2 inhibitor could be a promising drug candidate for the prevention of type 1 diabetes.

“We used not only cells that had the TYK2 gene knocked out, but also a chemical TYK2 inhibitor. This pharmacological molecule has already been approved in the United States for the treatment of psoriasis,” says Otonkoski.

According to the researcher, the effect of TYK2 inhibitors on the prevention of diabetes must be carefully studied in animal models. If the results are positive, the next step towards clinical trials can be taken.


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