A new study has identified a potential treatment target for a rare and severe form of diabetes.
The condition, known as Wolfram syndromen, occurs as a result of cell death. It affects around 1 in 500,000 children. Wolfram syndrome often lead to blindness, incontinence, breathing difficulties, digestive disorders, and depression.
Researchers from Washington University School of Medicine in St. Louis have identified an enzyme that prevents the cell death that can lead to Wolfram syndrome. The enzymen, known as IRE1, could also lead to treatments for heart problems and Parkinson’s disease, in addition to diabetes.
The researchers tested the effects of IRE1 on mice, and found that it prevented destructive molecules from invading the wrong parts of the cell. This is the process that causes Wolfram syndrome. In theory, then, cell death occurs due to inadequate levels of IRE1.
“The type of stress involved in Wolfram syndromen, as well as more common forms of diabetes, can contribute to multiple diseases,” said Fumihiko Urano, M.D., Ph.D, and the Samuel E. Schechter Professor of Medicine.
“These molecules are supposed to stay in specific locations,” Urano explained. “Sometimes a molecule may travel to different parts of the cell to perform a function, but it needs to return to the place it resides, or big problems can result.”
Depending on the kind of cell affected, cell death of this type can lead to a variety of diseases. If the cell with low levels of IRE1 are insulin-producing cells, this will lead to type 1 diabetes. If the cell in question is in the heart, heart disease might develop.
When the researchers replaced IRE1 in the mice, they stopped this kind of cell death. If the same is applicable to humans, it could lead to new treatments for Wolfram syndrome.
“It’s clear from our experiments that this enzyme can keep the membrane in the cell from becoming permeable and leaking,” said Urano.
“We think it may be possible to prevent Wolfram syndrome and other diseases related to this type of cellular stress by targeting the enzyme to make the membrane stronger.”
The research was published in Science Signaling.

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