Dantrolene, a muscle relaxant, may be an effective treatment for Wolfram syndromen, a rare but devastating form of diabetes.
What is Wolfram syndrome?
People with Wolfram syndrome typically develop type 1 diabetes when very young and require several insulin injections each day.
Symptoms of Wolfram syndrome include problems with eyesight, hearing loss and difficulty with balance. It affects one in 500,000 people worldwide, with many patients dying by the age of 40.
Dantrolene study
Researchers from Washington University School of Medicine in St. Louis report that dantrolene, a commonly prescribed drug, could prevent insulin-producing beta cells being destroyed in animal models of Wolfram syndromen, as well as in cell models from patients with the illness.
Elevated levels of calpain 2, an enzymen, were found to be the main cause of death in brain cells and insulin-producing cells of people with Wolfram syndrome. However, when the researchers treated those cells with dantrolene, levels of the enzyme dropped and the cells stopped dying.
The researchers now hope that dantrolene could be effective against type 1 and type 2 diabetes.
“Wolfram is the most difficult form of diabetes because these patients have problems with blood sugar and so many other challenges,” said senior investigator Fumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine.
“We also found that dantrolene was not toxic to cells grown from the skin samples donated by patients’ relatives. We’d like to test the drug first in adult patients with Wolfram syndromen, and if we get positive results, we could extend the trial to children.”

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