The Washington University School of Medicine in St. Louis is beginning a new clinical trial of the drug known as dantrolene to treat patients with Wolfram syndrome.

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Wolfram syndrome is a genetic disorder where a patient develops diabetes at a premature age and requires insulin injections every day. The disease also results in hearing loss, vision problems and difficulty with balance.

Dantrolene is a muscle relaxant that works by restoring a normal level of calcium in the muscles, thereby reducing a rise in body temperature. It is indicated to treat cerebral palsy, multiple sclerosis and muscle spasticity.

The drug demonstrated efficacy in treating Wolfram syndrome by preventing destruction of insulin-secreting beta cells in animal models, and in brain cells differentiated from skin samples taken from patients afflicted with the disease.

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"Nobody has ever tested dantrolene in patients with Wolfram syndrome, so our first and most important objective is to make sure it is safe."

The researchers at Washington University School of Medicine in St. Louis will test the safety of dantrolene, while examining 24 patients comprising 12 adult and 12 pediatric patients who exhibit symptoms of the disease.

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The patients will be subjected to extensive testing before and after being administered with the drug and the researchers will monitor the patients’ vision and brain function, as well as the function of their remaining insulin-secreting beta cells.

Principal investigator Fumihiko Urano said: “Nobody has ever tested dantrolene in patients with Wolfram syndrome, so our first and most important objective is to make sure it is safe.

“I am very hopeful, however. The major question that I get from every patient I see is, is there any treatment? And until now, I have had to say no. With any luck, perhaps this study can help change that.”

Image: Dantrolene is a muscle relaxant approved to treat patients with cerebral palsy, multiple sclerosis and muscle spasticity. Photo: courtesy of ROBERT BOSTON.