Muscle Relaxant May Be Viable Treatment for Rare Form of Diabetes

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Newswise A commonly prescribed muscle relaxant may be an effective treatment for a rare but devastating form of diabetes, researchers at Washington University School of Medicine in St. Louis report.

The drug, dantrolene, prevents the destruction of insulin-producing beta cells both in animal models of Wolfram syndrome and in cell models derived from patients who have the illness.

Results are published Nov. 24 in the Proceedings of the National Academy of Sciences (PNAS) Online Early Edition.

Patients with Wolfram syndrome typically develop type 1 diabetes as very young children and need insulin injections several times each day. The syndrome also causes hearing loss, vision problems and difficulty with balance.

The researchers found that elevated levels of an enzyme called calpain 2 were the main cause of death in brain cells and insulin-producing cells. Dantrolene blocks that enzyme and also prevents brain cell death in animal and cell models of the disorder.

Because the drug is approved by the Food and Drug Administration (FDA), clinical trials in Wolfram patients could get underway relatively quickly, said senior investigator Fumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine.

Wed like to test the drug first in adult patients with Wolfram syndrome, and if we get positive results, we could extend the trial to children, he said.

Dantrolene often is prescribed to people with cerebral palsy or multiple sclerosis to treat muscle spasticity. If effective in patients with Wolfram syndrome, dantrolene also may be a viable treatment for patients with more common forms of diabetes. Calpain 2, which is involved in calcium metabolism, is overactive in cell models of those more common forms of diabetes.

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Muscle Relaxant May Be Viable Treatment for Rare Form of Diabetes