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 blocked that enzyme and also prevented 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. Read more.