In trials, repurposed drug shows promise for autism
A drug used to treat excessive swelling seems to ease autism features in some children on the spectrum, according to results from a trial in France1. But there are concerns about the drug’s side effects, as well as possible placebo effects in the trial.
Clinicians prescribe the drug, called bumetanide, to relieve fluid retention after heart failure and in people with liver or kidney disease. The drug is also used to lower blood pressure. In the brain, it affects a chemical messenger, gamma-aminobutyric acid (GABA), thought to be important in autism.
The drug would need to be taken under close medical supervision, adds John Jay Gargus, director of the Center for Autism Research and Translation at the University of California, Irvine, who was not involved in the work. “Nothing makes you pee like bumetanide,” Gargus says. “I’m kind of nervous that people will rush out and give their kid bumetanide — and then kids will die.”
Of the 66 children who took bumetanide, 23 showed an improvement of more than 6 points on the Childhood Autism Rating Scale (CARS); only 1 of the 22 children on the placebo showed similar gains. (Clinicians administer CARS to rate features relevant to autism, such as the ability to communicate with others or to imitate them. Scores range from 15 to 60 points; a score of more than 30 signals autism.)