Bumetanide, a drug that’s long been used to treat high blood pressure, modestly improves social behaviors in children with mild forms of autism, according to the results of a small trial published in December in Translational Psychiatry1.
After three months of bumetanide treatment, 27 children, aged 3 to 11 years, improved more on the Childhood Autism Rating Scale (CARS) than did 27 children taking placebo. Children with severe symptoms of autism did not seem to benefit from the drug, however.
“Of course it doesn’t work for every kid, but the results were satisfactory enough to have virtually all of the parents want to continue when the trial was finished,” says lead investigator Yehezkel Ben-Ari, director of the Mediterranean Institute of Neurobiology at INSERM in Paris, France. He is also the founder and chief executive of Neurochlore, a company based in Marseille, France, that is developing bumetanide and related treatments for autism and other disorders.
Ben-Ari says many parents used the same phrase — “more present” — to describe their children’s behavior after taking bumetanide. The drug is a diuretic — meaning that it increases urine production — but it has no serious side effects, he says. “Some of the kids have been using the diuretic for two years now.”
Families and doctors are eager for new treatments for autism. Just two drugs, risperidone and aripiprazole, are approved to treat the disorder, and they improve irritability and aggression rather than social and communication difficulties.
Still, several experts are puzzled because the study used diagnostic measures of autism in unusual ways. They also say that the sample size is too small to know whether the effects are real.
“Until a big study with a better design is done, we can’t think of this as a real treatment,” says Elizabeth Berry-Kravis, professor of pediatrics, neurological sciences and biochemistry at Rush University Medical Center in Chicago. Berry-Kravis has led several clinical trials of treatments for fragile X syndrome and was not involved in the new work. “We have to think of this as an exploratory first step.”
Read more at…