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New research found that the prescription medication Bumetanide can improve symptoms associated with autism spectrum disorder. Getty Images
  • Bumetanide, a prescription medication, is being studied as a potential way to improve symptoms associated with autism spectrum disorder.
  • The drug was originally geared toward treating edema, a buildup of fluid in the body.
  • Researchers said that the study found that bumetanide improves brain function and reduces symptoms by reducing the amount of the brain chemical GABA.

Bumetanide, a prescription medication used to treat edema, may improve some symptoms in young children with autism spectrum disorder (ASD), according to early research.

A study published this week in Translational Psychiatry found that the drug modifies an amino acid in the brain called gamma aminobutyric acid (GABA) so that the ratio of GABA to glutamate in the brain is lower.

GABA and glutamate are vital for brain plasticity and learning capabilities.

Several institutions in China, along with a team from the University of Cambridge, participated in the study.

They enlisted 83 children from the ages of 3 to 6, and gave 42 of them 0.5 milligrams (mg) of bumetanide twice a day for 3 months. A control group of 41 children did not receive treatment.

The researchers say that bumetanide is safe to use in children as young as 3 years old. ASD can be diagnosed at 24 months and as early as 18 months old.

They evaluated the children’s symptoms using the Childhood Autism Rating Scale (CARS), which assesses behavior such as imitation, emotional response, and verbal and nonverbal communication. Children scoring above 30 on the scale are considered to have ASD.

Children had similar CARS scores before treatment. After, the group treated showed a significant reduction in CARS scores.

The researchers used brain imaging technology known as magnetic resonance spectroscopy to look at concentrations of neurotransmitters in the brain. The ratio of GABA to glutamate decreased over the 3-month span in children on bumetanide.

This occurred in the insular cortex and visual cortex. Those regions of the brain have roles in emotions and visual information processing.

“This is the first demonstration that bumetanide improves brain function and reduces symptoms by reducing the amount of the brain chemical GABA. Understanding this mechanism is a major step toward developing new and more effective drug treatments,” said Ching-Po Lin, a professor at National Yang-Ming University, in a statement.

“These children should have an opportunity for better quality of life and well-being,” noted Barbara Sahakian, a psychiatry professor at the University of Cambridge. “This study is important and exciting, because it means that there is a drug that can improve social learning and reduce ASD symptoms during the time when the brains of these children are still developing.”

The goal of the medication isn’t to reduce GABA levels, but to change GABA action, restoring its inhibitory effect by decreasing intracellular chloride, explained Dr. Nouchine Hadjikhani, a Harvard Medical School professor who has researched bumetanide in children with ASD.

Previous research has indicated ASD may be related to GABA, a neurotransmitter that controls how nerve cells communicate.

In adults, GABA turns nerve cells off. But in fetuses and young children, it switches nerve cells on and plays a role in nerve cell development.

Alterations in it may delay when neural circuits mature, which can impact network activity. Intervening at an early age could reduce some of the symptoms, researchers say.

Bumetanide was originally geared toward treating edema, a buildup of fluid in the body.

Previous research has shown it may also help conditions such as Rett syndrome, Down syndrome, some idiopathic autism, and Parkinson’s disease, noted Dr. Agnieszka Wroczyńska, a physician from Poland, in an email to Healthline.

Researchers point out more research needs to be done to verify the results.

A double-blind or randomized trial with more participants would be needed to confirm that the medication is an effective ASD treatment, the researchers say.

“This latest study was neither blinded nor placebo-controlled, making it impossible to know if any changes seen were due to the drug,” noted Dr. Jeremy Veenstra-Vanderweele, a psychiatrist at Columbia University.

Previous studies haven’t adequately controlled for decreases in potassium (that can occur while on the medication) or the need to supplement potassium in a substantial number of children.

“It is not yet clear if bumetanide offers any specific benefit in autism,” Veenstra-Vanderweele told Healthline. “I think that it is important to be sure that children are getting early behavioral interventions before considering something that has significant risks and unclear evidence of benefit.”

There’s already evidence that supports early behavioral and developmental interventions which don’t carry any accompanying medical risk.

“These should be sought and implemented prior to considering medications with unclear evidence of benefit,” he added.

Parents interested in exploring the medication should seek out doctors who have experience using it with autistic patients, said Dr. Richard E. Frye, a neurologist with Phoenix Children’s Hospital.

He prescribes the drug to some patients with ASD. He believes more doctors will prescribe the “very safe drug” as more evidence is published.

“I have seen it result in significant improvements in some patients,” he said, noting that it may only help a subset of them.

“I use it in patients that are non-responders to standard behavioral and medical therapies,” Frye added.

The medication may not be right for all children, Hadjikhani said.

“Of course, it will not work for every autistic individual, so in that sense it could be a false hope, but I think that for those who respond to that drug, changes are really quite impressive,” she said.

“Bumetanide does not offer an ‘autism cure,’ but it can significantly increase the quality of life of some autistic persons thanks [to] its potential to bring about improvements in sensory processing and hypersensitivity, cognition, and acquiring communication skills,” Wroczyńska added.

The medication has been effective in some children, but it’s not clear how to best predict a child’s response.

“It has been shown that the outcome in cases of severe autism is extremely poor,” she added.

Hadjikhani warned parents to make sure their child’s diagnosis is accurate before considering the drug.

The medication can affect electrolyte levels, so it will likely require pretreatment exams including bloodwork and EKG readings. Children should be supervised closely in the first weeks after beginning the medication, she added.

You need to make sure that the diagnosis is right, and that there’s no medical reason to not start the treatment, Hadjikhani said.

“It will only have positive effects on some, and no effect on others. So, taken with all the safety issues that need to be considered, it is in my opinion worth giving it a try, and if nothing happens after a few months, then just discontinue it,” she said.