Low-Cost “Water Pill” Could Ease Some Challenging Symptoms Of Autism, Study Finds

A pill that has been around for 40 years could be used to help with some symptoms of autism, a study has found.

The drug Bumetanide is a diuretic, or “water pill,” that stops the body from absorbing too much salt and helps reduce fluid build-up. Instead, it passes excess salt and fluid into the urine. The drug is typically used to treat fluid retention, or edema, in people with high blood pressure and cardiovascular, liver, or kidney disease. It was first approved for use in 1983.

A team of British and Chinese researchers studied the effect of Bumetanide on children with autism and found that it improved their brain function.

“I’m very excited about this. I think it could be a real breakthrough and very important for these families,” said co-author Professor Barbara Sahakian, a psychiatrist at the University of Cambridge.

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The water pill strengthens the signals between neurons in the brain. In particular, it helps correct an imbalance between GABA and glutamate neurotransmitters, which are important chemical messengers. An excess amount of GABA can affect brain development, as it controls how nerve cells communicate.

However, if doctors are able to correct the imbalance of GABA early in a child’s life, it could prevent some symptoms that can be more challenging for those with ASD as they grow older.

“We know GABA and glutamate are key chemicals in the brain for plasticity and learning and so these children should have an opportunity for better quality of life and wellbeing,” said Sahakian.

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The study was published in the journal Translational Psychiatry and led by researchers out of the University of Cambridge in the UK and multiple institutions in China.

The team recruited 83 children who were between the ages of three and six. They were divided into two groups: 42 children took a 0.5mg dose of bumetanide twice a day for three months, while 41 got no treatment at all.

After the three months were up, researchers assessed each group’s symptoms using the Childhood Autism Rating Scale (CARS). This rates behaviors like imitation, emotional response, and verbal and non-verbal communication. Those individual scores are then tallied up to determine their overall CARS score. Children with a CARS score above 30 are considered to have ASD.

Before the trial began, each child had similar CARS scores. After it ended, the group of treated children had a mean score of 34.51. The control group, who did not take the pills, had a mean score of 37.27. In addition, the group of treated children experienced a reduction in the number of individual items on CARS with a score of 3 or more. The treatment group had an average of 3.52 versus 5.49 for the control group.

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Researchers also used magnetic resonance spectroscopy (MRS) to look at the concentration of neurotransmitters in the brain. They found that, for the group of treated children, the ratio of GABA to glutamate decreased over the course of treatment in two key areas of the brain: the insular cortex, which affects emotions, empathy and self-awareness, and the visual cortex, which helps with integrating and processing visual information.

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

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Bumetanide is a cheap drug, costing mere pennies per pill. In addition, it’s a medication without any significant side effects.

“These findings are very promising and suggest we will be able to use the biomarker measure to identify which children with ASD will benefit most from bumetanide,” said Dr. Qiang Luo from Fudan University. “Further studies in a larger number of children will hopefully confirm whether bumetanide is an effective treatment for children with autism spectrum disorder.”

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