Oedema drug improves autism symptoms

A drug prescribed to treat oedema improves some of the symptoms in young children with autism, according to a study published today.

Researchers in China and the UK have found for the first time that bumetanide decreases the ratio of the GABA to glutamate in the brain in humans, it was announced.

Although previous studies in rats and small clinical trials involving children with ASD have suggested that bumetanide could help reduce symptoms of ASD, this research at a number of institutions across China and at the University of Cambridge, UK, has shown that the drug is safe to use and effective at reducing symptoms in children as young as three years old.

This research involved 83 children, aged three to six years, who were split into two groups: a cohort of 42 in a treatment group of received 0.5mg of bumetanide twice a day for three months, while the control group of 41 children received no treatment.

The research team used magnetic resonance spectroscopy to study concentrations of neurotransmitters within the brain and found that in two key brain regions – the insular cortex and visual cortex – the ratio of neurotransmitter GABA to glutamate, which are important for brain plasticity and promoting learning, decreased over the three-month period in the treatment group.

The findings, published in Translational Psychiatry, also showed that while both groups had similar scores on the Childhood Autism Rating Scale (CARS) before treatment, afterwards, the treatment group had a mean total score of 34.51, compared to the control group mean score of 37.27.

The treatment group also showed a significant reduction in the number of items on the CARS assigned a score greater than or equal to three, with the average number of 3.52 items in the treatment group compared to 5.49 items in the control group.

Professor Ching-Po Lin of National Yang-Ming University said: "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 towards developing new and more effective drug treatments.”

Professor Barbara Sahakian from the Department of Psychiatry at the University of Cambridge added: "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. We know that 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."

Lingli Zhang et al. Symptom improvement in children with autism spectrum disorder following bumetanide administration is associated with decreased GABA/glutamate ratios. Translational Psychiatry 27 Jan 2020

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