Diuretic Drug Could Help Treat Autism


Bumetanide, used for high blood pressure, showed promise in a small autism trial, reducing symptoms after three months of daily treatment.

The drug bumetanide, which has been used for decades to treat high blood pressure and other conditions, has now shown some promise in a small clinical trial for autism spectrum disorder. The drug reduced the overall severity of behavioral symptoms after 3 months of daily treatment.

The scientists published their findings in the journal Translational Psychiatry. Parents of the children who were treated reported that their children were more present and engaged in social interactions after taking the drug.

Autism interferes with the neurotransmitter GABA, which has the effect of dampening neural activity. Bumetanide could enhance the inhibitory effects of GABA and the drug has been used safely as a diuretic to treat a wide range of heart, lung, and kidney conditions. The scientists used a pool of 60 autistic children between the ages of 3 and 11, and randomly assigned them a daily pill of bumetanide or a placebo. The study was double-blind.

The children who got bumetanide improved by 5.6 points on a 60-point scale that’s used to assess behaviors related to autism spectrum disorder. That was enough to nudge the group average under the cutoff for severe autism and into the mild to medium category. The study didn’t look at whether the drug improves all symptoms equally, or some more than others.

Communication and social interactions were notably improved. This study and others suggest that drugs reducing the neural excitation by blocking glutamate or enhancing inhibition by boosting GABA may be helpful for treating autism.

Reference: “A randomised controlled trial of bumetanide in the treatment of autism in children” by E Lemonnier, C Degrez, M Phelep, R Tyzio, F Josse, M Grandgeorge, N Hadjikhani and Y Ben-Ari, 11 December 2012, Translational Psychiatry.
DOI: 10.1038/tp.2012.124

2 Comments on "Diuretic Drug Could Help Treat Autism"

  1. As with most pharmaceutical drugs, this has a long list of potential side effects. GcMAF, on the other hand, is a naturally occuring protein in a healthy immune system. Dr Bradstreet discovered that autistic children have a higher than normal level of nagalase, and this disrupts the body’s ability to make GcMAF. By reintroducing this protein, he found that 85% responded positively and some even went on to lose the label of autism as they no longer displayed autistic traits. See his speech on it (the paper to which he refers was peer-reviewed and published this week) https://www.youtube.com/watch?v=FZ5wog0oig0 . I fail to understand why FirstImmune GcMAF does not get more publicity. I’ve met parents whose children are now verbal after starting the therapy. Seeing the joy on their faces, this new discovery should be shouted from the rooftops. Its making me feel that the ‘conspiracy theorists’ have a point, that media is owned and managed by big money including big pharma.

  2. Interesting.
    Makes sense to use an existing approved drug off label, rather than going throuhg the IND/NDA process.
    The main issue here is what other systems are being affected, even if the therapeutic dose for ASD mitigation is lower than that for high blood pressure.

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