Bumetanide to treat brain disorders: when the empire strikes back!

Our attempts to treat children with Autism Spectrum Disorders (ASD) with the NKCC1 chloride importer antagonist have met with some success with several hundreds of children treated successfully in several trial made in France, China, Sweden, Great Britain, Holland and Tunisia. Yet a large phase 3 (400 children in Europe, Brazil, Australia and US) failed. However, a recent Dutch study shows that bumetanide is efficient in children /adolescents having particular EEG features. These are “corrected“ by Bumetanide in parallel with the clinical amelioration, and in fact EEG enables to predict whether the patient will respond or not to the treatment. In this reply paper; with Enrico Cherubini, we reply to some of the criticisms made by a Finish group who has repeatedly advocated to let bumetanide down that ought not to be used in clinical trials. We explain that firstly, the reliance on rodents measures are not always relevant to the sick human brain and secondly that even if the site and mechanism of actions will reveal wrong, the fact that it does treat at least a subpopulation of children who have no other treatments calls for more prudence and careful conclusions. 

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