Treating autism with a diuretic: a long procedure


Several readers have asked me about the current status of Bumetanide as a treatment for autism. The process in Europe is controlled by EMA (European Medicines Agency), the equivalent of the FDA in the United States.  

Bumetanide affects the function of the GABAA receptor.  If you click on the site index, you can refer to Bumetanide and read the research and my own son's very positive experience of using this drug since December 2012.     

Most readers in the UK and USA have difficulty getting their doctor to prescribe bumetanide, since autism is currently an off-label use.  Many readers elsewhere have been able to access this drug and are seeing its positive impact.

Dr Ben-Ari, whose research has been outlined in those earlier posts, has kindly provided this update:-




Treating autism with a diuretic: a long procedure 


We have started some time ago testing the possibility of using a diuretic to treat Autism Spectrum Disorders (ASD) relying on our promising experimental observations made in rodent. Indeed we discovered in 2 animal models of ASD (the in utero Valproate model and the Fragile X one) that cortical neurons have elevated intracellular chloride that shifts a major inhibitory mechanism to excitatory leading to perturbations of the behaviorally relevant brain oscillations (Tyzio et al Science 2014 and Eftekhari et al Science 2014). Correcting these elevated levels of chloride with a diuretic that reduces intracellular chloride ameliorated the electrical and behavioral signatures of ASD in these rodents. Relying on these and indirect observations, we had conducted with Dr Lemonnier a pilot trial followed by a phase 2 randomized double blind placebo control study on 54 children aged 3 to 11yrs old. We obtained promising results (Lemonnier et al Trans Psychiat 2012). Thus is followed now by a larger EMA approved trial with 80 children 2 to 18 yrs old that will be terminated next fall.

The procedure is long and complex as this requires many clinical controls and large sums of investments. This is however mandatory as  one cannot propose a treatment unless this has been tested and approved by the clinical authorities. Indeed, there have been many false hopes in the treatment of ASD, and one cannot give false promises without having all the elements needed that confirm that the treatment does improve the situation and has limited or no side effects.

We cannot therefore make any suggestion and promise as to the success or failure of the approach in spite of our compelling animal data and preliminary clinical observations. We follow the requirements and when and if our trials are successful , we shall pursue until we obtain an authorization to market this drug.

Sincerely


Information is available at
www.neurochlore.fr






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