Bumetanide Fails Phase 3 Trials

September 16, 2021

Hopes Fade as Promising Drug to Treat Autism Shows No Sign of Effectiveness

Bumetanide is a generically-available diuretic drug used to treat high blood pressure.  For years, the medication was thought to have potential for treating autism due to its effect on the GABA neurotransmitter pathway, which is speculated to be disrupted in autism. Last week, Servier and Neurochlore, the French companies sponsoring bumetanide’s trials, had to abandon their quest to bring the first medication to market to treat autism, after the drug showed a comprehensive failure in two phase 3 trials. The phase 3 studies involved two cohorts of different aged children with autism. The younger children were aged 2 to 6 and the older subjects were aged 7 to 17. At the end of the trials, both cohorts failed to show any improvement on the drug as measured by the childhood autism rating scale (CARS) as well as on the social responsiveness scale (SRS). The failure is a crushing blow to the autism community, as the drug showed promise in mouse model studies last year.  However, all may not be lost for the drug. Another recent study has shown that bumetanide may be beneficial for a subset of children with autism who suffer panic disorders linked to the parietal and central parietal regions of their brain as indicated by EEG. This subgroup showed a decrease in repetitive behaviors and exhibited more sociability after taking the drug. 

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