6 clinical trials now confirm the efficacy of Bumetanide to treat Autism Spectrum Disorders

With three new trials published this year, there are now 6 clinical trials performed by 5 different groups that support the efficacy of Bumetanide to treat Autism Spectrum Disorders (ASD) symptoms.
They are summarized here below: 

  1. Lemonnier, Ben-Ari and colleagues, 2012: monocentric double-blind randomized trial, 60 children (3-11 years old), assessed by CARS (Lemonnier et al., Transl Psych, 2012). 
  2. Du et al., 2015: monocentric trial, 60 children (mean age 4.5 years old), half received ABA behavioral treatment and half were also treated with Bumetanide. Children with ABA+Bumetanide had a significantly better improvement than ABA alone, assessed by ABC and CGI (Du et al., J of Child and Adolesc Psychopharma, 2015). 
  3. Lemonnier, Ben-Ari and colleagues, 2017: multicentric double-blind randomized trial, 88 children (2-18 years old), assessed by CARS, SRS and CGI (Lemonnier et al., Transl Psych, 2017).
  4. Zhang et al., 2020: monocentric randomized open-label, 83 children (3-6 years old), assessed by CARS and CGI with ASD that received 0.5mg twice daily had a significantly improved amelioration also evaluated with CARS and CGI versus untreated patients (Zhang et al., Transl Psych, 2020).
  5. Hajri et al., 2019: monocentric open-label trial, 29 children (mean age 7.9 years old), assessed by CARS (Hajri et al., La Tunisie Medical, 2019).
  6. Van Andel, Bruining and colleagues, 2020: monocentric trial, 15 patients with Tuberous Sclerosis (8-21 years old), assessed by ABC, autistic features were attenuated but not seizure frequency (Van Andel et al., Mol Autism, 2020).

In addition, 2 eye tracking and functional MRI trials confirm the Bumetanide-dependent amelioration of visual communication and brain imaging alterations that are in keeping with the suggested underlying mechanisms (Hadjikhani et al., Autism, 2015; Scientific Reports, 2018). Furthermore, even though Sprengers et al. (J Am Acad Child Adolesc Psychiatry, 2020) failed to find with SRS-2 an amelioration by Bumetanide over placebo (90 children, 7-15 years old) they did observe a significant effect with the Repetitive Behavior Scale. However, this failed trial is primarily ascribed to the very high placebo values, the children receiving supportive care and EEG recordings. The authors conclude that Bumetanide might be efficient in a subpopulation of children with ASD (Sprengers et al., J Am Acad Child Adolesc Psychiatry, 2020).     

Collectively, these trials are in keeping with numerous experimental studies showing a loss of GABAergic inhibition that is attenuated by Bumetanide. We hope that the large final phase 3 trial presently performed by the French Pharmaceutical company Servier in association with our startup company Neurochlore will be successful, leading to the first approved treatment of ASD.

Yehezkel Ben-Ari  
CEO Neurochlore, Président du fonds d’action IBEN, grand prix of the French Medical Research Council.

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