A new report contributes to the promising Bumetanide treatment for autism. Further studies should confirm this approach

The new publication by the team of Christopher Gillberg in Sweden extends previous positive observations and adds important information to the numerous previous trials on using Bumetanide to treat autistic children. Even though they performed an open-label trial of just six children (3-14 years old), it is important to note that all the participants had severe ASD (CARS score above 39) without comorbidities and that none of the parents wanted to interrupt the treatment after the 3-month trial period. Also, they used for the first time a parental scale to evaluate Bumetanide’s effect providing new insights on the parent’s perceptions of the benefits of the treatment. Their results show that Bumetanide treatment improved the CGI and parenting scores in the domains of interest in the world and attempts to communicate and clarity of communication. This report, although small, is in continuity with our groundbreaking positive results on the use of Bumetanide, a highly selective antagonist of the NKCC1 chloride importer, to treat autism first in a pilot open trial and then in two double-blind clinical trials. Currently, the large final phase 3 trial presently performed by the French Pharmaceutical company Servier in association with our startup company Neurochlore if successful will lead to the first approved treatment of ASD.    

Yehezkel Ben-Ari & Eric Lemonnier
Y B-A : CEO Neurochlore, Président de IBEN
E L : CHU Limoges

Références :

  • Fernell E. et al. Bumetanide for autism: open-label trial in six children. Acta Paediatrica 2020, https://doi.org/10.1111/apa.15723 
  • Ben-Ari Y, Lemonnier E. Using bumetanide to treat autism appears promising but futher clinical trials are needed to confirm this approach. Acta Paediatrica 2021;00:1-3. https://doi.org.10.1111/apa.15747