The inhibitory transmitter GABA has been suggested to play an important role in infantile autistic syndrome (IAS), and extensive investigations suggest that excitatory actions of GABA in neurological disorders are because of a persistent increase of [Cl−]I.
Aims: To test the effects of the chloride co-transporter NKCC1 diuretic compound Bumetanide that reduces [Cl−]I on IAS.
Methods: Bumetanide was administered daily (1 mg daily) during a 3 -month period and clinical and biological tests made. We used 5 standard IAS severity tests - Childhood Autism Rating Scale, Aberrant Behaviour Checklist, Clinical Global Impressions; Repetitive and Restrictive Behaviour and the Regulation Disorder Evaluation Grid.
Results: We report a significant improvement in IAS with no side effects.
Conclusion: Bumetanide decreases autistic behaviour with no side effects suggesting that diuretic agents may exert beneficial effects on IAS and that alterations of the actions of GABA may be efficient in IAS treatment calling for large scale randomized trials.