Curing autism with the diuretic bumetanide – CURE AUTISM

We have recently discovered that the diuretic NKCC1 chloride co-transporter antagonist Bumetanide provides a successful treatment of Autism Spectrum Disorders (ASD). In a double blind randomized clinical trial, ASD symptoms were ameliorated significantly in 54 children (3-11 years) treated daily with (1mg) during 3 months with little or no adverse events (paper submitted). Over 100 patients are on open treatment –some for over 3 years. Pilot studies also show amelioration in emotional face recognition & eye contact in eye tracking experiments and activation in fMRI studies of brain regions involved in face recognition and emotional responses that are inactive before treatment. Therefore, Bumetanide constitutes a successful strategy to treat ASD with several advantages over the 2 FDA approved drugs (Spiridone and Apiriprazol) endowed with side effects.
To generalise the use of bumetanide in ASD, we shall develop two formulations of Bumetanide, perform a EU phase III trial and experimental studies to determine chloride regulation in ASD related disorders. The aims of the 3 groups of the task force are:
i) Neurochlore: Founded by the 3 leaders of this program, with an international patent, Neurochlore will develop 2 new formulations adapted for children and adolescents, an oral liquid form and a sustained release minitablets form. This program was presented to AFSSAPS. Contacts with EMA and FDA are planned soon. Neurochlore with an external CRO will implement a pivotal phase III study. Several experts and consultants in Autism, galenics and clinical development will assist Neurochlore.
ii) The imaging group in Lausanne and Brest will perform more imaging and eye tracking data to characterize the effect of treatment on activation of areas involved in emotion processing and social interactions.
iii) INMED team & Neurochlore in Marseille will measure intracellular chloride in animal models of ASD & related developmental disorders (fragile X, Rett and Prader Willi). If successful, parallel pilot clinical trials will be performed.
Financial aspects: After an international site visit of patients treated with the diuretic by an expert commission, The US Simon foundation has decided to invest 1.5M€ in Neurochlore to develop the treatment. The costs of the clinical trials, regulatory steps and operation of Neurochlore are estimated at 3.5- 4M over 3 years.