Bumetanide in Autism and Epilepsy – Drug Licensing Status

In Europe, drugs have to be licensed by the European Medicines Agency, or each country’s national drug’s agency.  In the US ,the regulatory body is the Food & Drug Administration (FDA).

Drug approval is a slow and costly process and results in different drugs being available in different countries, or the same drug being licensed for different age groups or illnesses in different countries.

Bumetanide has been licensed around the world for many years for use in adults.

 
Bumetanide

Bumetanide is a loop diuretic originally licensed more than 30 years ago to treat heart failure.  It has a secondary function; in the brain bumetanide blocks the NKCC1 cation-chloride co-transporter, and thus decreases internal chloride concentration in neurons. In turn, this concentration change affects the action of the neurotransmitter GABA, which has been shown to be useful for treatment of neonatal seizures, that quite often are not responsive to traditional GABA-targeted treatment. Bumetanide is therefore currently under evaluation as a prospective antiepileptic drug.

Bumetanide is also being investigated by a French researcher, Dr Ben-Ari, for use in treating childhood autism.

Bumetanide was developed with older patients in mind and was never licensed for children, let alone babies.  Experimentally, Bumetanide has been used in babies for many years.


Bumetanide in Neonatal Seizures – NEMO project

You may have read in earlier posts about the research done using bumetanide to treat neonatal seizures.  There is now a 15 strong consortium including Duke University in the US, Great Ormond Street Hospital in the UK and Dr Ben-Ari’s INMED in Marseille, who have joined forces to bring this therapy to the market. The initiative is called the NEMO project and they have a slick website explaining their work.

The clinical trials are being carried out at 7 sites across 5 European countries.  The phase 1 study has been completed.

The objective is to develop a bumetanide formulation for neonatal seizures and obtain a drug license called a Paediatric Use Marketing Authorization (PUMA), from the European Medicines Agency.  The drug can then be prescribed to babies within Europe.

 
Bumetanide in Autism

Dr Ben-Ari is soon to conduct a phase 2 clinical trial of the use of bumetanide for autism in multiple European centres.  This study follows on from the already completed initial studies in France.

Some parents, mainly in France, are already using bumetanide for their children with ASD.  Most others clearly want the drug to fully certified for use in childhood autism before trying it, or are unable to obtain it, since it is a prescription only medicine.

Dr Ben-Ari informed me that he expects his bumetanide formulation is likely to be licensed for prescription in Europe to autistic children within four years’ time.

The FDA has more onerous (costly) requirements than its European counterpart and so the drug will only be licensed in Europe as a therapy for childhood ASD.




 

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