Thursday, November 9, 2017

A New Old Drug Against an Old Disease - African Trypanosomiasis 

There has been a truly exciting drug development against African trypanosomiasis. The previous arsenical drugs used were either very toxic to the patient and the more recent (2009) combination of nifurtimox-eflornithine (NECT) had to administered as a combination of pills and 14 intravenous infusions. The NECT was not toxic and could kill parasites even after they entered the brain. The down side was that this treatment could not easily be used in rural African villages. The same organization that developed NECT in 2007 rediscovered fexinidazole, a compound that Sanofi had developed as an general antiparasitic drug but had  not pursued it any further. Researchers supported by the Drugs for Neglected Diseases initiative found both in animal models and in humans that this drug  taken orally cured 99% of people in the early bloodstream stage of the disease and 91% of people in  the tertiary stage where the trypanosomes had entered the brain!  The drug was taken through clinical trials and should be approved by the European Medicines Agency for use  in infected humans in the Democratic Republic of the Congo by the end of 2018. This may prove to be the magic bullet against this devastating disease in tropical Africa. I myself hope that resistance will not be rapidly developed. The best way to avoid development of resistance would be to combine another effective drug with fexinidazole, which unfortunately remains to be discovered.

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