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One Drug Can Treat Three Neglected Diseases That Kill Thousands Yearly

The tsetse fly - Photo from Wikipedia

One drug can now treat three deadly, neglected tropical diseases, the BBC reports.

The drug, made by testing three million compounds and animal testing, has been called the “new hope” for fighting parasitic infections Chagas disease, leishmaniasis, and sleeping sickness. Similar parasites cause these three diseases, leading a team of scientists to posit that one treatment might be effective against them all.

Chagas disease, or America trypanosomiasis, comes from the Trypansosoma cruzi parasite in kissing or assassin bugs. Chagas causes an enlargement of the heart and digestive system, which can be fatal. Primarily found in Latin America, cases of this disease have been confirmed in other parts of the world.

Leishmaniasis is spread through the bite of sandflies carrying the Leishmania parasite. It produces a wide variety of symptoms based on where the infection started, including anemia and fever, leading to the destruction of the nose, mouth and throat linings. The disease is found in Asia, Africa, and the Americas.

Sleeping sickness is caused by the Trypanosoma brucei parasite, carried by tsetse flies. Officially known as Human African trypanosomiasis, the disease is nicknamed for its ability to cause a coma when the parasites penetrate the brain. The disease is found in sub-Saharan Africa.

These three diseases combined infect over 20 million people and kill more than 50,000 each year, scientists say.

There are drugs to treat these diseases individually, but they are expensive, toxic and often need to be administered using an intravenous drip, which is not practical in poor areas.

In a laboratory, researchers tested three million compounds created by Novartis, a drug company, to determine which ones could kill multiple parasites.

They were able to come up with one base compound which underwent thousands of modifications to make it twenty times more potent.

The new, upgraded compound – codenamed GF6702 – was tested on mice. It proved effective against Leishmania, Trypanosoma brucei and Trypansosoma cruzi in the mice.

Dr. Elmarie Myburgh, one of the researchers from the University of York, says this is the first time a treatment targeting all three parasites has been created, making it unique.

To me this is obviously a big deal, I’m in this field to try and make a difference, to get to a cure, and we’re working hard in the hope that it gets to patients.

The new drug works by attacking the proteasomes in the parasites, which recycles waste proteins. This does not affect similar chemical processes in mammals.

Richard Glynne of the Genomics Institute of the Novartis Research Foundation told the BBC that the attack on proteasomes was “not by design, more by serendipity.”

Before this discovery, the assumption was that proteasomes were too similar across all species to come up with a non-toxic drug.

Glynne says there may be a need to have three separate drugs, ultimately. “The biology of the diseases is different. For example in sleeping sickness the parasite is in the brain, so you need a drug that gets into the brain, so there are tweaks that may be required.”

Dr. Stephen Caddick of the Wellcome Trust research charity which helped fund the study, says that there is a continuous challenge to make affordable medicines for people suffering from these diseases, living in poverty-stricken regions, and getting the drugs to them. “This is quite an important piece of research, I’m excited by it, but there’s still a long way to go,” he says.

The scientists are working to refine the drug and are completing safety tests before starting on human trials.

The study was published in the journal Nature.

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