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New Drug Can Kill Parasites That Cause Three Neglected Tropical Diseases Affecting Millions Worldwide

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The so-called neglected tropical diseases, which are mostly caused by parasites, kill thousands of people per year, but there are few available treatments because research for and development of such drugs receive little funding.

Findings in new research, however, may eventually pave the way for an effective treatment that can cure these diseases.

Researchers have identified a new drug capable of treating these parasitic infections. In a letter published in the journal Nature on Monday, Aug. 8, researchers reported a new class of drug that can treat chagas disease, leishmaniasis and sleeping sickness.

Chagas disease is contracted through the kissing bug that bites people on the face. Leishmaniasis is transmitted by sand flies and is characterized by skin sores, while sleeping sickness, also known as trypanosomiasis, is spread by the tsetse fly and could potentially lead to serious neurological conditions.

These debilitating diseases, which affect 20 million around the globe and kill no fewer than 50,000 people per year, are all caused by parasites known as kinteoplastids. Although these diseases have different symptoms, the parasites that cause them have similar biology and genomic sequence, which suggests that these three diseases could be treated using a single class of drugs.

"We found that these parasites harbor a common weakness. We hope to exploit this weakness to discover and develop a single class of drugs for all three diseases," said study researcher Frantisek Supek from the Genomics Institute of the Novartis Research Foundation.

The researchers tested three million compounds on parasites, and eventually identified a compound called GNF6702 that worked against all the parasites. The chemical binds to its target and prevents it from functioning. The researchers then refined it to become more potent before they proceeded to conduct tests on mice.

"It's a breakthrough in our understanding of the parasites that cause the three diseases, potentially allowing them to be cured," said Jeremy Mottram from the University of York.

"This early phase drug discovery project will now move towards toxicity testing prior to human trials."

The compound cleared parasites from the animals and did not appear to have adverse effects in mice, suggesting it may have fewer side effects compared with existing drugs. The drug, however, still needs to be tested in human studies.

"Our data provide[s] genetic and chemical validation of the parasite proteasome as a promising therapeutic target for treatment of kinetoplastid infections, and underscore the possibility of developing a single class of drugs for these neglected diseases," the researchers wrote in their study.

Photo: Michael Wunderli | Flickr 

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