Biotech, NGO Partnership Advances Sleeping Sickness Drug

Tsetse fly (Agricultural Research Service, USDA)

A consortium of two biotechnology companies and non-government organization have completed preclinical studies of a potential treatment for sleeping sickness. The partners — Anacor Pharmaceuticals in Palo Alto, California, SCYNEXIS Inc. in Research Triangle Park, North Carolina, and Drugs for Neglected Diseases initiative (DNDi) in Geneva, Switzerland — published their findings in the journal PLoS Neglected Tropical Diseases.

Sleeping sickness or human African trypanosomiasis (HAT) is a parasitic disease transmitted to humans by tsetse fly bites that acquired their infection from human beings or from animals harboring the human pathogenic parasites.

The disease, according to the WHO, threatens millions of people in 36 countries in sub-Saharan Africa. Many of the affected populations live in remote areas with limited access to adequate health services. In the last 10 years, over 70 percent of reported cases occurred in the Democratic Republic of Congo.

The drug candidate SCYX-7158 (registered as AN5568) made by Anacor, was the identified in a screening of Anacor’s potential boron compounds at the Sandler Center for Drug Discovery of the University of California, San Francisco. With DNDi and SCYNEXIS, the team discovered and optimized a type of boron-containing small molecules, called benzoxaboroles, to deliver SCYX-7158 as an oral preclinical drug candidate.

The paper reports on lab tests of SCYX-7158 with mice that showed the compound cured mice infected with the HAT pathogen, both in the blood and in the brain. These results suggest that the compound can be effective against stage 1 and stage 2 of the disease, thus making it a candidate for human clinical trials.

The research was supported by grants from the Bill & Melinda Gates Foundation, government ministries in France and Spain, and the organization Doctors Without Borders/Médecins Sans Frontières.

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