The Drugs for Neglected Diseases initiative (DNDi), Anacor Pharmaceuticals and Scynexis Inc have now successfully completed pre-clinical studies for the first new oral drug candidate discovered specifically to combat Human African Trypanosomiasis (HAT), also known as sleeping sickness.

Sleeping sickness, which threatens millions of people in 36 countries in sub-Saharan Africa, is fatal if left untreated. The disease is caused by parasites transmitted by the bite of a tsetse fly and is often asymptomatic for years until the infection reaches “stage 2” where it crosses into the central nervous system and brain.

Without effective treatment, sleeping sickness is fatal. Currently available treatments are limited to drugs developed decades ago that are either highly toxic, difficult to administer in resource-limited settings, or are only effective in one stage of the disease. In addition, prior to being treated, the stage of the disease must be determined using a diagnostic spinal tap to extract cerebrospinal fluid from the patient.

Two-thirds of all reported sleeping sickness cases are found in the Democratic Republic of the Congo (DRC), where healthcare is often inaccessible to large parts of the population due to violent conflict, great distances patients must travel to health facilities and extreme poverty.

According to Dr Miaka Mia Bilenge, special advisor, National Control Programme for HAT in the DRC, if  the new drug candidate proves to be successful, it could become a major weapon to control sleeping sickness.

In pre-clinical studies, the new drug candidate, SCYX-7158, also registered as AN5568, demonstrated safety and the ability to cross the blood-brain barrier making it efficacious against stage 1 and stage 2 of the disease. In addition, its oral formulation, short duration of therapy and excellent pre-clinical safety profile imply that SCYX-7158 (AN5568) could change the way sleeping sickness is treated, reduce its incidence in humans, and contribute to elimination of the disease.

The successful development of this new compound was the result of a collaboration between Anacor Pharmaceuticals, a biopharmaceutical company in Palo Alto, California, Scynexis, a drug discovery and development company based in Research Triangle Park, North Carolina, and DNDi, a not-for-profit Research and Development (R&D) organization that develops new drugs to address the needs of the world's most neglected populations.

The  initial successful results of pre-clinical studies of the new compound will soon advance to phase I human clinical trials, reveals the open-access journal PLoS Neglected Tropical Diseases.

“People in the DRC and many countries in Africa have hoped for a safe, easy-to-use, and effective treatment against sleeping sickness for generations. We very much look forward to the start of human trials, and we hope that at long last this will give us a tool for sustainable elimination of this dreaded disease,” stated Dr Miaka Mia Bilenge.

“This innovative collaboration, expounding upon the expertise and engagement of two biotech companies and a not-for-profit R&D organization, has brought excellent results thus far, and this in an incredibly short time frame.  It is possible to stimulate patient needs-driven R&D for neglected tropical diseases, even if there is no commercial market,” said Dr Bernard Pécoul, executive director of DNDi.