A new study in The Lancet Infectious Diseases finds that a single oral dose of the drug acoziborole is 95% effective 18 months after treatment for sleeping sickness in adults and adolescents, regardless of disease stage.
Human African trypanosomiasis (HAT), more commonly known as sleeping sickness, is a neglected tropical disease most often seen in west and central Africa, especially the Democratic Republic of the Congo (DRC). The disease is caused by the parasite trypanosomes.
Though treatments exist, they are cumbersome: either a daily injection for at least 7 days during the early stages of the disease or an intravenous drip administered while hospitalized if the disease is caught in later stages. In 2019, a 10-day oral medication was introduced, but it requires extensive medical supervision.
In the current study, acoziborole, a drug co-developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, was given just once at a dose of 950 milligrams to 208 participants, including 167 people with late-stage disease and 41 with early- or intermediate-stage illness. Participants were followed for 18 months.
After 18 months, 95% (159 of 167) of patients with late-stage sleeping sickness treated with acoziborole were cured (no trypanosomes were present in body fluids), and all participants with early to intermediate illness were cured.
The authors acknowledged there was no control arm in the study. However, the positive results could lead the drug to help halt HAT by 2030.
In a commentary, Jacques Pepin, PhD, of the University of Sherbrooke, Canada, wrote, "Acoziborole represents an extraordinary advancement in the treatment of this neglected disease and might be key to the interruption of HAT transmission."