New Drug Offers Hope for Eliminating Sleeping Sickness by 2030

A new single-dose drug, acoziborole, is poised to dramatically simplify the treatment of sleeping sickness, too known as human African trypanosomiasis (HAT), and could help the World Health Organization (WHO) achieve its goal of eliminating the disease by 2030. The drug received a positive recommendation from a committee of the European Medicines Agency and could be available for use by early next year.

Understanding Sleeping Sickness

Sleeping sickness is a parasitic disease transmitted by the bite of the tsetse fly, found in sub-Saharan Africa. The Centers for Disease Control and Prevention (CDC) notes that without treatment, one form of the illness can progress from mild symptoms to death within weeks. The disease is historically linked to Joseph Conrad’s novel, Heart of Darkness.

Acoziborole: A Breakthrough in Treatment

Current treatments for sleeping sickness can be challenging to administer, particularly in remote areas with limited healthcare infrastructure. Previous treatments included intravenous drugs with potentially fatal side effects for nearly one in 20 patients. Even the current first-line oral treatment, fexinidazole, requires a 10-day course and can cause severe side effects like nausea, vomiting, and heart-rhythm disturbances.

Acoziborole offers a significant improvement. It is administered as a single dose of three pills, making it easier to use and more accessible. According to NPR, clinical trials have shown the most significant side effect to be mild to moderate headache.

Impact on Public Health Efforts

Dr. Gerardo Priotto, who leads the WHO’s efforts against sleeping sickness, emphasized the transformative potential of acoziborole. “Its single-dose, well-tolerated regimen can dramatically simplify patient care, improve access to treatment and accelerate progress toward the elimination of sleeping sickness,” he stated. The drug removes many of the barriers associated with previous therapies, which required specialized staff, equipment, and reliable infrastructure.

The Drugs for Neglected Diseases Initiative (DNDi), a nonprofit organization, developed acoziborole in collaboration with pharmaceutical firm Sanofi and with funding from organizations like the Gates Foundation. Dr. Stéphane Hugonnet, who worked on the clinical trials, noted that the harsh side effects of older treatments often discouraged people from seeking care.

Addressing Challenges and Future Steps

Sleeping sickness disproportionately affects impoverished communities in remote areas where tsetse flies thrive. The disease progresses in two stages: an early stage with fever and headaches, and a later stage with more severe neurological effects, including disrupted sleep patterns. Acoziborole is effective against both stages of the disease.

Researchers are now exploring whether blood tests can be used to initiate treatment immediately, rather than waiting for confirmatory tests, which are more time-consuming and expensive. The next steps involve review by the Democratic Republic of Congo Ministry of Health and the WHO to update treatment guidelines and authorize the drug’s use globally. However, concerns remain about potential funding cuts that could hinder access to the drug in countries that need it most.