New Single-Dose Drug Offers Hope to Eliminate Sleeping Sickness by 2030

A modern single-dose medication, acoziborole, offers a potentially transformative step toward eliminating sleeping sickness – also known as human African trypanosomiasis (HAT) – by 2030, according to a recent report. The European Medicines Agency has given the drug a positive recommendation, paving the way for its employ as early as next year. This development addresses a long-standing challenge in treating the parasitic disease, which is spread by the bite of the tsetse fly.

Sleeping sickness, famously depicted in Joseph Conrad’s Heart of Darkness, can progress rapidly from mild symptoms to death if left untreated. The disease is endemic to sub-Saharan Africa, and historically has been categorized into two forms: West African sleeping sickness, caused by Trypanosoma brucei gambiense, and East African sleeping sickness, caused by Trypanosoma brucei rhodesiense. The new drug specifically targets the more prevalent West African form, accounting for over 90% of cases.

A Simpler Treatment for a Complex Disease

Current treatments for sleeping sickness are often complex and carry significant side effects. Older therapies involved intravenous drugs that could cause a severe burning sensation and, in nearly one in 20 patients, proved fatal. Even the current first-line oral treatment, fexinidazole, requires a 10-day course and can cause nausea, vomiting, and heart-rhythm disturbances. Acoziborole, by contrast, is administered as a single dose of three pills, dramatically simplifying treatment and reducing the burden on both patients and healthcare systems.

“For decades, available treatments were demanding to use,” explains Dr. Gerardo Priotto, who leads the World Health Organization’s efforts against sleeping sickness. “Therapies required staff, equipment and reliable infrastructure… These challenges were especially severe in remote, rural areas, where most cases occur and health services are limited.” The ease of administration of acoziborole is expected to significantly improve access to treatment in these underserved regions.

The tsetse fly transmits the parasite through its bite, thriving in the savanna woodlands and vegetation near water sources common in areas where people rely on fishing, hunting, and agriculture. This geographic and socioeconomic context contributes to the disease being considered a “disease of the poor,” as noted by Dr. Peter Hotez, dean of the National School of Tropical Medicine at the Baylor College of Medicine.

Clinical Trial Results and Side Effects

Clinical trials of acoziborole have shown promising results, with the most significant side effect reported being mild to moderate headache. This is a substantial improvement over existing treatments. Dr. Stéphane Hugonnet, who worked on the clinical trials for the Drugs for Neglected Diseases Initiative (DNDi), notes that previous treatments often discouraged patients from seeking care due to their harsh side effects. Dr. Hugonnet emphasizes that the new drug’s improved tolerability could encourage more people to come forward for treatment.

The development of acoziborole is a collaborative effort between DNDi, pharmaceutical firm Sanofi, and funding partners including the Gates Foundation, which also provides financial support for NPR’s global health coverage. This partnership highlights the importance of collaborative approaches to tackling neglected tropical diseases.

The Two Stages of Sleeping Sickness

Sleeping sickness progresses in two stages. The early stage presents with flu-like symptoms such as fever and headaches. As the parasite crosses the blood-brain barrier, the disease enters a later, more severe phase characterized by neurological symptoms like confusion and convulsions. This stage also disrupts the body’s natural sleep-wake cycle, leading to daytime sleepiness and nighttime wakefulness – hence the name “sleeping sickness.” Without treatment, the disease can lead to coma and death.

Acoziborole is effective against both stages of the disease, offering a significant advantage over some previous treatments that were only effective in one stage. Dr. Wilfried Mutombo Kalonji, head of West and Central Africa Clinical Operations for DNDi, expressed optimism about the drug’s potential, stating, “We can’t dream to have better than this.”

Streamlining Diagnosis and Treatment

Researchers are now exploring ways to further streamline the diagnosis and treatment process. A new trial is underway to investigate whether blood tests could allow for immediate treatment initiation, rather than waiting for confirmatory tests, which are more expensive and time-consuming, and often unavailable in remote areas. Monica Mungier, who studies sleeping sickness at the Johns Hopkins Bloomberg School of Public Health, cautions that the parasite can sometimes evade detection, meaning a negative blood test doesn’t always guarantee the absence of infection. However, if reliable blood tests prove sufficient, it could dramatically accelerate treatment access.

The Centers for Disease Control and Prevention (CDC) provides detailed information about sleeping sickness, including its causes, symptoms, and prevention. The WHO (WHO) is actively working towards eliminating the disease by 2030, and the approval of acoziborole represents a major step towards achieving this goal.

Looking Ahead: Funding and Continued Vigilance

The next steps involve review and authorization by the Democratic Republic of Congo Ministry of Health and the WHO, which will inform treatment guidelines globally. However, global health experts express concern that potential funding cuts from the U.S. And other Western nations could hinder access to the drug in countries that need it most.

The success of efforts to eliminate sleeping sickness relies heavily on the dedication of African researchers and patients who participated in the clinical trials, often under challenging conditions. The collaborative spirit and resilience demonstrated throughout the process offer hope for a future free from this debilitating disease.