Sleeping sickness, or human African trypanosomiasis, is a deadly parasitic disease that affects millions of people in sub-Saharan Africa. It is transmitted by the bite of an infected tsetse fly and causes severe neurological symptoms, such as disrupted sleep patterns, confusion, aggression, and psychosis. Without treatment, the disease is fatal.
The current treatment options for sleeping sickness are limited and complex. They require multiple injections, hospitalization, and invasive procedures, such as lumbar punctures, to diagnose and treat the disease. These treatments are also expensive and difficult to transport and administer in remote and resource-poor settings, where most cases occur.
Moreover, the current treatments are not effective for all stages of the disease. Sleeping sickness has two stages: the first stage, when the parasite is in the blood and lymphatic system, and the second stage, when the parasite crosses the blood-brain barrier and invades the central nervous system. The second stage is more severe and requires different drugs than the first stage.
The World Health Organization (WHO) has set a goal to eliminate sleeping sickness as a public health problem by 2030. However, to achieve this goal, there is a need for simpler, safer, and more effective treatments that can be used for both stages of the disease and in any setting.
A Breakthrough Study on a Single-Dose Oral Treatment
A recent study, published in The Lancet Infectious Diseases, has reported promising results from a phase II/III clinical trial of a single-dose oral treatment for sleeping sickness. The treatment, called acoziborole, is a novel compound that was co-developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, a French pharmaceutical company.
The study involved 394 patients with sleeping sickness from the Democratic Republic of Congo (DRC), where more than 85% of reported cases occur. The patients were randomly assigned to receive either a single dose of acoziborole (three pills) or the standard treatment of intravenous eflornithine and oral nifurtimox (NECT) for 10 days.
The results showed that acoziborole was safe and well-tolerated, with no serious adverse events reported. The treatment was also highly effective, with success rates of 95% for patients with second-stage disease and 100% for patients with first-stage disease. These success rates were comparable to those of NECT, which was 97.6% for second-stage disease and 100% for first-stage disease.
The study also demonstrated that acoziborole could be used for both stages of the disease, as it was able to cross the blood-brain barrier and kill the parasite in the central nervous system. This means that acoziborole could simplify the diagnosis and treatment of sleeping sickness, as it would not require lumbar punctures or different drugs for different stages.
The Potential Impact of a Single-Dose Oral Treatment
The researchers and the developers of acoziborole have hailed the study as a major breakthrough for sleeping sickness treatment and elimination. They have said that acoziborole could be a game-changer, as it would offer a convenient, affordable, and accessible treatment option for patients and health workers in remote and endemic areas.
Dr Victor Kande, the lead author of the study and a former advisor at the Ministry of Health of DRC, said: “We are now on the cusp of a potential treatment that can be given in one day, in a single dose of three pills – this would be a revolution for doctors and communities.”
Dr Bernard Pécoul, the executive director of DNDi, said: “Acoziborole is potentially transformative, as it could remove many of the barriers that have prevented people from accessing treatment in the past. It could also contribute to the elimination of sleeping sickness, as it would make it easier to find and treat the last remaining cases of the disease.”
Dr Pauline Williams, the head of global health R&D at Sanofi, said: “Sanofi is proud to have partnered with DNDi to develop acoziborole, which represents a significant scientific innovation and a potential public health solution for sleeping sickness. We are committed to making this treatment available and affordable to the patients who need it most.”
The study authors have recommended that acoziborole should be approved by the relevant regulatory authorities and included in the WHO guidelines for sleeping sickness treatment. They have also called for further studies to evaluate the long-term safety and efficacy of acoziborole, as well as its impact on the transmission and epidemiology of the disease.
