A “potentially transformative” drug to treat sleeping sickness could set the world on course to eradicate the fatal disease by 2030, according to a landmark study.
Researchers have found that giving a single dose of a drug called acoziborole is 95 per cent effective in patients with severe sleeping sickness, and 100 per cent effective in those diagnosed early.
The results have been described as a major breakthrough, as previous treatments have been complicated and costly – involving long hospital stays, invasive procedures and the transportation of specialist medication to remote places.
Sleeping sickness, also known as human African trypanosomiasis, is a parasitic pathogen spread by tsetse flies. Fatal without treatment, it can cause debilitating disruption of sleep patterns, uncontrollable aggression and psychosis.
“Sleeping sickness is a nightmare disease that affects patients in some of the most remote settings in west and central Africa, where distance from hospital can be measured in days,” said Dr Victor Kande, a former advisor at the Ministry of Health of the Democratic Republic of Congo (DRC) – where most cases are found – and lead author of the paper, published in the Lancet.
“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.”
Historically, treatment for sleeping sickness involved a daily injection for at least seven days, plus a drip in hospital for those in the later stages of the disease. But around 85 per cent of reported cases are in DRC, often in impoverished, rural and sometimes dangerous regions where it was difficult to provide the required care.
Then came a breakthrough. In 2019, a drug called fexinidazole – a tablet that could be taken once a day for 10 days to treat the most common form of the disease – was rolled out in the DRC.
But acoziborole, which was co-developed by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, a French pharmaceutical, marks an even bigger step forward – and researchers say it could help to eliminate the spread of the disease by 2030.
“Acoziborole has the potential to have a major impact on sleeping sickness treatment,” said Dr Wilfried Mutombo Kalonji, a project leader at DNDi and co-author of the paper.
“We now have the promise of single-dose, oral treatment that doesn’t need to be taken with food, removes the need for hospitalisation, removes the need for complex or invasive diagnosis or disease staging, and requires minimal training. In many ways, it doesn’t get any simpler than this.”
During the 18-month trial, acoziborole was given to 208 patients at 10 hospitals across the DRC and Guinea. There were no safety red flags, with Sanofi set to submit the data to the European Medicines Agency in the hope of rolling it out more broadly.
The regulator has been involved in designing the trial and approved the decision to have no control arm due to the challenges of enrolling patients.
This is partly because the toll has dropped sharply in the last 20 years. In 1998, at least 40,000 cases were reported each year – in 2020, that figure stood at less than 1,000. But the disease has a history of resurgence, necessitating the development of effective treatments.
Experts now hope that acoziborole could allow other countries to follow the likes of Benin, Uganda and Rwanda, which wiped out at least one form of sleeping sickness earlier this year.
“With these new data, we have hope that we may be able to finally eliminate the disease, once and for all,” said Dr Antoine Tarral, head of the sleeping sickness programme at DNDi and co-author of the paper.
