2023 Highlights
The quest to eliminate neglected diseases
From the first reported case to the very last
In 1827, a surgeon in what is today Bangladesh published a detailed account of a disease that was causing patients to come to hospital with grossly enlarged spleens, anaemia, and fever. This would become one of the first recorded outbreaks of visceral leishmaniasis, which came to be known as kala-azar, the Hindi for ‘black fever’.
Two hundred years later, in October 2023, medical history was made again in Bangladesh. The country became the first ever to receive official validation from the World Health Organization (WHO) for eliminating kala-azar as a public health problem.
This remarkable global health milestone was achieved thanks to a host of factors: successful control of sandflies that transmit the disease, vigilant disease surveillance, a network of assiduous community health workers, and as many experts were quick to point out: the decisive impact of introducing new, efficacious, and safe treatments for the deadly disease.
DNDi was proud to be part of this huge step forward for neglected diseases and to be recognized by national authorities for helping to catalyze progress for patients. Along with our partners in South Asia, we conducted a four-year clinical trial in Bangladesh and India that led to the adoption of these new treatments, specifically single-dose liposomal amphotericin B, which is safe, drastically shortens the length of treatment for kala-azar, and has cure rates of over 95%.
Aarti Devi, from Bihar, India, is one of over 1 million female Accredited Social Health Activists (ASHAs) working across the country to support maternal care, childhood immunization, nutrition, control of neglected tropical diseases, and other public health priorities. Acting as a critical bridge between the healthcare system and the community, ASHAs have played a central role in India’s strides towards eliminating visceral leishmaniasis as a public health problem.
India also introduced liposomal amphotericin B following the evidence generated by our trial, and it too now hopes to announce elimination of kala-azar in the coming years.
These victories serve as proof of the vital role of innovation – the development of new treatments, diagnostics, and preventative tools – in eliminating neglected diseases.
Perhaps nowhere is this link between innovation and elimination clearer than with the case of the most common form of African sleeping sickness. After decades of facing toxic, ineffective, and potentially fatal treatments, patients across endemic countries in sub-Saharan Africa now have access to fexinidazole, the first safe, simple, all-oral treatment for the disease developed by DNDi, Sanofi, and national control programmes in Africa.
Sleeping sickness cases are declining rapidly, even in the Democratic Republic of the Congo (DRC) – long the epicentre of this nightmare disease, with twice as many new cases over the past three decades as all other countries combined. Meanwhile, Guinea, which had the highest burden of the disease in West Africa and whose health system was devastated by the 2014 Ebola epidemic, is now hoping to soon receive WHO certification for eliminating sleeping sickness as a public health threat.
But elimination poses its own challenges.
As recent victories in disease elimination prove, investment in medical innovation to go that last mile can pay off, breaking vicious circles of illness and poverty for generations to come.
Complacency from global health donors or treatment programmes in endemic countries is a major threat. After all, history has shown that sleeping sickness and kala-azar both occur in cyclical waves – the minute the focus moves elsewhere, resurgence can and will occur. Climate change and human migration could also wreak havoc on the best-laid plans to control these diseases, particularly in areas prone to civil unrest.
Elimination requires sustained deployment of the package of tried-and-true public health tools that have underpinned progress in countries like Bangladesh, India, and the DRC: active disease surveillance, vector control, and prompt access to diagnosis and treatment.
Sustainable elimination also requires its own new dose of medical innovation. As cases get fewer and rarer, meeting the WHO’s 2030 targets for zero sleeping sickness and eliminating kala-azar as a public health problem will require therapeutic options that are not just safe and effective, but also specifically designed to take us across the elimination finishing line. Sustaining elimination might only be possible with drugs that can be taken in remote areas with poor health infrastructure: ideally oral treatments that combine high efficacy with an excellent safety profile, where treatment length is short, the threat of drug resistance is low, and the interactions with drugs used for other common diseases, such as malaria, are minimal.
This is why DNDi’s investments in R&D for sleeping sickness and kala-azar must continue.
In 2023, we continued work to complete the development of acoziborole, our novel sleeping sickness treatment to accelerate elimination, in partnership with Sanofi. Acoziborole is safe and well-tolerated, but importantly, it can be administered in a single dose – so it can be given to infected patients and also people with potential infection. Along with the Institute of Tropical Medicine Antwerp and other partners, DNDi is running the STROGHAT clinical trial in a remote region of Northwest DRC to demonstrate how acoziborole can be used to treat entire villages when suspected cases of sleeping sickness occur. If approved, acoziborole – along with new point-of-care tests for sleeping sickness and vector control efforts – could be the tool that national control programmes use to stamp out the last cases of the disease in the most hard-to-reach areas. For good.
Meanwhile, with South Asian countries looking to soon eliminate kala-azar, countries in Eastern Africa are also looking to jumpstart their elimination plans. While existing treatments for kala-azar in Eastern Africa are effective, entirely new medicines are needed. Current treatments require hospitalization, have potential toxicity, and are beginning to show resistance.
To address these limitations, we launched clinical trials in 2023 and 2024 in India and Ethiopia, respectively, for a promising new patient-friendly oral drug for leishmaniasis: LXE408. If the clinical trial is successful, the treatment could be taken at home and easily given to patients at the primary healthcare level, making it much easier to cure sick patients quickly and halt onward transmission – boosting prospects for the sustainable elimination of the disease.
As recent victories in disease elimination prove, investment in medical innovation to go that last mile can pay off, breaking vicious circles of illness and poverty for generations to come.
Photo credits: Lameck Ododo-DNDi, Matt Bouch-DNDi, Scholars-DNDi
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