The Drugs for Neglected Diseases initiative (DNDi) has received new grant support totaling USD 20 million to accelerate the development of innovative new drugs for patients in sub-Saharan Africa with sleeping sickness (human African trypanosomiasis – USD 12 million) and river blindness (onchocerciasis – USD 8.2 million) from the Bill & Melinda Gates Foundation.
These grants will run until the end of 2027 and will help deliver a single-dose cure for sleeping sickness, a terrifying and fatal disease, and greatly aid efforts to develop a short-course new treatment for the millions of people at risk of river blindness in Central and West Africa.
’While enormous progress has been made in controlling sleeping sickness and river blindness in Africa, sustainable elimination of these diseases will only be possible with the development of easy-to-use oral medications,’ said Luis Pizarro, Executive Director of DNDi. ‘Going the last mile towards elimination will require continued investment in new health tools – we cannot stop innovating for the millions of people at risk of these diseases.’
The financial support will help develop:
1- The first single-dose oral treatment for sleeping sickness, to support sustainable elimination of the disease:
- Sleeping sickness is transmitted by the tsetse fly. The parasite attacks the central nervous system, causing severe neurological disorders, convulsions, and coma. The disease is nearly always fatal without treatment. While cases are currently declining, 3 million people live in areas at moderate to very high risk in sub-Saharan Africa, particularly in the Democratic Republic of Congo (DRC), which is home to more than 60% of all cases.
- In 2018-9, DNDi and partners, in particular Sanofi, delivered fexinidazole, the first all-oral treatment for Trypanosoma brucei gambiense sleeping sickness (the most common form of the disease, found in West and Central Africa). Fexinidazole is now implemented and in use in endemic countries and eliminates the need for systematic hospitalization of patients, improves the management of sleeping sickness patients, and facilitates the integration of sleeping sickness treatment into health systems.
- A second all-oral, and importantly single-dose treatment, acoziborole, has been tested in Phase II/III clinical studies in DRC and Guinea to assess its safety and efficacy for Trypanosoma brucei gambiense sleeping sickness. The results, published in November 2022 in The Lancet Infectious Diseases, show treatment success rates of up to 95%.
- Once implemented, this breakthrough treatment would bring a simple, safe, and effective treatment that – together with a rapid diagnostic test – could be administered at point of diagnosis. This important feature would be a game-changer for sustainable elimination of the disease.
- DNDi, with financial support from the Gates Foundation, will pursue the final steps of acoziborole development, with studies in adults seropositive for Trypanosoma brucei gambiense sleeping sickness, children, and breastfeeding women, as well as support access (manufacturing, registration, and introduction) to the treatment in endemic countries.
2- A new drug to address debilitating filarial disease. This new medicine will enable test-and-treat strategies and shorten the time to onchocerciasis elimination:
- River blindness is transmitted by the bite of an infected blackfly and causes severe itching, skin lesions, and eventually blindness. An estimated 19 million people are infected by the disease worldwide. The current approach to eliminating river blindness is based on the mass distribution of ivermectin, which has been tremendously successful in reducing disease prevalence. But these programs need to be repeated annually for 10-12 years as current treatments only kill the juvenile worms that migrate into the eyes and cause river blindness, but not the adult worms, which can live more than 10 years in the human body.
- DNDi aims to develop a safe, effective, and field-adapted ‘macrofilaricidal’ drug that can kill adult worms. This could be used for individual patient treatment with the potential for use as preventive chemotherapy to accelerate the elimination of river blindness in Africa in areas that are difficult to treat.
- DNDi and partners have already successfully advanced a compound known as emodepside through first-in-human Phase I to proof-of-concept Phase II development thanks to previous support from the Gates Foundation. This renewed funding from the foundation will allow DNDi to further progress emodepside in a second stage of the Phase II clinical trial in DRC.
Both these investments build on previous financial support to DNDi’s earlier-stage R&D activities in filarial diseases and sleeping sickness projects from the Gates Foundation.
‘We would like to thank the Bill & Melinda Gates Foundation for supporting the activities and mission of DNDi for the past 17 years’ said Luis Pizarro. ‘With support from the foundation, we will continue to pursue our vision that all neglected patients have the right to the best medical innovation and science that exists.’
The Drugs for Neglected Diseases initiative (DNDi) is a not-for-profit medical research organization that discovers, develops, and delivers safe, effective, and affordable treatments for neglected people. DNDi is developing medicines for sleeping sickness, leishmaniasis, Chagas disease, river blindness, mycetoma, dengue, paediatric HIV, advanced HIV disease, cryptococcal meningitis, and hepatitis C. Its research priorities include children’s health, gender equity and gender-responsive R&D, and diseases impacted by climate change. Since its creation in 2003, DNDi has joined with public and private partners across the globe to deliver 13 new treatments, saving millions of lives.
Ilan Moss, Head, Media and Content, DNDi North America
Frédéric Ojardias, Media Relations Manager, DNDi in Geneva
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Photo credit: Lameck Ododo-DNDi