Abstract
Human African Trypanosomiasis (HAT or sleeping sickness) is a life-threatening neglected tropical disease that is endemic in 36 sub-Saharan African countries. Until recently, treatment options were limited and hampered by unsatisfactory efficacy, toxicity, and long and cumbersome administration regimens, compounded by infrastructure inadequacies in the remote rural regions worst affected by the disease. Increased funding and awareness of HAT over the past two decades has led to a steady decline in reported cases (<1000 in 2018). Recent drug development strategies have resulted in development of the first all-oral treatment for HAT, fexinidazole. Fexinidazole received European Medicines Agency positive scientific opinion in 2018 and is now incorporated into the WHO interim guidelines as one of the first-line treatments for HAT, allowing lumbar puncture to become non-systematic. Here, we highlight the role of global collaborations in the effort to control HAT and develop new treatments. The long-standing collaboration between the WHO, Sanofi and the Drugs for Neglected Diseases initiative (Geneva, Switzerland) was instrumental for achieving the control and treatment development goals in HAT, whilst at the same time ensuring that efforts were led by national authorities and control programs to leave a legacy of highly trained healthcare workers and improved research and health infrastructure.
Highlights
Human African Trypanosomiasis (HAT), or sleeping sickness, is a life-threatening, neglected tropical disease (NTD)
T. b. gambiense is currently responsible for 98% of HAT cases [5], with the highest disease burden in the Democratic Republic of Congo (DRC) where 37.5 million people were estimated to be at some level of risk of g-HAT between 2012 and 2016 [1,2]
Humans are the principal reservoir for g-HAT and the progressive disease course occurs in two stages: a hemolymphatic phase with common signs and symptoms including fever, headache, pruritus, weakness, asthenia, anemia, and lymphadenopathy; and a meningo-encephalic stage occurring when the parasites have crossed the blood–brain barrier with resulting sleep disturbances and neuropsychiatric symptoms that may lead to coma and death if left untreated [4,5,6]
Summary
Human African Trypanosomiasis (HAT), or sleeping sickness, is a life-threatening, neglected tropical disease (NTD). It is considered endemic in 36 sub-Saharan African countries, where around 60 million people are estimated to be at some level of risk of HAT, primarily those living in ‘foci’ in poor and remote rural areas where health infrastructure is poor or non-existent [1,2,3]. We emphasize the role played by the public–private partnership between Sanofi (formerly Aventis) and the Drugs for Neglected Diseases initiative (DNDi, Geneva, Switzerland), together with a range of governmental national sleeping sickness control programs (NSSCPs; such as the Le Programme National de Lutte contre la Trypanosomiase Humaine Africaine of the DRC [PNLTHA]) and non-governmental organizations (NGOs), in development of the first oral-only treatment for HAT, Fexinidazole Winthrop (fexinidazole)
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