Scientific Publications-2009-HAT

2009

Nifurtimox-eflornithine combination therapy for second-stage African Trypanosoma brucei gambiense trypanosomiasis: a multicentre, randomised, phase III, non-inferiority trial by Priotto G, Kasparian S, Mutombo W, Ngouama D, Ghorashian S, Arnold U, Ghabri S, Baudin E, Buard V, Kazadi-Kyanza S, Ilunga M, Mutangala W, Pohlig G, Schmid C, Karunakara U, Torreele E, Kande V. In Lancet. 2009 July; 374:56-64.
This pivotal Phase III study assessed the efficacy and safety of nifurtimox-eflornithine combination therapy (NECT) for second-stage disease compared with the gold-standard regimen of eflornithine in 287 patients at four HAT treatments centres in the Republic of the Congo and the Democratic Republic of Congo. The results showed NECT to be comparable with eflornithine in efficacy (>90% cure in both treatment arms at 18-month follow-up) and to be well tolerated. Patient follow-up at 18 months after treatment was 93%, which is exceptionally high for these types of studies in HAT and further contributes to the robustness of the study. In comparison with eflornithine, NECT is easier to administer, more affordable and with simpler logistics, and potentially protective against the emergence of resistant parasites. The authors conclude that NECT represents an improved alternative for stage 2 HAT treatment and is suitable for first-line use in HAT control programmes.
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An accompanying Lancet editorial calls the NECT study ‘more than a small victory over sleeping sickness‘.
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Drug discovery for neglected diseases: View of a public-private partnership by Chatelain E, Don R. In Antiparasitic Antibacterial Drug Discovery by Paul M. Selzer (Ed); 2009 Apr: Wiley-Blackwell.
In answer to the lack of modern and effective drugs for diseases such as human African trypanosomiasis (HAT; sleeping sickness) and Chagas disease which present no financial viability for the pharmaceutical industry, new models of drug discovery have been developed.
The Drugs for Neglected Diseases initiative (DNDi) has adopted a model closely related to that of a virtual biotechnology company for identifying and optimizing drug leads.
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Drug Screening for Kinetoplastid Diseases: A Training Manual for Screening in Neglected Diseases by Ioset JR, Brun R, Wenzler T, Kaiser M, Yardley V. DNDi and Pan-Asian Screening Network 2009 Apr: 74pp.
The production of this research manual is one of the deliverables of the Pan-Asian Network for Drugs for Neglected Diseases from Natural Substances. This comprehensive manual is a practical and user-friendly guide for essays available to screen natural products against pathogens responsible for some of the neglected diseases. It brings recommendations and protocols related to these essays, describes the principles of good scientific practice, and lists information about materials requested for the essays, institutions using them as well as key references.
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