Develop a new treatment for sleeping sickness to replace the complex and toxic melarsoprol arsenic-based drug that kills 5% of those treated
current phase of drug development
updated 29 Feb 2020
Better, simpler, shorter treatment for sleeping sickness patients and health staff
The first improved treatment for sleeping sickness
Before 2009, the best treatment for sleeping sickness, eflornithine, was extremely complex to distribute and administer in regions affected by the disease. All-too-often, doctors would have no choice but to use melarsoprol, a highly toxic, arsenic-based drug that killed 1 in 20 patients.
In 2009, results from DNDi clinical trials showed that a simpler and shorter nifurtimox-eflornithine combination therapy (NECT) was safe and effective to treat sleeping sickness. NECT has significant practical benefits in comparison to eflornithine: there are fewer intravenous infusions (14 instead of 56), treatment length is shorter, and it is more cost effective. As a result, NECT treatment is not only better for patients, it also simplifies the logistics and staffing needed at treatment centres, which are often in remote areas.
- Indication: stage-2 gambiense sleeping sickness (human African trypanosomiasis)
- Dosage: 14 intravenous eflornithine infusions for 7 days & 3 times a day oral nifurtimox for 10 days
- Recommended as first-line treatment in all 13 endemic countries, all of which receive free supplies from WHO via drug donations by Sanofi and Bayer
- 100% of stage-2 gambiense sleeping sickness patients treated with NECT in endemic African countries
- Added to the WHO Essential Medicines List in 2009, and the WHO Essential Medicines List for Children in 2013.
- Developed in partnership between DNDi, Médecins Sans Frontières, Epicentre, the HAT Platform, Swiss Tropical & Public Health Institute, national control programmes of the Democratic Republic of the Congo and Republic of Congo, with support of the World Health Organization and drugs donated by Sanofi and Bayer
‘It was 2007 when I began to feel tired and weak. I’d sleep during the day but not at night. I was so weak. I received malaria treatment in 2008, but it didn’t work. I tried traditional medicines, but it didn’t work either. Finally, in 2012 a mobile team came to the village, and we found out I had sleeping sickness. I went to the hospital and received NECT. I’m cured and feel fine now.’
Jean de Dieu Liyande Walo, remote village of Yalikombo
Key scientific articles
Prescription of concomitant medications in patients treated with Nifurtimox Eflornithine Combination Therapy (NECT) for T.b. gambiense second stage sleeping sickness in the Democratic Republic of the Congo. PLOS Neglected Tropical Diseases, January 2020
by Kuemmerle A, Schmid C, Kande V, Mutombo W, Ilunga M, Lumpungu I, Mutanda S, Nganzobo P, Ngolo D, Kisala M, and Valverde Mordt O.
Treatment options for second-stage gambiense human African trypanosomiasis. Expert Review of Anti-infective Therapy, September 2014
by Eperon G, Balasegaram M, Potet J, Mowbray C, Valverde O, and Chappuis F.
Nifurtimox-eflornithine combination therapy for second-stage gambiense human African trypanosomiasis: Médecins Sans Frontières experience in the Democratic Republic of the Congo. Clinical Infectious Diseases, January 2013
by Alirol E, Schrumpf D, Amici Heradi J, Riedel A, de Patoul C, Quere M, and Chappuis F.
In-hospital safety in field conditions of Nifurtimox Eflornithine Combination Therapy (NECT) for T. b. gambiense sleeping sickness. PLOS Neglected Tropical Diseases, November 2012
by Schmid C, Kuemmerle A, Blum J, Ghabri S, Kande V, Mutombo W, Ilunga M, Lumpungu I, Mutanda S, Nganzobo P, Tete D, Mubwa N, Kisala M, Blesson S, and Valverde Mordt O.
NECT is next: Implementing the new drug combination therapy for Trypanosoma brucei gambiense sleeping sickness. PLOS Neglected Tropical Diseases, May 2010
by Yun O, Priotto G, Tong J, Flevaud L, and Chappuis F.
Nifurtimox-eflornithine combination therapy for second-stage African Trypanosoma brucei gambiense trypanosomiasis: a multicentre, randomised, phase III, non-inferiority trial. The Lancet, July 2009
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, and Kande V.
- 11 July 2013 – Three Neglected-Disease Treatments Newly Added to WHO Essential Medicines List for Paediatric Use
- 22 September 2009 – Improved treatment for sleeping sickness now available
- 19 May 2009 – Major progress achieved on R&D landscape for sleeping sickness, one of the most neglected diseases
- 15 May 2009 – NECT added to WHO Essential Medicines list as combination treatment against sleeping sickness
- 9 December 2008 – Positive results for improved treatment against sleeping sickness
*Project cost includes direct and indirect costs, but it does not include in-kind contributions.
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