ScienceDaily (Nov. 19, 2007) — A small clinical trial in Uganda, conducted within a long-established Médecins Sans Frontières (MSF) treatment program for African sleeping sickness, has found that a new combination treatment using the drugs nifurtimox and eflornithine holds promise and deserves further evaluation.

The parasitic disease African sleeping sickness (human African trypanosomiasis, or HAT) threatens millions across remote and conflict-affected regions of sub-Saharan Africa, and causes about 15 000 reported cases every year. Without treatment, HAT progresses from stage 1, infection of the blood and lymph, to stage 2, invasion of the central nervous system, and then to death.

There are few drugs available for treating stage 2. The historical mainstay, melarsoprol, is highly toxic and is ineffective in some areas due to parasite resistance. Eflornithine is the only viable alternative, already established as safe and efficacious, but it is difficult to administer--it is given intravenously and requires 24 hour nursing care, placing an additional workload on the already fragile health systems in HAT-endemic countries.

The new trial, by Gerardo Priotto (Epicentre, Paris, France) and colleagues at MSF and Epicentre, reports on a series of 48 patients treated with a novel combination of nifurtimox (a drug registered for Chagas disease) and eflornithine. Seventeen patients were recruited as part of a terminated randomized trial (previously published in PLoS Clinical Trials) and 31 in a subsequent case series study.

Despite the low sample size, the findings were promising: no cases of treatment failure, no treatment terminations, and no HAT- or treatment-related deaths.

"Nifurtimox plus eflornithine may be the best treatment hope for stage 2 HAT patients in the next decade, while new drugs are developed," say the authors. "A larger, multi-centric trial of the combination is ongoing."

Citation: Checchi F, Piola P, Ayikoru H, Thomas F, Legros D, et al. (2007) Nifurtimox plus Eflornithine for Late-Stage Sleeping Sickness in Uganda: A Case Series. PLoS Negl Trop Dis 1(2): e64. doi:10.1371/journal.pntd.0000064

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