With 95% efficacy, drug could eradicate rare sleeping sickness, researchers say

Although African trypanosomiasis, also known as sleeping sickness, has fallen in prominence to become extremely rare, the drug acoziborole may help the World Health Organization's goal of eradicating the parasitic disease by 2030, according to researchers. 

In a phase 2/3 trial, 208 hospitalized patients with the disease in the Democratic Republic of Congo and Guinea were given acoziborole. Among the 167 study participants with late-stage African trypanosomiasis, it was found to be 95.2 percent effective. Of the 41 people who had an early-stage or intermediate-stage diagnosis, all of them "reached treatment success" after 18 months, according to the study's results published Nov. 29 in The Lancet

Fewer than 100 cases of East African trypanosomiasis and about 600 of West African trypanosomiasis were reported to the WHO in 2020, according to the CDC. The former progresses faster than the latter, with East African trypanosomiasis worsening within weeks and the other progressing within three years. Although it's rare, the disease — which can cause extreme fatigue and personality changes —  can lead to death if left untreated. 

The study's authors concluded that one dose of acoziborole could help the WHO's goal of stomping out the risk of sleeping sickness by 2030. 

"Given the high efficacy and good safety at all stages of disease, acoziborole eliminates the need for routine lumbar puncture at diagnosis and during follow-up, which requires trained staff, is associated with complications and is a source of anxiety for patients," the researchers concluded.

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