A drug brings hope for the eradication of sleeping sickness

Is the eradication of “sleeping sickness” – or African trypanosomiasis – within reach? It is the hope carried by the results of a phase 2 trial, published Wednesday, November 30 in The Lancet Infectious Diseases and according to which acoziborole – a molecule identified more than fifteen years ago for its antiparasitic properties – could make it possible to treat, in a single oral dose, this fatal disease. With, as a possible corollary, the eradication of the disease in the short term.

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The first results obtained on some 200 patients are striking. Among the patients enrolled at an advanced stage of the disease, more than 95% no longer showed any symptoms eighteen months after a single dose of 960 mg of acoziborole. “And if we stick to those who have completed the protocol, we obtain 98.1% success, specifies Antoine Tarral, doctor at the non-governmental organization Drugs for Neglected Diseases Initiative (DNDi), who coordinated this work. These are spectacular results. » In all the patients enrolled at an early stage of the disease, the treatment proved to be effective, with no sign of relapse eighteen months after taking the treatment.

The authors further consider that the drug is well tolerated; the four deaths occurring in the cohort during the eighteen months of follow-up are, they write, “not related to the disease or the treatment studied”. Currently underway, a larger trial, on 900 patients, with a randomized double-blind protocol – patients and clinicians participating in the trial not knowing which of the compared treatments they are receiving or administering – will have to confirm the safety. of the molecule.

600 cases in 2020

In its most common form, which represents more than 95% of cases, the disease, transmitted by the tsetse fly, progresses slowly. The parasite multiplies in subcutaneous tissue, blood, and lymph nodes, causing headaches, joint pain, and other nonspecific symptoms. Then, when the trypanosome crosses the blood-brain barrier and infects the central nervous system, the disease is manifested by severe neurological disorders, irrepressible drowsiness, and then death.

Transmission of the disease has been dramatically reduced over the past two decades, from nearly 40,000 reported cases in 1998 across Africa (arguably much more in reality), to around 600 cases in 2020, a majority in the Democratic Republic of the Congo.

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