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Investigational Drug for Treatment of Infections With Free-Living Amebae

(MMWR 62:666, 2013)—Infections caused by free-living amebae (FLA) are severe and life-threatening. These infections include primary amebic meningoencephalitis caused by Naegleria fowleri and granulomatous amebic encephalitis caused by Balamuthia mandrillaris and Acanthamoeba species. Although several drugs have in vitro activity against FLA, mortality from these infections remains >90% despite treatment with combinations of drugs.

Miltefosine is a drug used to treat leishmaniasis and also has shown in vitro activity against FLA, but as an investigational drug, it has not been readily available in the United States. With Centers for Disease Control and Prevention (CDC) assistance, however, miltefosine has been administered since 2009 for FLA infections as single-patient emergency use with permission from the Food and Drug Administration (FDA). Although the number of B. mandrillaris and Acanthamoeba species infections treated with a miltefosine-containing regimen is small, it appears that a miltefosine-containing treatment regimen does offer a survival advantage for patients with these often fatal infections. Miltefosine has not been used successfully to treat a Naegleria infection, but the length of time it has taken to import miltefosine from abroad has made timely treatment of fulminant Naegleria infections difficult.

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