The Centers for Disease Control and Prevention (CDC) now has an expanded-access investigational new drug protocol in effect with the US Food and Drug Administration to make miltefosine directly available to treat infections caused by free-living amebae (FLA).
The infections include primary amebic meningoencephalitis caused by Naegleria fowleri and granulomatous amebic encephalitis caused by Balamuthia mandrillaris and Acanthamoeba species. Even with combination treatment with drugs that have in vitro activity against FLA, the mortality rate from these infections exceeds 90%.
CDC Offers Investigational Drug. JAMA. 2013;310(15):1553. doi:10.1001/jama.2013.278652