What is "miltefosine"

n. (context medicine English) An antiprotozoal drug, ''2-(hexadecoxy-oxido-phosphoryl)oxyethyl-trimethyl-azanium'', used in oral treatment of leishmaniasis.

Miltefosine ( INN, trade names Impavido and Miltex) is a broad-spectrum phospholipid antimicrobial drug. Chemically, it is a derivative of lysophospatidylcholine. It was developed in the late 1980s as an experimental cancer treatment by German scientists Hansjörg Eibl and Clemens Unger. Simultaneously, but independently, it was found that the drug could kill Leishmania parasites, and since the mid-1990s, successful clinical trials were conducted. The drug became the first (and still the only prescribed) oral drug in the treatment of leishmaniasis. It is now known to be a broad-spectrum antimicrobial drug, active against pathogenic bacteria and fungi, as well as human trematode Schistosoma mansoni and its vector host, the snail Biomphalaria alexandrina. It can be administered orally and topically.

On 19 March 2014, the US Food and Drug Administration approved miltefosine for any form of leishmaniasis, specifically for patients of 12 years of age and older, and became the first approved drug for cutaneous or mucosal leishmaniasis.

In 2013, the US Centers for Disease Control and Prevention recommended miltefosine as a monotherapy for granulomatous amoebic encephalitis and primary amoebic meningoencephalitis, two fatal protozoal diseases. Historically, only four survivors have been recorded out of 133 confirmed infections in North America. One American survived the infection in 1978 and one individual from Mexico in 2003. In 2013, two children survived and recovered from primary amoebic meningoencephalitis after treatment with miltefosine.

In the target cell, it acts as an protein kinase B (Akt) inhibitor. Therefore, it is also under investigation as a potential therapy against HIV infection.