Miltefosine – discovery of the antileishmanial activity of phospholipid derivatives

Abstract

Miltefosine (hexadecylphosphocholine, Impavidotrade mark), a novel antiprotozoal drug used for the treatment of visceral and cutaneous leishmaniasis, was identified and evaluated independently in the early 1980s as a potential anticancer drug in Germany and as an antileishmanial drug in the UK. Although miltefosine is not the most active compound of its class against Leishmania parasites in vitro, the early demonstration of activity after oral administration in experimental models of visceral leishmaniasis helped to bring this compound to the attention of WHO TDR for further development in a unique collaboration model with the pharmaceutical industry (Zentaris GmbH). Miltefosine is active against most Leishmania species, including those that cause cutaneous disease.