miltefosine

Miltefosine is an oral small molecule antiprotozoal and antimicrobial agent primarily used to treat various forms of leishmaniasis, including visceral, cutaneous, and mucosal leishmaniasis. Originally developed in the 1980s as an anti-cancer agent, it is now the only recognized oral treatment for these neglected tropical diseases. Miltefosine has also been used off-label for infections caused by free-living amoebae such as Acanthamoeba, Balamuthia mandrillaris, and Naegleria fowleri. Its mechanism of action involves disruption of lipid-dependent cell signaling pathways in parasites; specifically, it inhibits phosphatidylcholine biosynthesis (selectively targeting parasite enzymes), disrupts mitochondrial function via inhibition of cytochrome-c oxidase leading to apoptosis-like cell death, and disturbs intracellular calcium homeostasis by activating plasma membrane Ca2+ channels in Leishmania species[2][6][8]. It may also inhibit Akt (protein kinase B) involved in cell cycle regulation[2]. Miltefosine is approved for use in patients aged 12 years or older[1][5].

Brand names

Impavido

Other names

hexadecylphosphocholine