Miltefosin, representing the prototype of a new phospholipid structure, was introduced for the palliative treatment of skin metastases in patients with breast cancer. It is highly active against the human leukemia tumor cells xenograft in nude mice, leading to growth inhibition and regression of large established tumors. Its mode of antitumor activity is not mediated by the host immune system but by its pharmacological effects at the level of the cancer cell membrane, distinctly different from that of the classical cytostatic drugs which interact with cell proliferation at the level of DNA replication. Protein kinase C inhibition has been suggested as a possible mechanism.
A phospholipid drug with antineoplastic and antiprotozoal/antifungal properties, also acts as an Akt inhibitor, and under investigation as a potential therapy against HIV infection.
【Antimicrobial activity】Concentrations of 1–5 μm inhibit the promastigotes and amastigotes of Leishmania spp. and the epimastigotes and amastigotes of T. cruzi. Inhibitory concentrations against T. brucei spp. and E. histolytica are closer to 50 μm. Acanthamoeba spp. are variably susceptible, depending on the experimental conditions.
【Clinical Use】Visceral leishmaniasis/Cutaneous leishmaniasis.
【Pharmaceutical Applications】An alkylphospholipid, originally investigated as an anticancer compound, formulated for oral administration.
【Storage】-20°C
