DFMO

DFMO, or difluoromethylornithine, is a medication also known as eflornithine. It is an irreversible inhibitor of ornithine decarboxylase (ODC), the rate-limiting enzyme in polyamine biosynthesis. By inactivating ODC, DFMO depletes cellular polyamines such as putrescine and spermidine, which are required for cell growth and proliferation. This mechanism underpins its activity against certain parasites and tumor cells that rely on polyamine synthesis.

Medical use: The primary clinical use of DFMO is in the treatment of late-stage human African trypanosomiasis (sleeping sickness) caused by Trypanosoma brucei gambiense. It is administered intravenously and has also been used as part of nifurtimox-eflornithine combination therapy (NECT), which aims to shorten treatment duration and hospital stay.

Other uses: Eflornithine is also formulated as a topical cream for cosmetic use to slow facial hair growth in women, a preparation distinct from its antiparasitic therapy.

Research and development: DFMO has been studied as an anticancer agent due to its ability to disrupt polyamine synthesis, with clinical trials conducted for neuroblastoma and other solid tumors. While it showed promise in some settings, it is not broadly approved as an anticancer drug. Ongoing research continues to explore combination strategies and targeted delivery to enhance efficacy and safety.

History and regulation: DFMO emerged in the late 20th century as both an antiparasitic and antineoplastic candidate. It remains a component of sleeping sickness management in regions where Trypanosoma brucei gambiense is endemic, as reflected in international guidelines and programs.