Azelaic acid: potential as a general antitumoural agent.

Azelaic acid is a naturally occurring straight-chained 9-carbon atom dicarboxylic acid which is non-toxic, non-teratogenic, and non-mutagenic. Its antiproliferative and cytotoxic effect on a variety of tumoural cell lines in culture, due to inhibition of mitochondrial oxidoreductases of the respiratory chain and of enzymes concerned with DNA synthesis is well established; normal cells are unaffected at similar dosages and times of exposure. Human melanoma cells xenotransplanted onto athymic nude mice are significantly affected by administration of azelaic acid. Clinically, in humans, it has already been shown to cause regression of melanoma in situ and primary invasive malignant melanoma. These results rank azelaic acid as a potential general antitumoural agent. It can be administered topically, focally, orally, intravenously, intra-arterially, and intralymphatically, all without local or general ill-effects, and is metabolized without harmful side-products. Simultaneous administration by different routes can ensure delivery of high concentrations at lesional sites and for sustained periods. Courses can be repeated. In addition to melanoma, cutaneous and bronchial squamous cell carcinoma, bladder and breast cancers, and leukaemia would seem to be ideal candidates for further clinical investigation and trial of the anti-cancer potential of azelaic acid, as prime, adjuvant, and palliative therapy, and for disseminated disease.