Clinical Use of Cantharidin

Cantharidin has a long history of use by dermatologists as a treatment for molluscum contagiosum and warts, which continues to this date (Nickolls and Tear 1954; Stoughton and Bagatell 1959; Bagatell et al. 1969; Graziano et al. 1988; Nicholls et al. 1990; Moed et al. 2001). In the United States, the Food and Drug Administration (FDA) approved cantharidin for this purpose prior to 1962. Subsequently, the Food Drug and Cosmetic Act was amended to require drug efficacy data. The data were not supplied by the manufacturers, and cantharidin was removed from the U.S. market (Moed et al. 2001).

As an antitumor agent, cantharidin has been used as a traditional medicine in China for over 2,000 years. It was also used briefly as an antitumor drug in Europe during the 1800s, but by the early 1900s it was generally considered by physicians as too toxic for internal use (Oaks et al. 1960). More recently, the clinical use of cantharidin was found effective against primary hepatoma, but its usefulness was limited by severe toxicity in mucous membranes (Sakoff et al. 1999). Interestingly, in contrast to many types of conventional chemotherapy, cantharidin has been shown to stimulate the production of white blood cells by the bone marrow in a limited number of patients (McCluskey and Sakoff 2001), but renal toxicity has hindered its development for use as an antitumor drug (McCluskey and Sakoff 2001; McCluskey et al. 2002b).

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