Hexadecylphosphocholine: preclinical and the first clinical results of a new antitumor drug.

Dose-response studies on cytotoxic alkyl lysophospholipids with various chemical structures revealed that a long alkyl chain and a polar group are essential for antitumor activity. The combination of both the long alkyl chain and a phosphocholine group thus results in alkyl phosphocholines. Preclinical studies with hexadecylphosphocholine (He-PC) as a representative compound indicate distinct antineoplastic activity on leukemia cells of human origin. He-PC is highly effective in inhibiting the growth of chemically induced rat mammary carcinomas. Even more striking is the fact that a high percentage of the tumors regressed completely. In a clinical phase I trial on breast cancer patients with local recurrences, topically applied He-PC resulted in regression of skin metastases. A phase II trial for topical treatment and a phase I trial for orally applied He-PC have been initiated to further evaluate the antitumoral activity of this new compound.