Hepsulfam is an anticancer agent. It also displays excellent antileukemic activity (a median IC50: 0.91 μg/mL in a panel of different tumors).

Tumors:0.91 μg/ml

Hepsulfam is more toxic to L1210 leukemia cells than is busulfan, its structural homologue. Consistent with the difference in toxicity, hepsulfam induces DNA interstrand cross-links in L1210 mouse leukemia cells, whereas busulfan does not. Hepsulfam is more cytotoxic to two human leukemia cell lines (111-60 and K562) and to two human colon carcinoma cell lines (BE and HT-29) than is busulfan. As in 11210 cells, hepsulfam causes a higher level of DNA interstrand cross-links than busulfan. Hepsulfam (1.0 μg/mL) is active in eight of 37 tumors (22%) in the clonogenic assay. Hepsulfam demonstrates a clear in vitro toxicity to human bone marrow cells (CFU-GM) from healthy donors. Evaluation of equitoxic concentrations in vitro shows a higher activity of hepsulfam, especially in non-small cell lung cancer[1]. Hepsulfam is also more cytotoxic to the human leukemia cell lines when the concentrations are reduced 10-fold and the duration of drug exposure is increased to 12 h[2].

The preclinical activity of hepsulfam shows a possible role of this compound in the treatment of solid human malignancies. Hepsulfam demonstrates superior in vivo activity in a large cell lung cancer xenograft and a gastric carcinoma model. The increased bone marrow toxicity of hepsulfam as compared with the busulfan might be critical for further clinical application[1].