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 exhibits greater toxicity towards L1210 leukemia cells compared to busulfan, its structural analogue, by inducing DNA interstrand cross-links in these cells, an effect not seen with busulfan. This compound also shows increased cytotoxicity against human leukemia (111-60 and K562) and colon carcinoma (BE and HT-29) cell lines relative to busulfan, correlating with higher levels of DNA interstrand cross-links. In a clonogenic assay, hepsulfam demonstrated activity in 22% (8 out of 37) of tumors at a concentration of 1.0 μg/mL. Additionally, it significantly affects human bone marrow cells (CFU-GM) from healthy donors, indicating its potency. In vitro evaluations reveal hepsulfam's superior activity, notably against non-small cell lung cancer, even at tenfold reduced concentrations and extended exposure times (12 h), underscoring its potential therapeutic benefit in a broad spectrum of cancers.

The preclinical activity of hepsulfam suggests its potential in treating solid human malignancies, demonstrating superior in vivo efficacy in large cell lung cancer xenograft and gastric carcinoma models. However, its increased bone marrow toxicity compared to busulfan may be significant for future clinical applications[1].