Anti-Human Immunodeficiency Virus and Anti-Hepatitis-B Virus Activities and Toxicities of the Enantiomers of 2'-Deoxy-3'-oxa-4'-thiocytidine and Their 5-Fluoro Analogs in Vitro

Journal of Medicinal Chemistry
1995.0

Abstract

Reverse transcriptase (RT) is essential for the replication of human immunodeficiency virus (HIV), and clinically approved RT inhibitors face challenges of significant side effects and drug resistance, creating a need for new agents with low toxicity. Herein, we describe the antiviral activities of both enantiomeric forms of novel optically active 2'-deoxy-3'-oxa-4'-thiocytidine (dOTC) and their 5-fluoro analogues (dOTFC), a class of 2,4-disubstituted 1,3-oxathiolane nucleosides that hybridize 4'-thio and isonucleoside structures. We evaluated their anti-HIV-1 activity in cord blood mononuclear cells (CBMCs) and multiple cell lines (MT-4, Jurkat, H9, U937, CEM), and anti-hepatitis B virus (HBV) activity in the transfected human hepatoma cell line 2.2.15. Results show that compounds 2, 3, and 4 are potent and selective inhibitors of HIV-1 replication in vitro, with no cytotoxicity at concentrations up to 500 μM. Compound 1 exhibits potent anti-HIV-1 activity but only modest anti-HBV activity (IC50 = 17.5 μM), which is 1000-fold less potent than lamivudine, and is more cytotoxic than AZT in Jurkat, U937, and CEM cells. Among the enantiomers, 1 is 3-4 fold more potent than 2-4 but less selective, while 2 displays superior selectivity. Compounds 3 and 4 have similar potency and no cytotoxicity, an unprecedented observation in nucleoside research. The 5-fluoro substituent has negligible effects on the potency and cytotoxicity of 2 and 4 but reduces the cytotoxicity of 3. In conclusion, we report the first examples of enantiomerically pure 4'-thio nucleoside analogues with potent and selective anti-HIV-1 activity in primary lymphocytes and cell lines. Further details on resistance profiles and cellular metabolism will be reported in forthcoming publications.

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