Novel concepts in the treatment of human immunodeficiency virus type 1 (HIV-1) infections by HIV-1-specific reverse transcriptase inhibitors. 1995

J Balzarini
Rega Instituut Katholieke Universiteit Leuven.

Reverse transcriptase (RT) is an appropriate target for anti-HIV chemotherapy. In recent years several entirely new leads for the design of different structural classes of HIV-1-specific RT inhibitors were provided (also called non-nucleoside RT inhibitors or NNRTI). We performed profound studies on (i) the structure-antiviral activity relationship of TSAO (a prototype NNRTI compound), (ii) the biochemical and molecular mechanism of anti-viral action of TSAO and other NNRTI's, (iii) metabolism and antimetabolic effects of TSAO's in cell culture, and (iv) the pharmacokinetic properties of TSAO in mice. In addition, the molecular basis of resistance development of HIV-1 RT against other NNRTI's have also been profoundly studied. A mapping of the resistance mutations in the binding pocket of the RT and the sensitivity/resistance spectrum of the most important NNRTI's that are subject of clinical trials have been determined. Based on the information that became evident from these studies, a molecular model of interaction of the NNRTI inhibitor TSAO with the binding pocket in the HIV-1 reverse transcriptase has been proposed, and now provide the rational basis for the development of second generation TSAO molecules that may become suppressive to mutant HIV-1 strains. Also, resistance development could be markedly delayed, modulated, attenuated, or even fully suppressed by at least two different original approaches: (i) the knock-out drug concentration principle that exploits the limited capacity of mutant HIV-1 reverse transcriptases to respond to high NNRTI drug concentrations and (ii) the rational paired drug combination therapy that exploits the mutually exclusive sensitivity/resistance properties of NNRTI drugs against the different NNRTI-specific resistance mutations in the RT. Our findings have provided us with several powerful tools to have a more efficient chemotherapeutic impact on the HIV-1 infection and to better control the emergence of viral resistance.

UI MeSH Term Description Entries
D008958 Models, Molecular Models used experimentally or theoretically to study molecular shape, electronic properties, or interactions; includes analogous molecules, computer-generated graphics, and mechanical structures. Molecular Models,Model, Molecular,Molecular Model
D004351 Drug Resistance Diminished or failed response of an organism, disease or tissue to the intended effectiveness of a chemical or drug. It should be differentiated from DRUG TOLERANCE which is the progressive diminution of the susceptibility of a human or animal to the effects of a drug, as a result of continued administration. Resistance, Drug
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000818 Animals Unicellular or multicellular, heterotrophic organisms, that have sensation and the power of voluntary movement. Under the older five kingdom paradigm, Animalia was one of the kingdoms. Under the modern three domain model, Animalia represents one of the many groups in the domain EUKARYOTA. Animal,Metazoa,Animalia
D000998 Antiviral Agents Agents used in the prophylaxis or therapy of VIRUS DISEASES. Some of the ways they may act include preventing viral replication by inhibiting viral DNA polymerase; binding to specific cell-surface receptors and inhibiting viral penetration or uncoating; inhibiting viral protein synthesis; or blocking late stages of virus assembly. Antiviral,Antiviral Agent,Antiviral Drug,Antivirals,Antiviral Drugs,Agent, Antiviral,Agents, Antiviral,Drug, Antiviral,Drugs, Antiviral
D012194 RNA-Directed DNA Polymerase An enzyme that synthesizes DNA on an RNA template. It is encoded by the pol gene of retroviruses and by certain retrovirus-like elements. EC 2.7.7.49. DNA Polymerase, RNA-Directed,RNA-Dependent DNA Polymerase,Reverse Transcriptase,RNA Transcriptase,Revertase,DNA Polymerase, RNA Directed,DNA Polymerase, RNA-Dependent,RNA Dependent DNA Polymerase,RNA Directed DNA Polymerase
D013141 Spiro Compounds Cyclic compounds that include two rings which share a single atom (usually a carbon). The simplest example of this type of compound is Spiro[2.2]pentane, which looks like a bow tie. Compounds, Spiro
D013936 Thymidine A nucleoside in which THYMINE is linked to DEOXYRIBOSE. 2'-Deoxythymidine,Deoxythymidine,2' Deoxythymidine
D014529 Uridine A ribonucleoside in which RIBOSE is linked to URACIL. Allo-Uridine,Allouridine,Allo Uridine
D015497 HIV-1 The type species of LENTIVIRUS and the etiologic agent of AIDS. It is characterized by its cytopathic effect and affinity for the T4-lymphocyte. Human immunodeficiency virus 1,HIV-I,Human Immunodeficiency Virus Type 1,Immunodeficiency Virus Type 1, Human

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