AIDS TREATMENT NEWS Issue # 185, October 15, 1993
In the laboratory, the drug is active against all strains of HIV which have been tested so far. Resistance is expected to be less of a problem with antisense compounds than with other classes of drugs. Animal studies have found that antisense drugs can work comparably in animals as in the laboratory.
An antisense drug targets specific genetic information in viruses, other infectious organisms, cancer cells, or human cells, to prevent one selected gene from working. The drug targets what is called mRNA (messenger RNA), to prevent the production of the particular protein which is specified by the gene.
The importance of antisense is that, once a technology to make one antisense drug has been developed, other potential drugs can be designed just by changing the code, as long as there is a known sequence to be targeted (which is often the case). Therefore, it may be possible to quickly develop drugs against a wide variety of diseases. Each drug should be highly specific against a particular disease, by targeting sequences which do not normally occur in the body. Therefore, it should be possible to give effective doses with few side effects.
An early laboratory study of an antisense compound against HIV was published in 1986 (see AIDS TREATMENT NEWS #58, June 3, 1988). But developing the basic technology of antisense has been difficult, since it is necessary not only to code for specific sequences, but also to do so in a way that can work as a drug in the body. Now that the technology is advanced enough for human testing to begin, further progress may be rapid.
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