Medscape Awards in Infectious Diseases: Most Important Antiviral Agent

John G. Bartlett, MD


June 12, 2013

Gone, but Not Forgotten

AZT is no longer a very important antiviral agent, but it was selected because of its place in history:

The potential use of AZT by HIV-infected patients was not a chance observation, but was tested in clinical trials on the basis of science by thoughtful investigators at the National Cancer Institute;

The FDA trial for approval was a record-setting small and short trial, as well as highly controversial (even the chair of the external advisory board voted against approval), but it was very convincing;

The trial outcome and approval silenced the skeptics who claimed that it was impossible to effectively treat a retroviral infection, and the observed success, albeit modest and short-lived, is clearly an important contribution to the succession of the 28 drugs subsequently approved by the FDA for HIV;

Jerome Horwitz. Photo courtesy of Wayne State University School of Medicine.

The huge price tag was responsible for the launch of ADAP, which currently provides financial assistance for HIV drugs for about 210,000 people with HIV infection; and

The use of AZT to prevent perinatal transmission of HIV was the beginning of the near-end to this tragedy of thousands of babies born with a fatal disease. If you mention "076" to virtually any HIV-savvy audience, they know this trial. The number of perinatally acquired infections in the United States in 1994 promptly fell when 076 was published (Figure 1 from the 076 Trial appears here), making this one of the most effective HIV prevention strategies in the United States and, subsequently, in the world.

Unfortunately, the man who discovered AZT, Jerome Horwitz, never filed for the patent. His widow claimed that it did not matter because he was not in it for the money, but to help mankind, and his interest in science meant that he could avoid cleaning chicken coops for his father's poultry business.[7]


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