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Posted: 27 May 2011
30 years in 30 weeks, 1987

Categories: comment, opinion piece

Important discoveries were made in 1987, including the confirmation that HIV can be transmitted through breast milk to infants from infected mothers. In addition,  specific regions of the envelope protein that mediate interactions with the CD4 receptor were identified. But undoubtedly one of the most important findings of that year were the results of a clinical trial which produced a dramatic result: only a single death occurred in the treated group (n=145) vs. 19 in the placebo control group (n=137) after 24 weeks following treatment with AZT. It was quickly appreciated that the results were short-lived, undoubtedly due to the emergence, as Doug Richman’s group showed 2 years later, of the emergence of drug-resistant viral variants. Nevertheless, the 1987 trial results were an important proof of concept that it was possible to develop antiretroviral drugs that might control HIV and greatly encouraged drug companies and academic researchers to pursue other lines of anti-HIV drug development, leading to the highly successful combination therapies available today.  In her commentary, Dr. Fischl describes the results of that trial and the reaction of the scientific community to the results.

The efficacy of azidothymidine (AZT) in the treatment of patients with AIDS and AIDS-related complex. A double-blind, placebo-controlled trial

N Engl J Med. 1987 Jul 23;317(4):185-91.

Fischl MA, Richman DD, Grieco MH, Gottlieb MS, Volberding PA, Laskin OL, Leedom JM, Groopman JE, Mildvan D, Schooley RT, et al.

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1986 < All years > 1988

Commentary by Dr. Margaret Fischl


We presented in the NEJM the initial placebo-controlled clinical trial that evaluated the first drug (Zidovudine, initially called AZT) to treat the underlying cause of AIDS, the human immunodeficiency virus (HIV).  A companion paper also appeared in the NEJM that described the tolerance and toxicity of Zidovudine.  As we already recognized that HIV infection could cause a spectrum of diseases from debilitating earlier symptomatic manifestations to serious life-threatening opportunistic infection, the study was designed to evaluate a strategic approach to treating HIV infection. 

Because HIV infection resulted in a progressive destruction of the immune system that put patients at risk for multiple infections, the study was designed to enroll patients manifesting the single most common opportunistic infection PCP (Pneumocystis carinii pneumonia[1]), an infection for which we could predict outcome and fatality and thereby would be able to rapidly assess effectiveness of the treatment intervention. As one of several centers doing this study, we quickly could see the notable improvement in patients receiving Zidovudine that had not been seen when treating specific opportunistic infections and complications of HIV infection.

Our study critically established that drug therapy could be successfully directed against HIV and that treatment could improve survival and mobility for a disease that was rapidly recognized as a debilitating disease with high mortality rates. This study started an era of progressive development of treatments for HIV infection in collaboration with the pharmaceutical industry, which in just over a decade, advanced to highly active or potent antiretroviral therapy that would suppress HIV infection, preventing AIDS.

When testifying before the U.S. Food and Drug Administration, the argument was made that Zidovudine should be broadly indicated for HIV infection and not limited to patients with HIV infection with PCP.  There was every indication based on overall outcome in patients that Zidovudine was directed at HIV, and resulted in improvement in CD4 number and function with resultant improved outcome. In 1986, at the time of the study, HIV-RNA assays were not yet available andit is interesting that the initial name of the drug, azidothymidine (AZT) was not the final approved name for the drug; such a name already existed for another drug.

Our study laid the groundwork for an accelerated development of treatments for HIV that, in collaboration with the pharmaceutical industry, the academic community, and our patients, advanced to treatments that remarkably changed the outcome of what was believed at the time to be an untreatable infection.  Treatment of HIV is a success story in modern clinical medicine.  When initially described in the early 1980’s, AIDS was almost always fatal.  Now, thirty years later, treatment can suppress the HIV virus leading to near normal life.  Such progress has only been possible because of Herculean efforts by large numbers of basic and clinical researchers in academia, the pharmaceutical industry, and the NIH, with financial support from NIH, from industry, from academic institutions, and from philanthropy.  The patients involved in the studies need special recognition and thanks.  Now that successful treatments are available, this same community is now moving forward to find a cure. 

[1] now known as Pneumocystis jiroveci pneumonia

About the author:  Fischl, along with her colleagues O’Sullivan, and Scott at the University of Miami’s Miller School of Medicine, is largely responsible for eliminating the transmission of HIV from mother to child during pregnancy.
Dr. Fischl has more than 20 years of experience in directing a large multidisciplinary HIV/AIDS research program that brings together faculty across multiple departments within the University of Miami. As the director of the AIDS Clinical Research unit since 1986, she has been responsible for the implementation of the program, the development of policies and procedures for the conduct of HIV/AIDS clinical trials, and identifying and prioritizing individual clinical trials and research projects.

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