The Story Behind the First AIDS Drug

The Story Behind the First AIDS Drug
From TIME - March 19, 2017

Today, if someone is diagnosed with HIV, he or she can choose among 41 drugs that can treat the disease. And theres a good chance that with the right combination, given at the right time, the drugs can keep HIV levels so low that the person never gets sick.

That wasnt always the case. It took seven years after HIV was first discovered before the first drug to fight it was approved by the U.S. Food and Drug Administration (FDA). In those first anxious years of the epidemic, millions were infected. Only a few thousand had died at that point, but public health officials were racing to keep that death rate from spikingthe inevitable result if people who tested positive werent treated with something.

As it turned out, their first weapon against HIV wasnt a new compound scientists had to develop from scratchit was one that was already on the shelf, albeit abandoned. AZT, or azidothymidine, was originally developed in the 1960s by a U.S. researcher as way to thwart cancer; the compound was supposed to insert itself into the DNA of a cancer cell and mess with its ability to replicate and produce more tumor cells. But it didnt work when it was tested in mice and was put aside.

Two decades later, after AIDS emerged as new infectious disease, the pharmaceutical company Burroughs Wellcome, already known for its antiviral drugs, began a massive test of potential anti-HIV agents, hoping to find anything that might work against this new viral foe. Among the things tested was something called Compound S, a re-made version of the original AZT. When it was throw into a dish with animal cells infected with HIV, it seemed to block the virus activity.

The company sent samples to the FDA and the National Cancer Institute, where Dr. Samuel Broder, who headed the agency, realized the significance of the discovery. But simply having a compound that could work against HIV wasnt enough. In order to make it available to the estimated millions who were infected, researchers had to be sure that it was safe and that it would indeed stop HIV in some way, even if it didnt cure people of their infection. At the time, such tests, overseen by the FDA, took eight to 10 years.

Patients couldnt wait that long. Under enormous public pressure, the FDAs review of AZT was fast trackedsome say at the expense of patients.

Scientists quickly injected AZT into patients. The first goal was to see whether it was safeand, though it did cause side effects (including severe intestinal problems, damage to the immune system, nausea, vomiting and headaches) it was deemed relatively safe. But they also had to test the compounds effectiveness. In order to do so, a controversial trial was launched with nearly 300 people who had been diagnosed with AIDS. The plan was to randomly assign the participants to take capsules of the agent or a sugar pill for six months. Neither the doctor nor the patient would know whether they were on the drug or not.

After 16 weeks, Burroughs Wellcome announced that they were stopping the trial because there was strong evidence that the compound appeared to be working. One group had only one death. Even in that short period, the other group had 19. The company reasoned that it wouldnt be ethical to continue the trial and deprive one group of a potentially life-saving treatment.

Those resultsand AZTwere heralded as a breakthrough and the light at the end of the tunnel by the company, and pushed the FDA approve the first AIDS medication on March 19, 1987, in a record 20 months.


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