Azidothymidine, or AZT, is also referred to as zidovudine. AZT is a drug that used to delay the development of acquired immunodeficiency syndrome, usually referred to as AIDS.
AIDS developed in patients that are infected with the human immunodeficiency virus, or HIV. Azidothymidine is part of a group of drugs that we know as NRTIs, or nucleoside reverse transcriptase inhibitors.
In 1987, Azidothymidine, or AZT, was the first of this group of drugs to be approved for use by the U.S. FDA (Food & Drug Administration) in AIDS patients with the purpose of prolonging their lives.
So Azidothymidine is an active drug used to treat HIV and an effective tool to prolong the lives of patients suffering from AIDS (Acquired Immunodeficiency Syndrome) and ARC.
ARC (Arthrogryposis-renal dysfunction-cholestasis) syndrome is a rarely occurring but fatal disorder caused when someone’s VIPAR or VPS33B genes are mutated.
HIV/AIDS is a rather well-known disease whereas ARC (short for AIDS-Related Complex) is less frequently occurring and causes cholestasis, congenital joint contractures, or renal tubular dysfunctions.
The rapid development of AZT, however, and the aggressive way this drug was marketed, has led to serious questions and discussions. Currently, there are numerous trials underway across the world to study the effectiveness of AZT and its toxicity when it is administered for longer periods of time.
These trials are also conducted to learn more about the potential benefits and risks in patients that are suffering from HIV-related conditions. They are also set up to determine whether AZT may be used to prevent evolution towards AIDS or ARC in asymptomatic carriers of the HIV virus.
AZT was developed long before we were confronted with the AIDS epidemic. Initially, the drug was developed for an entirely different purpose. In the mid-1960s, a Michigan Cancer Foundation researcher, Dr. Jerome Horowitz, was looking for compounds that could be used to insert the DNA of cancer cells in patients to disrupt cell replication.
He discovered AZT but when this drug was tested in small animals, it was found to be not effective so the study and further development were abandoned.
In the mid-1980s, so 2 decades later, pharmaceutical company Burroughs Wellcome (today known as GlaxoSmithKline) studied compounds to fight the booming HIV epidemy in the U.S.
The company was looking to identify compounds to disrupt the HIV virus and found that “Compound S”, a resynthesized variation of the molecule that Horowitz studied two decades earlier, was capable of blocking the activity of the virus.
Burroughs Wellcome immediately passed the results to the FDA and the U.S. National Cancer Institute, which subsequently led to one of the most controversial (but very memorable) drug trials in the history of the nation.