Few chemicals have a reputation as dark as thalidomide. Developed as a sedative in 1954 by researchers at Chemie Gruenenthal, thalidomide is a synthetic derivative of the naturally occurring amino acid glutamate, structurally similar to other sedatives. Once in the body it acts on the central nervous system through an unknown mechanism to promote sleep and prevent nausea.
Unlike the barbiturate sedatives commonly prescribed in the 50s, thalidomide has virtually no hangover effect and does not lead to dependence, features that made it a popular prescription for pregnant women experiencing morning sickness and insomnia. As such, it was initially touted as a wonder drug that could ease the discomforts of early pregnancy. However, soon after its emergence on the market, epidemics of birth abnormalities were documented worldwide. The most striking symptom of the thalidomide-affected babies was the malformation of their arms and legs, which were short, misshapen, and often absent. Other effects included defects of the ears, eyes, face, and heart. Many affected children died within their first year.
For years, drug manufacturer Chemie Gruenenthal denied claims that thalidomide had any connection to the rise in birth abnormalities, despite mounting evidence. Eventually they were sued for compensation and finally made responsible for the marketing of an improperly tested drug. It was removed from shelves and banned from use.
Thalidomide’s most far-reaching effect has been the changes to drug development it has spurred. The thalidomide tragedy led to a review of the American Food and Drug Administration’s role in drug approval, granting them new power over the regulation of drug safety and the restructuring of regulatory strategies. This new regulatory framework allowed thalidomide to be reviewed and evaluated for its beneficial effects to the immune system.
Today, thalidomide is used as a treatment for multiple myeloma and some complications of leprosy. It is currently being evaluated for use as an anti-HIV therapeutic. Stringent rules are now in place for men or women who are prescribed thalidomide: patients must agree to abstain from sex or use two effective forms of birth control to prevent any unplanned pregnancies. While the use of thalidomide will always be controlled, it shows promise for safe and effective use that may one day outshine its past.