Thalidomide: The Tragedy of Birth Defects and the Effective Treatment of Disease

Abstract

Thalidomide was a widely used drug in the late 1950s and early 1960s for the treatment of nausea in pregnant women. It became apparent in the 1960s that thalidomide treatment resulted in severe birth defects in thousands of children. Though the use of thalidomide was banned in most countries at that time, thalidomide proved to be a useful treatment for leprosy and later, multiple myeloma. In rural areas of the world that lack extensive medical surveillance initiatives, thalidomide treatment of pregnant women with leprosy has continued to cause malformations. Research on thalidomide mechanisms of action is leading to a better understanding of molecular targets. With an improved understanding of these molecular targets, safer drugs may be designed. The thalidomide tragedy marked a turning point in toxicity testing, as it prompted United States and international regulatory agencies to develop systematic toxicity testing protocols; the use of thalidomide as a tool in developmental biology led to important discoveries in the biochemical pathways of limb development. In celebration of the Society of Toxicology's 50th Anniversary, which coincides with the 50th anniversary of the withdrawal of thalidomide from the market, it is appropriate to revisit the lessons learned from the thalidomide tragedy of the 1960s.

Emerging biologics in inflammatory bowel disease

“…Thalidomide has potent teratogenicity (e.g., amelia and phocomelia) [91]. It is labeled as a pregnancy category X drug by the US Food and Drug Administration (FDA), which signifies that it is contraindicated in pregnancy [92].…”

Research on Acupuncture in Pregnancy and Childbirth: The U.S. Contribution

“…15 The ACOG's opinion came shortly after the July 1998 U.S. Food and Drug Administration approval 16 of thalidomide, which also intensified concerns about adverse effects of medical treatment in pregnancy. Thalidomide had once been used to treat pregnancy symptoms but was later banned 17,18 after the discovery that it caused severe birth defects. Although the FDA approved the drug only to treat leprosy and created restrictions on prescribing thalidomide to women, 16 concerns about risk of misuse in pregnancy nevertheless remained.…”

Challenges and Future Consideration for Pharmacovigilance

“…Its importance has been critical to public health and has become an integral part of effective clinical practice. The first systemic international efforts to address drug safety were initiated in 1961, following the thalidomide tragedy [2,3]. When thalidomide was introduced, the pharmaceutical industry comprised of number of emerging competing companies and many of them were not dedicated to drug development.…”

“…Also there were very few or no laws regulating drug development and marketing. Thalidomide use by pregnant mothers resulted in birth of thousands of congenitally deformed infants [2]. In response, efforts were initiated to address the drug safety issues by developing a system for detecting previously unknown or poorly understood adverse effects of medicines.…”