Birth defects caused by use of thalidomide

Example: Thalidomide From 1956 to 1962, approximately 10,000 children were born with severe malformities, including phocomelia, because their mothers had taken thalidomide during pregnancy. In 1962, in reaction to the tragedy, the United States Congress enacted laws requiring tests for safety during pregnancy before a drug can receive approval for sale in the U.S. Phocomelia presents at birth very short or absent long bones and flipper-like appearance of hands and sometimes feet.

Example: Thalidomide Researchers, however, continued to work with the drug. Soon after its banishment, an Israeli doctor discovered anti- inflammatory effects of thalidomide and began to look for uses of the medication despite its teratogenic effects. He found that patients with erythema nodosum leprosum, a painful skin condition associated with leprosy, experienced relief of their pain by taking thalidomide. Teratogenic = Causing malformations in a fetus

Thalidomide Further work conducted in 1991 by Dr. Gilla Kaplan at Rockefeller University in New York City showed that thalidomide worked in leprosy by inhibiting tumor necrosis factor alpha. Kaplan partnered with Celgene Corporation to further develop the potential for thalidomide. Subsequent research has shown that it is effective in multiple myeloma, and it is now approved by the FDA for use in this malignancy. There are studies underway to determine the drug's effects on arachnoiditis, Crohn's disease, and several types of cancers.

Clinical Trials Phase I: Drug is tested on healthy volunteers to determine toxicity relative to dose and to screen for unexpected side effects

Clinical Trials Phase II:Drug is tested on small group of patients to see if drug has any beneficial effect and to determine the dose level needed for this effect.

Clinical Trials Phase III: Drug is tested on much larger group of patients and compared with existing treatments and with a placebo

Clinical Trials Phase IV: Drug is placed on the market and patients are monitored for side effects