Big news in the myeloma world on Friday! Thalidomide was approved by the FDA for treatment of multiple myeloma. Thalidomide is an old drug with a dark past. Thalidomide was introduced in 1956 by Chemie Grunenthal, a West German pharmaceutical company. In Europe in the early 1960s, thalidomide was prescribed for insomnia for adults and to pregnant women for nausea. Thalidomide was known by the brand name Contergan in Europe, and Kevadon in Canada and the United States.
After several years, a German human geneticist, Dr. Widukind Lenz (1919-1995), recognized and published the connection between taking thalidomide in pregnancy and certain types of birth defects. The most common thalidomide birth defect is called phocomelia. This term describes a wide range of deformities of the arms and legs. It is estimated that 10 -12,000 babies were born around the world with varying degrees of deformity. About 5000 of these babies survive today. Although thalidomide was never sold in the United States, it was widely sampled and birth defects did occur. Thalidomide was never approved for use in the United States largely because of the work of FDA medical reviewer Frances Oldham Kelsey. Ms Kelsey, although she was under pressure from the pharmaceutical industry, refused to approve thalidomide because of emerging concerns about the possible side effects.
Thalidomide resurfaced in 1965 when an Israeli dermatologist Jacob Sheskin prescribed thalidomide as a sedative for leprosy patients. He noted that patients with erythema nodosum leprosum had resolution or significant improvement of their skin lesions within days. This lead to research regarding thalidomide's apparent immunomodulatory properties.
Bart Barlogie at the University of Arkansas was one of the first hematologists to use thalidomide for multiple myeloma. His 1999 research on a group of 84 patients with advanced myeloma showed significant regression of disease in one third and complete remission in two patients. Since then, many myeloma patients have benefited from the off label use of thalidomide, among them my husband.
Thalidomide is not a very kind drug, however. Side effects include sedation, constipation, skin rash, and permanent peripheral neuropathy. Richard has significant neuropathy, but he feels three years of almost complete remission was worth it.
So why is it a big deal that the FDA has approved thalidomide to treat multiple myeloma? Some insurance companies will not pay for thalidomide because they consider it experimantal. Now with the approved status, they can't use that argument anymore. And it is expensive. Part of the added costs are from the extensive safeguardsCelgene, has in place to prevent pregnant women from taking the drug. The thalidomide story is fascinating and there's so much more. Here is a compilation of links about the history of thalidomide.
The IMF has much information about research on thalidomide treatment of myeloma as well as practical information for patients.