Magazine article Drug Topics

Second Time Around

Magazine article Drug Topics

Second Time Around

Article excerpt

Thalidomide approved for leprosy

complication

Looked upon with loathing by victims of its horrific fetal effects and their families, the very drug that gave rise to the term teratogenicity has returned from the nevernever land of medicinal nightmares.

Actually, thalidomide was never completely banned from worldwide use. After reports of the drug's link to the tragic birth defect phocomelia (shortened limbs) in the 1960s, it was withdrawn from the countries where it was marketed. And it had never been approved for sale in the United Statesmostly due to concerns over reported cases of peripheral neuropathy.

But in the years following, researchers have never taken their eyes off thalidomide. After demonstrating efficacy in a variety of disease states, the drug was granted orphan status for tuberculosis and several AIDS-related conditions. And just recently, the Food & Drug Administration-ironically, the same agency that established a stringent drug approval process as result of the thalidomide tragedy-approved thalidomide for the treatment of erythema nodosum leprosum (ENL). The severe and debilitating leprosy-associated complication manifests as painful skin nodules, which disappear after several days but frequently recur and may form pustules that ulcerate and cause scarring. Other symptoms, such as fever, joint inflammation, malaise, weight loss, and abnormal blood counts, cause patients to become fatigued and chronically ill.

Thalidomide is thought to improve symptoms by inhibiting the production of tumor necrosis factor alpha (TNFalpha), an inflammatory cytokine found in high amounts in patients suffering from ENL. The agent will be marketed by Celgene Corp. under the brand name Thalomid.

The FDA's decision was based on overwhelming data from several clinical trials in which more than 90% of patients treated with thalidomide for ENL improved, with skin lesions and fever disappearing within a few days and other disease manifestations resolving in one week or more. In addition, an ongoing trial that began in 1975 has found that 83% of patients suffering from ENL had complete control of the leprosy complication during the first year, and another 13% reported partial control. More than 90% of those who had complete control remained free from ENL for 14 years.

The benefits of thalidomide certainly seem indisputable, but are they hefty enough to outweigh the potential consequences of introducing a well-known teratogen into the vast U.S. market? Pharmacists appear to be sounding a cautious Yes. Michael Wynd, Pharm.D., clinical assistant professor at New Jersey's Rutgers University College of Pharmacy and clinical pharmacy specialist at Hackensack University Medical Center, believes that "since thalidomide is used for a variety of off-label refractory conditions besides its newly approved indication, the drug could impact many patients and should be made available to them with adequate control."

Similarly, Henry Cohen, Pharm.D., M.S., associate professor of pharmacy practice at Arnold & Marie Schwartz College of Pharmacy, Brooklyn, N.Y., agrees with the FDA's approval decision. Cohen, also clinical coordinator of pharmaceutical services and clinical R.Ph. at Kingsbrook Jewish Medical Center, Brooklyn, believes that thalidomide is "no worse" than some other problem-prone products such as potentially toxic chemotherapies and another pregnancy category X drug Accutane (isotretinoin). …

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