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Release: June 23, 1999

Thalidomide is more accessible for dermatological treatments at UI

IOWA CITY, Iowa -- In the 1950s and 60s, thalidomide gained notoriety by causing serious birth defects in the children of mothers who took the prescription drug as a sedative. Now, the drug is accessible for dermatological uses. Authorized physicians nationwide, including University of Iowa Health Care dermatologists, prescribe the drug to effectively treat certain severe skin conditions.

The UI department of dermatology began using thalidomide in December to treat patients with skin lupus whose disfiguring skin lesions have not responded to conventional treatment such as anti-malarial drugs, said Richard Sontheimer, M.D., UI professor and head of dermatology. Sontheimer is one of three UI dermatologists registered with the Federal Drug Administration (FDA) to prescribe the drug.

"Three out of four patients with discoid or subacute cutaneous lupus will respond to the anti-malarial drugs, but for people who don't, thalidomide is rapidly becoming one of our most useful next lines of treatment," Sontheimer said.

Lupus affects about one in 20,000 individuals and occurs in women more than men. People with skin lupus have persistent, scaly lesions on their face, scalp, arms and hands that can result in disfiguring scarring, hair loss and too much or too little pigmentation.

Thalidomide begins to work much faster than anti-malarial drugs, in about two weeks rather than two months, to heal skin lesions and halt hair loss or the progression of pigmentation problems. Thalidomide also offers an advantage over other anti-inflammatory drugs and immunosuppressants, which have previously been used as alternative treatments.

"Immunosuppressants like prednisone may treat the lesions, but they also block the body's ability to respond to infections and ward off tumor cell formation," Sontheimer said. "Thalidomide just doesn't seem to present the risk of making a person more susceptible to infection or cancer."

The benefits of thalidomide are not long term but usually last several months. Patients with skin lupus will usually need follow-up treatments after their initial course of the drug.

Sontheimer said many people with skin lupus may otherwise have their health intact, but the severe disfigurement can make their lives extremely difficult.

"Having to deal with the chronic, visible lesions and other symptoms every day is a great burden," he explained. "The condition puts some people at significant risk for depression. After treatment with thalidomide, our patients really appreciate having less attention brought to their skin."

Sontheimer has also prescribed thalidomide to patients with pyoderma gangrenosum, a skin condition that produces painful and disfiguring ulcers. He said there's also great potential for thalidomide in treating other skin and mucosal membrane conditions such as sacroidosis and apthous ulcers produced by Bechet's disease and AIDS.

"It's understandable that there's a lot of emotion surrounding thalidomide and that the earlier use of the drug scared many people away from using it," Sontheimer said. "However, it's a very useful and safe drug in many different ways.

"Thalidomide has one of the most restrictive protocols for any new drug ever released onto the U.S. market. It's easier to get morphine, by far. We must follow very strict guidelines in prescribing thalidomide, including risk notification and extensive education concerning side effects for our patients."

Sontheimer added it is absolutely critical that all aspects of the prescribing protocol be adhered to compulsively by both patients and physicians.

Thalidomide was first developed in the 1950s to treat nausea and sleeplessness in pregnant women. Thought to be safe, the drug caused thousands of affected babies to be born with foreshortened and malformed arms and legs. The drug was prescribed primarily in Europe and never received FDA approval in the United States. Some families in the U.S. armed services stationed overseas were affected when pregnant American women received the drug in Germany or Italy.

"After the drug was withdrawn, there was strong pressure to never again use the drug," Sontheimer said. "Activist groups lobbied to keep thalidomide off the market because they didn't think any benefit would be enough to overcome the risk of more people being born with birth defects."

In the mid 1970s, researchers discovered the drug could help treat autoimmune connective tissue diseases. A few years later, at his previous position at the University of Texas Southwestern Medical School, Sontheimer started using thalidomide to treat patients with skin lupus.

"We had uniformly good results with thalidomide, but the drug was hard to get," Sontheimer said. Physicians needed an FDA permit for each patient's treatment. The approval process took up to three months.

Research and clinical use of thalidomide in countries such as Mexico, where there has been greater access to the drug, revealed its effectiveness in treating various skin diseases. A recent University of Rochester study further documented that thalidomide clears skin dramatically for people with difficult skin lupus. The FDA approved the drug in September 1998 for severe complications of leprosy treatment.

"Once a drug is approved for one disease," Sontheimer explained, "it is legal for physicians in their best medical judgment to use the drug for another disease where it might be useful, even if the FDA has not yet approved it for this new indication."

Boston University is tracking thalidomide use by the 1,000 physicians with FDA approval to prescribe the drug. Sontheimer and his patients participate in the study.

Before using thalidomide, patients must sign consent forms and review a packet of materials from Celgene, the Warren, N.J., company that manufactures the drug. The packet includes a video and a letter from the Thalidomide Victims Association of Canada about the drug risk to pregnant women.

Women must not conceive or bear a child while on thalidomide, Sontheimer said. In fact, they must agree to use two effective forms of birth control during treatment as well as four weeks before and after the treatment. Men using the drug must also use a barrier form of birth control. In addition, physicians are careful to keep daily dosages of thalidomide at 50 to 100 milligrams, well below the 300 to 400 milligram level suspected to cause sensory nerve damage. Minor drug side effects include sleepiness and/or constipation.

Sontheimer said that at first researchers didn't fully understand how thalidomide worked to successfully treat skin problems. He says there's now "good evidence" that the drug has a powerful effect on blocking inflammatory processes in the body by suppressing cytocine formation.

Thalidomide also blocks angiogensis, the formation of new blood vessels. The drug is being used at other institutions to treat diseases in which new blood vessel formation is a problem, such as retina conditions. Thalidomide is also being used in certain systemic disorders such as Crohn's disease, a form of inflammatory bowel disease.

"Thalidomide is a very interesting drug that's had a bad reputation," Sontheimer said. "We can't hold back on developing prudent uses of the drug. It's potentially too important for too many patients with a number of difficult-to-manage conditions for it not to be explored."