People With Treatment-Resistant Crohn's Disease May Benefit From Thalidomide

Dec. 8, 1999 (Baltimore) -- Thalidomide, the drug so many people have heard of because of its association with causing birth defects, may be useful in treating people with severe Crohn's disease who have not responded to other treatments, according to two studies in the December issue of the journal Gastroenterology.

"In our patients, the response was really quite dramatic," says Eric Vasiliauskas, MD, associate clinical director of the inflammatory bowel center at Cedars-Sinai Medical Center in Los Angeles, and lead author of one of the papers, in an interview with WebMD. "Most patients noticed an improvement within two weeks and were able to taper down on their prednisone or in some cases discontinue it entirely. It's actually quite exciting."

"Based on the observations made in this open-label series, thalidomide appears effective in inducing clinical response and remission in patients with Crohn's disease," writes Eli Ehrenpreis, MD, of the department of gastroenterology at the University of Chicago Hospitals, and lead author of the other paper.

Crohn's is an inflammatory disease of the intestines. It can attack any part of the digestive tract causing ulceration, bleeding, pain, diarrhea, weight loss, and many other uncomfortable symptoms. Most therapy is directed at controlling the inflammation by drugs that suppress the immune system. These drugs have significant side effects themselves, especially when used for an extended period of time.

Patients in both studies were very carefully selected to receive thalidomide, and received extensive education on its possible side effects. Says Vasiliauskas, "Patients in our study had to watch a video about the drug, they had to be educated by their physician, and they received the drug in a box with a picture of a thalidomide victim on it. The pills themselves come in a blister pack with a picture of a pregnant woman with an X over her on it. There's really no way you can fail to get the message."

The obvious concern is that someone who is taking thalidomide will become pregnant. Dr. Bruce Sands of the gastroenterology unit at Massachusetts General Hospital in Boston, wrote an editorial accompanying the studies. He writes, "Patients must undergo detailed counseling on the teratogenic [birth defect] potential of the drug, and acceptable means of avoiding conception during treatment must be documented." In an interview with WebMD, Dr. Sands adds, "I would use thalidomide in my patients who are refractory [resistant] to every other treatment. At this point, I see thalidomide primarily as a desperation measure."

Side effects of thalidomide treatment seen in these studies included drowsiness, skin rashes, and peripheral neuropathy, where sensations of tingling or numbness may occur. The peripheral neuropathy is potentially the most serious, according to Dr. Vasiliauskas.

Dr. Vasiliauskas says, "Our results support the need for a large, placebo-controlled study to validate the usefulness of thalidomide in these patients. I think because of the potential side effects, thalidomide really has to be used very carefully. I think newer generation drugs the pharmaceutical companies are working on will soon supplant it."

Vital Information:

  • For patients with severe Crohn's disease who do not respond to other treatments, the drug thalidomide may be an effective therapy.
  • To receive thalidomide, patients must undergo extensive education on the potential side effects, most notably birth defects, and acceptable birth-control methods.
  • A large clinical trial is needed to validate the use of thalidomide in these patients.