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
"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
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."
- 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
- A large clinical trial is needed to validate the use of thalidomide in