CONTACT: BECKY SOGLIN
2130 Medical Laboratories
Iowa City IA 52242
(319) 335-6660; fax (319) 335-9917
Release: June 23, 1999
Thalidomide is more accessible for dermatological treatments
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
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
"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
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
"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."