Thalidomide Disfigured Children in 1950s, Improves
Lives for Older People with IPF
Drug relieves disabling cough for seniors - average
age 67 - with deadly lung disease, Idiopathic Pulmonary Fibrosis, study
Risk of severe, life-threatening birth defects caused by
For all people taking thalidomide:
Thalidomide must not be taken by women who are pregnant or who
could become pregnant while taking this medication. Even a
single dose of thalidomide taken during pregnancy can cause
severe birth defects National Library of Medicine
Sept. 17, 2012 It is probably a very hard pill to
swallow for senior citizens old enough to remember to shocking images on
the fuzzy TV screens of the 1950s that showed the severe birth defects
caused by thalidomide, when it was taken by pregnant women. Now,
however, a clinical trial shows the drug has the potential to improve
the lives of people mostly seniors stricken with deadly idiopathic
In the first clinical trial to demonstrate an
effective treatment for constant, disabling cough among people with IPF,
researchers at the Johns Hopkins University School of Medicine found
that taking thalidomide significantly reduced the cough and improved
quality of life.
IPF is a progressive, fatal disorder that causes
the lungs to become stiff and scarred, preventing oxygen from leaving
the lungs to go to the rest of the body. The cause is unknown. Up to 80
percent of people with IPF have a dry, nagging cough, for which no
effective treatment is available.
Thalidomide is a potent anti-inflammatory drug that
was used to treat morning sickness and aid sleep in the 1950s. It was
taken off the market in 1961 after it was shown to cause severe birth
defects when women took the drug during pregnancy.
Today, thalidomide is prescribed with strict
controls to treat several diseases, including multiple myeloma and
kidney cancer. It had not been studied for people with lung disease
"We performed a randomized, double-blind,
placebo-controlled trial of thalidomide in patients with IPF to
determine its effectiveness in suppressing cough," says lead author
Maureen R. Horton, M.D., a pulmonary disease specialist and associate
professor of medicine and environmental health sciences at the Johns
Hopkins University School of Medicine.
"We found that low-dose thalidomide significantly
reduced the cough and also improved the patients' quality of life, as
demonstrated on established questionnaires known as the Cough Quality of
Life Questionnaire and the St. George's Respiratory Questionnaire," says
For the study, patients either took low-dose
thalidomide pills or a placebo for three months. Then there was a
two-week "wash out" period in which the patients took nothing, followed
by another three months when those who had taken the thalidomide went on
the placebo and those who had been given the placebo started taking
Neither the patients nor the researchers knew which
group the individuals were in.
Twenty patients completed both arms of the study,
15 men and five women. All were over age 50 and the mean age was 67.
Horton says patients often noticed the difference
within two weeks of taking the thalidomide. When they stopped the drug,
their cough came back. "At the end of the study, all of the participants
said they wanted to continue taking the medicine because their cough had
On average, the patients reported that the
frequency of their coughing decreased about 63 percent while they were
taking thalidomide, and their respiratory-specific quality of life, such
as the ability to do daily activities, improved about 20 percent. They
also reported that the aspects of their life impacted by their cough
also improved while they were on the drug.
"The constant cough caused by the disease can
affect the quality of life in many ways," says Horton. "Some no longer
go to church or to social gatherings because people think they are
infectious. Other patients may have more pronounced urinary incontinence
due to the cough, for example, so it has wide-ranging effects."
Side effects, such as constipation, dizziness and
malaise were reported by 74 percent of the participants while they were
taking thalidomide, and by 22 percent of those who were on a placebo.
Horton says the idea of testing thalidomide for
cough among IPF patients came about because the drug is known to have a
powerful effect on decreasing inflammation. Horton and her colleagues
conducted a previous smaller study to see if thalidomide would help
treat the IPF disease itself, and while the results of that research
were not conclusive, they noticed that the patients had significant
About 80,000 people in the United States have IPF,
although it may be underdiagnosed. The risk increases with age.
Treatments for the symptoms include oxygen therapy and pulmonary
rehabilitation, but there is no effective treatment for the constant
cough, which is a hallmark of IPF. Life expectancy after diagnosis is
only about three to five years. The only cure is a lung transplant.
"Although the results were significant, this was a
small study and we believe that a larger trial is warranted to confirm
these promising results and also assess the drug's impact on the disease
itself," says Horton. "We have some hope that this therapy may be able
to slow the progression of IPF, but that would have to be tested in a
Results of their study are scheduled to be
published in the Annals of Internal Medicine on Sept. 18 in an article
titled "Thalidomide for the Treatment of Cough in Idiopathic Pulmonary
The researchers report that Celgene Corp. provided
both the funding for the trial and the study medication, but had no role
in the study design, conduct, analysis or manuscript preparation.
The study authors are Maureen R. Horton, MD;
Victoria Santopietro; Leena Mathew, BS; Karen M. Horton, MD; Albert J.
Polito, MD; Mark C. Liu, MD; Sonye K. Danoff, MD and Noah Lechtzin, MD.