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Dermatology |
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| 19 Oct 2009 | Viewed: 166 | |
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Investigators have identified a drug that is currently approved to treat certain types of cancer, Gleevec,
that could provide the first treatment for scleroderma, a chronic
connective tissue disease for which a treatment has remained elusive.
The news was presented at the annual meeting of the American College of
Rheumatology on October 18 in Philadelphia.
"There has never been a drug that has been shown to be
effective for this condition. I think there is a very good chance of
Gleevec becoming a real treatment for a previously untreatable
disease," said Robert Spiera, M.D., an associate attending
rheumatologist at Hospital for Special Surgery who led the study.
For the study, investigators at Hospital for Special Surgery
enrolled 30 patients with diffuse scleroderma, a widespread severe form
of the disease, and gave them 400 mg of Gleevec per day. Patients were
evaluated monthly for 12 months during treatment and were seen for
follow-up three months after discontinuing the drug.
To measure the effectiveness of the drug, researchers used a
tool known as the modified Rodnan skin score, a measure of how much
skin is affected by the disease. "The skin score seems to be a very
good marker of disease status and most scleroderma trials use this as
an outcome measure," said Dr. Spiera, who is also an associate
professor at Weill Cornell Medical College. The investigators also
measured lung function using tests for forced vital capacity (FVC), the
maximum volume of air that a person can exhale after maximum
inhalation, and diffusion capacity, a measurement of the lung's
capacity to transfer gases. Lung disease is the main cause of mortality
in scleroderma.
The investigators reported an interim analysis of their
results, although the study is ongoing. At one year, the investigators
saw a 23 percent improvement in skin scores. The researchers also saw
an improvement in forced vital capacity scores by 9.6 percent and
diffusion capacity scores by 11 percent in the 18 patients who had
completed one year of treatment.
"The lung function data was really exciting," Dr. Spiera said.
"In patients with scleroderma, you usually see lung function tests
getting worse over time, and if doctors try a therapy for a year and a
patient doesn't get any worse, we get pretty excited. What is amazing
to me in this study is that we actually saw improvements in both lung
function tests."
The study is the largest single center trial of Gleevec in
scleroderma to date with the longest duration of treatment and
follow-up. Before this trial, test tube studies of human cells
indicated that Gleevec might have some activity in combating the
disease, and the drug was shown to be effective in a rodent model of
the disease. Only anecdotal evidence, however, had been published on
the drug's effectiveness in treating the disease in humans. Dr. Spiera
said the findings of his open-label study need to be interpreted
cautiously, and ultimately corroborated by evidence from a randomized
controlled trial, the gold standard of clinical trials.
Until now, no drug has been shown to be effective in treating
scleroderma in a clinical trial. Several years ago, a small study
provided some evidence that a chemotherapy drug called cyclophosphamide
may help scleroderma patients, but the benefit was minimal and this
drug causes side effects including infertility and secondary cancers.
Dr. Spiera's study was funded primarily through the Rudolph Rupert
Scleroderma Program at the Hospital for Special Surgery. Novartis, the
manufacturer of Gleevec, provided some monetary support and donated
drug. The company is not involved in the design or analysis of the
trial. Gleevec is approved in the United States for two types of
cancer: chronic myeloid leukemia and gastrointestinal stromal tumor.
Systemic scleroderma affects not only the skin, but also underlying
blood vessels, and often muscles and joints, as well as the
gastrointestinal tract, kidneys, lungs and heart. According to the
Scleroderma Foundation, roughly 300,000 individuals have scleroderma in
the United States and roughly a third of these have the systemic kind.
The disease typically strikes in the prime of patients' lives, when
they are 30-50 years old.
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| News Source: medical news today |
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