Could The Drug Gleevec Be The First Possible Treatment For Scleroderma? PDF Print E-mail
Saturday, 17 October 2009 17:55
Investigators have identified a drug that is currently approved to treat certain types of cancer, Gleevec, that could provide the first treatment for Scleroderma. The news will be 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.

For a link to the original article and more information on the Hospital for Special Surgery, click here.
 
More articles :

» Four More Tips For Coping With Your Scleroderma

Hosted by Jennifer Romero, jenniferforhealing is an excellent blog which seeks to spread awareness and provide support and hope for those who are suffering from Scleroderma and Lupus. In a recent post on Coping with Autoimmune Disease, Jennifer...

» What The Science Says About Stress And Relaxation Techniques

In the past 30 years, there has been considerable interest in the relaxation response and how inducing this state may benefit health. Research has focused primarily on illness and conditions in which stress may play a role either as the cause of the...

» Understanding Autoimmune Diseases

When an intruder invades your body – like a cold virus or bacteria on a thorn that pricks your skin – your protects you. It tries to identify, kill, and eliminate the invaders that might hurt you. But sometimes problems with your immune system...

» Facing Life's Challenges

At 57 years of age Anne Wersching doesn’t have a single wrinkle on her face.Sitting in her lounge room in Bayswater North, Ms Wersching looks well, save for a few red marks on her cheeks.But Ms Wersching has a little-known disease called...

» Orphan Drug Status Granted For Revimmune In Treatment of Systemic Sclerosis

, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RevimmuneTM, the company's proprietary system-of-care based on high-dose administration of Cytoxan(R) (), for the treatment of two...

» Cytokines in Systemic Sclerosis: Focus on IL-17

Julie Baraut, Dominique Farge, Elena Ivan-Grigore, Franck Verrecchia, and Laurence MichelSystemic sclerosis (SSc) is an autoimmune disease characterized by progressive sclerosis of the skin and internal organ dysfunction. Cytokine production and...

Comments  

 
+1 #1 adelwyn 2009-10-17 18:22
This really does sound promising, and I am looking forward to hearing additional findings from the American College of Rheumatology's annual meeting...
Quote | Report to administrator
 

Add comment

Do feel free to leave your comments, as they would add value and knowledge to the community. However, please refrain from making any disparaging, uninformed, or unrelated comments. Thanks :)

Security code
Refresh