Cancer Drug Arouses Hope for Patients Suffering with Scleroderma

Newswise — A convergence of research has created a great deal of speculation on the far-reaching implications of Gleevec for patients suffering with the autoimmune disease scleroderma. Autoimmune diseases are developed when the body’s immune system attacks healthy cells and tissues, often resulting in devastating chronic pain and organ damage.

First, Novartis brought Gleevec to market in 2001. It was a product originally developed as a treatment for leukemia; but through clinical observations, it was found also to impede inflammation, a chronic symptom of autoimmune diseases such as systemic scleroderma.

Second, researchers at the University of California at San Francisco found evidence that Gleevec could reverse diabetes in mice by blocking a cell component called platelet-derived growth factor receptor (PDGFR). This receptor functions as part of a signaling system that regulates cell growth and inflammation.

Third, a research report in the New England Journal of Medicine reported that PDGFR is a significant factor in the development of scleroderma.

Today, Dr. Daniel Furst, a professor of rheumatology at the University of California at Los Angeles, is currently leading no less than five clinical studies into the safety and efficacy of utilizing Gleevec in the treatment of scleroderma. He believes that the ramifications of this research could have effects not only in scleroderma but amongst other autoimmune diseases.

“This research also helps understand systemic sclerosis and other forms of scleroderma as well as having implications for illnesses such as liver cirrhosis and chronic obstructive lung disease,” says, Dr. Furst. Virginia Ladd, President and Executive Director of the American Autoimmune Related Diseases Association (AARDA), is encouraged by this sequence of events. “This is the first time that patients who suffer from scleroderma have a real possibility for an effective treatment. It is cause for hope for those involved in the fight against autoimmune diseases like scleroderma.”

There is currently no effective treatment for scleroderma. No drug currently can treat the problem at the cellular level however, doctors attempt to treat patients’ discomfort, by treating each symptom individually. Therefore, patients must deal with multiple specialists, multiple pills, and multiple side effects.

Kerri Connolly, Education Manager of the Scleroderma Foundation, a member of the National Coalition of Autoimmune Patient Groups is optimistic about the future, “…At this time, Scleroderma treatment is based only on symptoms/manifestations inflicted on the patients. We will still need time and patience since the "verdict" isn't out yet regarding Gleevec and its efficiency to treat Scleroderma. What this is showing is the progress the research community is making getting to the molecular level of the disease.”