Houston Scleroderma Study Testing
Chemotherapy
and Stem Cell Transplantation
HOUSTON – (June 22, 2006) – The University of Texas Medical School at Houston is part of a multi-center clinical trial that will examine the long-term effects of chemotherapy and stem cell transplantation in the treatment of scleroderma.
Maureen Mayes, M.D., professor in the Department of Internal Medicine, is now enrolling patients who have hardened skin and organ damage as a result of the autoimmune disease. Scleroderma, which causes the body to attack its own tissues, can be life-threatening.
Maureen Mayes, M.D.
Nationally, 226 patients are being recruited for the randomized study. Mayes, who is the principal investigator for the Houston site, said half of the patients will receive cyclophosphamide, a form of chemotherapy that will be administered intravenously once a month for one year at Memorial Hermann-Texas Medical Center.
The other patients will receive a high dose of chemotherapy over a five-day period at The University of Texas M. D. Anderson Cancer Center. On the sixth day, stem cells harvested from the patient’s own blood will be administered intravenously.
“We will follow the patients for five years and track their disease activity,” Mayes said. “We’re looking at the long-term effects of both these treatments. At the very least, we are hoping to see the disease stabilize so that patients don’t develop further organ damage. In the best case scenario, we are hoping to see improvement in skin and internal organ involvement.”
Patients who developed their first symptoms of scleroderma within the last four years and have diffuse skin disease with some internal organ involvement are eligible to participate in the clinical trial.
“Ordinarily, with early-diffuse scleroderma, patients can be demonstratively and dramatically worse in five year,” Mayes said. “We want to determine what will give them the best long-term outcomes and improve their quality of life.”
Preliminary research results are promising, Mayes said. In a study published this week in the New England Journal of Medicine, Mayes and other experts in the Scleroderma Lung Study Research Group found that cyclophosphamide had a significant, but modest, beneficial effect on patients’ lung function, skin thickening and health-related quality of life.
In a separate study, about 80 percent of survivors who received stem cell transplantation experienced improved skin, lung function and quality of life, Mayes said. She noted that 10 percent of the patients in the pilot study died. “We have made changes to make it much safer, but there is still a risk,” Mayes said. “We’re doing everything we can to educate patients about the risks associated with both arms of this trial.”
An estimated 300,000 people in the United States have scleroderma, which, in its most severe form, can cause damage to the skin, lungs, heart, kidneys, gastrointestinal tract and vascular system.
There are 20 rheumatology centers, including the Scleroderma Center at the UT Professional Building, that are screening patients for the clinical trial.
For information on the clinical trial in Houston, call 713-500-6454. For information on other clinical trial sites in the country, call 866-909-SCOT or visit www.sclerodermatrial.org.
Media Contact: Meredith Raine
Media Hotline: 713-500-3030