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17 décembre, 2006 12:18
New treatment gives hope to patients with scleroderma
By SHERRI DEATHERAGE GREEN
For The Chronicle
Stephanie Daniels couldn't bend down to put on her socks.
She noticed the first symptoms of scleroderma about 2 1/2 years ago while attending St. Edward's University in Austin. The disease's name translates to "hard skin," and for 23-year-old Daniels, it began as sore joints and dry spots on her chest.
Scleroderma can be so mild it doesn't require treatment, but Daniels' case quickly became severe. The disease attacked internal organs, sapping her energy and greatly restricting her mobility. Eventually, doctors told her, she might need a lung transplant.
"There wasn't very much hope," said Daniels, who finished her psychology degree while battling the disease. "I was in pretty bad shape."
This summer, Daniels became the first scleroderma patient to receive an adult stem cell treatment in Houston through a new clinical trial.
'It's like you're trapped'
More than 100,000 Americans have scleroderma, about 80 percent of them women. The most common symptom of the autoimmune disease is hardened skin, but damage to the lungs, heart, kidneys, gastrointestinal tract and vascular system can occur.
"Your skin gets so tight around your body, it's like you're trapped in a leather jacket," Daniels explained.
The University of Texas Medical School at Houston is one of several institutions nationally enrolling patients in a study comparing the effectiveness of a standardized course of chemotherapy against high-dose chemotherapy, plus stem cell transplantation.
Chemotherapy long has been used for scleroderma, but medications and the length of treatment varied widely.
In one small study of patients receiving stem cells, 80 percent improved and 10 percent died because of disease progression or treatment complications.
"The primary danger from this sort of treatment is that it decreases your immune system," explained Dr. Maureen Mayes, a rheumatologist and professor of medicine at UT Medical School. "You are much more susceptible to infections."
Rescuing immune system
Patients in one arm of the study receive chemo once a month for a year at Memorial Hermann-Texas Medical Center. For the others, stem cells are harvested from the patients' own blood before treatment begins. They undergo radiation and a five-day regimen of high-dose chemo at the University of Texas M.D. Anderson Cancer Center before the stem cells are returned to the body intravenously.
"In this case, the stem cells are being used to rescue the immune system," Mayes said.
The radiation and chemo kill off all mature immune cells. The stem cells then migrate to the bone marrow and, over time, create a new immune system. These new cells carry no antibodies for past diseases, however, so patients must be reimmunized for things like mumps and measles.
Because of the risks involved, only patients with severe scleroderma are eligible for the study. To qualify, a patient's symptoms must have originated in the past four years and progressed to affect internal organs as well as skin. About 220 patients will be treated nationwide.
UT last month received a five-year, $7.5 million federal grant to establish the nation's first Center of Research Translation focusing on scleroderma. In addition to testing clinical treatments such as stem cells, scientists will study the molecular basis of the disease and possible genetic links.
Daniels didn't notice immediate results from stem cell treatment as she had hoped, but as the months go by, she has gotten progressively better. She has more energy, her skin in loosening and her mobility is improving.
"My spirits are so much better now because I realize the changes are coming," said Daniels, who plans to attend to graduate school and become a therapist.
For more information about the clinical trial, call 713-500-7118.
For questions or comments on the Health & Medicine page, contact
matthew.schwartz@chron.com.
