New Scleroderma Treatment
New Treatments Offer Hope in the Fight Against a Cruel Skin-Hardening Ailment
Before Steve Nickerson, a photographer at The Rocky Mountain News in Denver, began his treatments for systemic scleroderma, the illness had already sabotaged his body on multiple fronts.
His skin and fingers were so severely stiffened — “tough as rhino hide,” he recalled one doctor saying — that he could not tie his shoes and could barely hold his Nikon. His lungs became scarred. He became so weak that he could not climb a single step without gasping for breath.
Even eating became arduous: his mouth would not open sufficiently for a normal bite.
“I can tear an apple apart, sort of animal-like,” said Mr. Nickerson, who measures the progress of his treatments according to increased jaw opening. “I have gone from 23 millimeters to 27,” he said.
Scleroderma means “hard skin,” a hallmark of the illness that can turn hands purple as if from frostbite and can curl fingers into woodlike nonfunctioning. Rare and enigmatic, it is a chronic, often progressive rheumatic disease in which the immune system overproduces collagen, which can stiffen and thicken the skin, typically on the hands, arms, legs and face.
The symptoms and their severity can vary greatly among patients, and the illness takes two main forms. Systemic scleroderma — which can ravage not only the skin but internal organs like the heart, lungs and kidneys — can be life-threatening. Localized scleroderma can be limited to patches of thickened or discolored skin, while internal organs are spared. This form does not lead to systemic scleroderma and is not fatal.
There is no cure for scleroderma. But doctors are using a growing number of treatments for people like Mr. Nickerson, who have the more serious systemic form, also called systemic sclerosis. As recently as June, The New England Journal of Medicine reported on a new study in which a drug called cyclophosphamide, or Cytoxan, modestly helped slow thickening of the skin and deterioration of lung function.
“For people with scleroderma, it really is the best and worst of times,” said Chris Underation of the Scleroderma Foundation in Danvers, Mass. “The best part is that there are now more treatment options than ever, and more are coming. But the worst part is that many of these treatments are beyond the financial reach of most people if their insurance will not cover them.”
Some newer treatments like stem cell transplants have shown great promise over the last few years, said Mr. Underation, the group’s communications manager. “But these new treatments,” he added, “are considered experimental and can easily run into five figures each time a patient receives one. Costs quickly rise, and patients quickly become financially desperate.”
At the Rocky Mountain Cancer Center in Aurora, Colo., on roughly two consecutive eight-hour days every month, Mr. Nickerson, 49, is hooked up to an intravenous drip that is “exhausting, boring and time-consuming,” he said.
His friends and colleagues raised $83,500 for his intravenous immune globulin treatments, which infuse blood and give him new antibodies. Mr. Nickerson’s insurer considered the treatments experimental and denied coverage. They cost around $37,000 each; 13 were ordered.
“The treatments are saving my life,” said Mr. Nickerson, who felt strong enough last month to increase his work schedule from three days a week to four. “I was a wreck last year at this time.”
By studying scleroderma, researchers are learning more about autoimmune diseases and the process that underlies them, the odd phenomenon of a body attacking itself. “While scleroderma is a rare disease, the lessons we learn from it will help us in the treatment of many common and uncommon diseases,” said Dr. Fredrick M. Wigley, director of the Johns Hopkins Scleroderma Center in Baltimore. He is also Mr. Nickerson’s doctor.
About 300,000 Americans have scleroderma, including 100,000 with the more serious form, said Dr. Daniel E. Furst, professor of rheumatology at the University of California, Los Angeles.
About 20 percent of those with systemic sclerosis have a severe manifestation of the disease, Dr. Furst said, with 50 percent mortality over five years. Scleroderma occurs most frequently among women in their childbearing years, Dr. Furst said.
Emily Woods, 35, of Plano, Tex., is one of them. She was so sick at one point that she dropped to 87 pounds.
“She was close to death until a stem cell transplant saved her life,” said Mr. Underation of the Scleroderma Foundation. “She was unable to clothe and feed herself. Now she drives and is quite active and enjoying her young daughter.”
With more research, the face of the disease is changing, Dr. Furst said. “Until around 1995, many patients with the worst kind of scleroderma, particularly those with pulmonary hypertension, died very quickly,” he added. “In this last year, a lot of new and hopeful things have happened in scleroderma research.”
“We understand the genetic background of this disease better,” Dr. Furst continued. “And new drugs to treat pulmonary hypertension in scleroderma — increased blood pressure in the lungs, which formerly caused 50 percent mortality in two years from diagnosis — are coming out in a steady stream. Within a year, we hope there will be five drugs to treat pulmonary hypertension in scleroderma.”
There is other encouraging news. Besides controlling symptoms of pulmonary arterial hypertension and heart failure, Dr. Wigley at Johns Hopkins said, new drugs are being used to treat the gastrointestinal manifestations of the disease, stop the progression of lung disease and reverse the rapid renal failure that can occur.
New medicines can also control arthritis associated with the illness and help people cope, for example, by easing depression, he said.
Dr. Keith M. Sullivan, a professor of medicine who specializes in stem cell transplantation at the Duke University Medical Center, sees other developments on the horizon.
The research suggests that a regimen of intensive chemotherapy or total body irradiation, followed by a treatment in which stem cells are removed from a patient’s blood and then given back, “may actually reset the immune system,” Dr. Sullivan said, “leading to long-term control of autoimmune disease.”
“The patient receives back the purified stem cells, which regenerate the marrow and immune systems,” he said.
Beyond that, large clinical trials of new treatments for scleroderma also offer hope. Dr. Sullivan is the principal investigator for a trial called Scleroderma: Cyclophosphamide or Transplantation, or SCOT, and Dr. Furst is principal rheumatologist.
The trial compares the potential benefits of a stem cell transplant with high-dose monthly treatment with cyclophosphamide, a chemotherapy drug given for chronic immunosuppression.
A Web site, sclerodermatrial.org, provides patient information and the locations of centers across the United States that are enrolling patients with severe forms of systemic sclerosis.
“The proof of this hope lies not only in our scientific publications and data, but also in the patients’ own voice,” Dr. Sullivan said.
He described how, at a recent meeting to discuss the clinical trial, a patient who had been treated for scleroderma addressed the assembled doctors. “She was several years after transplant and described the new normalcy of her life,” Dr. Sullivan said. “Rarely have I seen a group of 80 doctors close their laptops, sheath their BlackBerrys and give undivided attention to such a compelling story. That is the hope and the message.”
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