Posted on 08/13/2006 9:25:54 PM PDT by Coleus
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 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, making them woodlike and 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 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 Underation, the group's communication 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 two consecutive eight-hour days every month, roughly, 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. 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 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 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 at Los Angeles.
About 20 percent of those with systemic sclerosis have a very severe manifestation of the disease, Furst said, with 50 percent mortality over five years. Scleroderma occurs most frequently among women in their childbearing years, Furst said.
Emily Woods, 35, of Plano, Texas, 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 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, Furst noted. "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," 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."
There is other encouraging news. Besides controlling symptoms of pulmonary arterial hypertension and heart failure, Wigley at Johns Hopkins said new drugs were being used to treat the gastrointestinal manifestations of the disease; others to stop the progression of lung disease; still others to 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," 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. Sullivan is the principal investigator for the Scleroderma: Cyclophosphamide or Transplantation, or SCOT, trial, and 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," Sullivan said.
A friend of my daughter went through this. She underwent stem cell therapy and is doing much better.
I didn't see mention in the article of whether adult or embryonic stem cells were used...but i kind of skimmed it and could have missed it.
If the stem cells come from another person, then it is called an allogenic stem cell transplant. So these are adult stem cells we're talking about.
The only reason I know about this is because my wife has lymphoma, and a stem cell transplant may be one of the treatment options in her future.
I would guess just the opposite. I don't think they like to mention adult stem cells because it will showcase how successful it is. They would jump at the chance to mention embryonic stem cells if there was any success that way it would rally support. I'm guessing the stem cells used in this case were adult.
IIRC embryonic stem cells usually create tumors so are not yet in use in humans. You will never read that in the MSM, though.
Thanks for the replies...interesting.
My prayers for your wife, vrcw1.
Thanks moonhawk.
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