Lupus! AHHHHHHH!
Posted on 2/2/2006, 3:33:14 AM by neverdem
Associated Press
CHICAGO — For all of her 20s, when Edjuana Ross should have been relishing the thrill of early adulthood, she was instead in and out of hospitals, battling a disease that attacked her skin, brain and heart.
Now, at 33, she has her life back, thanks to a stem-cell transplant from her own bone marrow, a drastic, experimental treatment that could be promising for patients with severe lupus.
Ross' illness is in remission for the first time since her diagnosis shortly after high-school graduation.
"I'm just trying to get used to being well, and it's a very weird feeling," Ross said.
The Park Forest, Ill., woman is among 48 patients with severe lupus who had the treatment at Northwestern Memorial Hospital. Thirty-three patients have had no disease symptoms since their transplants, some for more than seven years, said Northwestern's Dr. Richard Burt, who led the study.
Six patients died from causes unrelated to the treatment.
The probability of disease-free survival for five years was 50 percent, encouraging for those who failed more conventional treatment for the most severe form of lupus, a disease in which the body's immune system attacks its own organs and tissues.
"It turned out very well, showing that we could do this safely," Burt said.
The study appears in today's Journal of the American Medical Association. It expands on short-term results with fewer patients the same researchers reported in 2000.
The definitive test will be a randomized study that will compare results for transplant patients with those given standard lupus treatment.
About 1.5 million people nationwide have lupus, 90 percent of them women. Most are diagnosed during early adulthood.
Classic symptoms include rashes, joint pain and fatigue. Some patients have only mild cases, but many develop debilitating disease that randomly attacks vital organs. For about 5 percent, lupus is life-threatening and doesn't respond to conventional treatment.
Ross was one of these patients. Her symptoms persisted despite massive doses of the steroid prednisone. They included scarring rashes, joint pain and extreme fatigue.
Prednisone can have severe side effects, including weight gain, thinning bones and tooth damage.
A JAMA editorial says the results "do not necessarily represent 'cure,' " and it warns that many patients likely will have late relapses. But as a last-resort treatment, "the therapy offered substantial benefit," according to the editorial by Drs. Michelle Petri and Robert Brodski of Johns Hopkins University.
Adult Stem Cell Ping!
stem cell ping
She traces the familiar piano keys with her fingers, then tries a few notes.
"Horrible," says Jane Erickson, looking scornfully at her still stiff fingers. "I need a lot of work before I can play as well as I once did." But she can play. And she can breathe. She can breathe on her own without the oxygen tube and she can even think of singing again -- maybe, someday -- and she can walk up stairs and she can bake cookies. And she can get through a day without once thinking of dying.
Because Erickson, a Westminster Choir College graduate who served for years as music teacher and choral director at Millburn High, has thought a lot about dying. Thought about how the scleroderma that afflicts her was slowly turning her skin and internal organs, lungs and kidneys, into useless, stone-hard scar tissue.
"Look," she says, and squeezes the skin on her hand. It wrinkles. Six months ago, it didn't do that. Couldn't. Anymore than the surface of stone could wrinkle. Scleroderma is an auto-immune disease and, according to a national research foundation, there is no known cause nor cure. It can be managed but, in its severest form, its progression toward death cannot be stopped. "One of the first doctors I saw told me to go home and arrange my affairs," says Erickson, 58, married and the mother of two adult children. "I'd be dead in two years." But the Maplewood resident, always active with her kids' schools, her church, her neighborhood, had two enduring assets. A refusal to accept a dark inevitability.
And a lot of friends. Including many who were strangers. Among them, you, readers of this newspaper. Erickson wanted to be included in a clinical trial of the use of stem cells to restore her immune system to something like a normal state. One of her doctors, Gary Friedman of West Orange, calls it "resetting" a wayward immune system.
Her own stem cells -- not embryonic -- would be used.
The procedure, however, is done at only three hospitals in the nation -- in Massachusetts, Illinois and Colorado. Her health insurance carrier, Horizon Blue Cross/Blue Shield of New Jersey, refused to pay for it on the grounds it was "experimental." So her friends began raising money for her and their efforts were described here. Eventually, they raised nearly all of the $135,000 cost of the procedure and it was conducted in August at the University of Massachusetts Hospital in Worcester. Neither Erickson nor Friedman will say she is cured. Nor will they say she will someday have no symptoms at all.
"But she is a fully functioning person now," says Friedman, a transplant specialist who once directed the organ transplant program at St. Barnabas Medical Center in Livingston. "That's not what Jane was before the procedure." Erickson says she feels stronger every day, but her doctors have cautioned it will be a year before they know how much improvement she can expect. "But what I have already is a gift," says Erickson, who says she has stopped taking half the medications she needed to manage the disease before the transplant.
Friedman, the chief medical officer of the New Jersey Stem Cell Research and Education Foundation, says the apparent success of her treatment is evidence the procedure should be considered standard treatment and reimbursable by insurers. "We need insurance companies and the actuaries to learn about successes like this," he says. "They are businesses and need to know this improves their bottom line." Because, he says, the treatment ultimately is less expensive than maintenance medications. Erickson says her prescriptions cost her $50,000 annually -- and Horizon Blue Cross/Blue Shield paid most of that.
The teacher, forced into retirement by her illness, feels she owes a debt to those who helped her and wants to find a way to "pay it forward," to help others. She hopes, someday, to go back to the piano and singing she loved all her life -- but she wants to do more. To "calm and comfort" others with this disease and others like it, she says, so they know they will have a future. "I am living proof," she says.
Thanks for the scleroderma update.
WooHoo for UMass Medical!! I didn't know they were doing work with adult stem cell transplants!
Bump
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