Apr 3, 2003
The Arizona Daily Star
A Tucson woman suffering severe disability from multiple sclerosis will undergo the first stem-cell transplant ever done in Arizona to fight the ravages of this disease.
The experimental procedure will be performed next week at University Medical Center on Janice Coakley, 50, whose MS has put her in a wheelchair and is making speech difficult.
Once done mainly for leukemia and other potentially fatal blood disorders, stem-cell transplants - a variation of the bone-marrow transplant - are now being tried against many other diseases, including MS, rheumatoid arthritis, colitis, lupus, Parkinson's disease, even heart failure.
Only a handful of medical centers, including the University of Arizona's, are conducting these studies.
Because stem-cell transplants for such diseases are so new, and still experimental, they are being done mostly on patients suffering severe symptoms, who have failed other treatments.
"Yes, this is a last resort for me, but I'm very excited to be able to try it," Coakley said in an interview this week in her UMC hospital room.
"I just want it to level off. I don't want to go downhill anymore. If the transplant can accomplish that, I will be completely happy. This is a good chance for me."
Stricken with MS nearly 30 years ago, Coakley had only mild symptoms and was often in remission for the first decade.
But in 1987 the remissions ended, with symptoms steadily worsening until she ended up in a wheelchair about a year ago, too weak to continue working.
At this point, she is no longer responding to any drugs for MS, and faces only a plunge toward increasing and perhaps total disability.
"In the few cases where this has been tried on MS, the result has been stabilization of the disease in about half the patients," said Dr. Elliot Epner, an oncologist and transplant specialist at the Arizona Cancer Center, who is supervising Coakley's case.
"If her symptoms actually improved, that would be best-case, but it would be surprising. We don't really expect that," he said, though global statistics seem to offer more cause for optimism.
"What I hope to see is her disease stabilizing, so she doesn't lose anymore function and quality of life," he said.
Believed to be an autoimmune disease, MS is triggered when an environmental toxin, perhaps a virus, overstimulates a person's immune system, scientists think. Immune cells then go on a rampage, attacking the person's healthy cells.
In MS, it is the protective sheath around the nerves in the brain and spinal cord that is attacked, leaving those nerves open to damage, resulting in gradual disability.
About 300,000 Americans are battling MS, with widely varying degrees of disability. For unknown reasons, it strikes twice as many women as men.
In Coakley's transplant, her own stem cells - the immature building blocks that develop into red and white blood cells - will be used, rather than those from a donor. That eliminates the risk of rejection, making it a much safer procedure than a transplant that uses donor stem cells or bone marrow.
Coakley's stem cells were harvested from her blood about a month ago, then were purified of the immune defect linked to MS and frozen.
This week, Coakley is enduring high-dose chemotherapy to destroy her diseased immune system. Next week, her purified stem cells will be thawed and reinfused into her body, to begin building her a new, disease-free system. That is almost exactly how bone-marrow and stem-cell transplants have been done for years for leukemia and lymphoma patients - except that those patients must use donor stem cells.
But it was in doing such transplants on leukemia patients with autoimmune disorders, such as rheumatoid arthritis, that doctors found the transplant not only knocked out the cancer, it also knocked back the arthritis.
And so began, in just the last few years, a series of clinical trials at several medical centers, trying stem-cell transplants against various autoimmune diseases in small numbers of patients, with the first early results just coming in now.
To date, about 70 MS patients worldwide have undergone such transplants, with about 40 percent actually improving, about 40 percent stabilizing, and about 20 percent showing no response, records show.
In one of those studies, done at the University of Washington in Seattle, of 26 transplanted MS patients, 20 stabilized but five got worse, according to an American Medical Association report.
Tried on two patients with severe Crohn's disease - an autoimmune bowel disorder causing pain and chronic diarrhea - stem-cell transplants at Northwestern University in Chicago have put patients in remission, eliminating all symptoms to date, now a year later.
And nearly full function has returned to a severely disabled Parkinson's patient after stem cells were harvested from that patient's neural tissues, purified and reinfused in a trial at Cedars Sinai Medical Center in Los Angeles.
Now jumping on this pioneering bandwagon is the University of Arizona, with its own clinical trial of stem-cell transplants in patients with lupus, scleroderma, MS and rheumatoid arthritis - all autoimmune disorders.
So far, two lupus patients have undergone transplants here, with Coakley scheduled for next week. The trial is aiming to transplant a total of some 30 patients with these diseases, said Dr. Michael Graham, director of the UA's pediatric bone-marrow transplant program.
In one of the lupus patients - a Tucson teen transplanted 18 months ago - almost all symptoms have been eliminated, returning her to normal lifestyle, said Graham.
"She was very symptomatic before the transplant - spending several days in the hospital every month, with fluid in her lungs and other complications. She had a very limited lifestyle," he said.
With lupus, the body's immune system attacks organs and other body structures, causing a wide range of sometimes disabling symptoms, including kidney dysfunction.
"We can't say she is cured, but she has clearly improved dramatically," Graham said.
The other lupus patient was transplanted only two months ago, so it is too soon to know the results, he said.
But despite these promising case reports, doctors warn that stem-cell transplants remain only an unproven promise at this point - not yet a medical revolution.
No one yet knows how long the results will last. And the transplants are expensive - around $100,000 - require weeks of hospitalization and pose risks of infection, bleeding and toxicity from the high-dose chemotherapy.
And they have yet to be tested against drug therapy or the use of even more promising embryonic stem cells, now banned in this country.
Several Tucson neurologists said they had little knowledge of stem-cell transplants for MS - including Coakley's own neurologist.
"It's basically not on our radar screen yet. I'd say a lot of doctors are not aware yet that this is a possibility," said Dr. David Siegel.
"It's really experimental - unconventional. We definitely do not have the answer yet on whether this works."
After her transplant next Thursday - a simple transfusion - Coakley will spend two to four weeks in sterile isolation at UMC while doctors monitor her blood counts to determine if her immune system is rebuilding.
When they confirm it is, she will be allowed to go home, but she will remain in superhygienic conditions for months before she will be able to venture out normally in the real world.
Coakley, a stoic woman who has already beaten back breast cancer, says she is ready.
"It's OK, I'm strong. I can handle this."
© 2003 The Arizona Daily Star Online