March 23, 2004
The Associated Press
Doctors report promising results using huge doses of a potent chemotherapy drug to treat autoimmune diseases including multiple sclerosis, though only a handful of patients have been treated so far and one MS researcher said far more study is needed before any victory is declared.
The drug, cyclophosphamide, is given at such high doses that it destroys most or all of a patient's disease-fighting immune cells.
However, the stem cells within the patient's bone marrow survive the drug's onslaught, the doctors say, and then are stimulated with other drugs to rebuild the immune system from scratch - but without the bad triggers that caused the body to attack its own cells.
"Once the immune cells are destroyed, they come back no longer recognizing the stimulus that brought them on," Dr. Isadore Brodsky, director of hematology and oncology at Drexel University's Hahnemann University Hospital, said Monday. "The immune system comes back naive, so it's tolerant of whatever trigger caused the autoimmune response."
So far, only six MS patients have completed the regimen, which is administered over three to five days, and the first patient finished it just six months ago. All had advanced cases of MS and had tried at least three other types of therapy, from steroids to immune-suppressing drugs, with no benefit, said Brodsky. He is developing the treatment with his son, Dr. Robert Brodsky of John Hopkins University.
The vice president for research with the National Multiple Sclerosis Society, Stephen Reingold, said the results are too preliminary to draw any conclusions.
"Any study that claims extraordinary benefits based on a short-term, uncontrolled study with a small number of patients has to be treated cautiously," Reingold said. "The big questions here are how long it lasts and whether it leaves you open for infections and other problems."
Autoimmune diseases typically are suppressed with drug interferon, steroids, radiation and other chemo drugs that stop reproduction of the confused cells that treat the body's own cells like they're foreign invaders.
Brodsky's work involves killing the misdirected immune cells, not merely suppressing them.
He has used the drug since 1997 on more than 300 patients with autoimmune diseases of the blood, peripheral nervous and neuromuscular systems, and extended it to the handful of MS patients starting last fall.
Patient follow-up and more research is necessary but the initial results were "striking and unexpected," said Dr. Robert Schwartzman, a Hahnemann neurologist who has referred patients to Brodsky for treatment.
Several of his patients who had cognitive problems, difficulty walking, or other coordination troubles have seen much of their symptoms disappear in as little as three to six weeks, Schwartzman said.
MS patient Terry Davis, 47, of Pennsville, N.J., said she underwent Brodsky's treatment in September after other therapies failed. She lost her hair, experienced severe nausea and had a "flare up" of her MS symptoms after the treatment, but those symptoms all have since subsided and she no longer needs a cane or walker to get around, she said.
"There are no words to describe how dramatically this treatment has affected the quality of my life, physically and mentally," she said.
Results of the study, which was approved by the FDA, were presented
at a symposium in Philadelphia last week.
Copyright © 2004, The Associated Press