'HiCy' drug regimen may slow MS progression
Posted June 11th, 2008 by Mohit JoshiHealth Update United States
Washington, June 11 : Researchers at Johns Hopkins University School
of Medicine have found that a short-term, very-high dose regimen of
the immune-suppressing drug cyclophosphamide may slow progression of
multiple sclerosis (MS) and may even restore neurological function
lost to the disease.
The findings in nine people, most of whom had failed all other
treatments, suggest new ways to treat a disease that tends to
progress relentlessly.
"We didn't expect such a dramatic return of function. Although we're
very early in the game, we think this approach could be the linchpin
of a significant advance for MS treatment," says Douglas Kerr, M. D.,
Ph. D, associate professor of neurology at the Johns Hopkins
University School of Medicine.
Researchers have used the so-called HiCy treatments with some success
at Johns Hopkins for a variety of other immune system disorders,
including aplastic anemia, lupus and myasthenia gravis.
Cyclophosphamide kills immune-system cells but spares the bone marrow
stem cells that make them.
The usual method of delivering it in pulsed, small doses, however,
can cause the drug to build up to toxic concentrations in patients'
bodies, causing a variety of side effects, including a greatly
increased risk of infection.
Seeking an alternative way to use the drug, researchers reasoned that
HiCy might clear out the majority of a patient's immune system in one
fell swoop, then allow it to `reboot,' giving nerve cells a fresh
start and an opportunity to repair themselves.
In the current study, nine MS patients got a total single infusion of
200 milligrams per kilogram of cyclophosphamide intravenously over
four days, a dose several times higher than that given in pulsed
regimens but significantly lower than the total amount usually given
patients over time.
Before treatment, Kerr said, the study participants were `the worst
of the worst' among MS patients.
Eight of the nine patients had failed conventional MS treatments, and
several of them were wheelchair-bound.
Researchers said that the disease appeared to reverse course for
seven of the nine patients over two years following treatments.
Overall, the patients, men and women ranging in age from 20 to 47 at
the beginning of the study, experienced a 40 percent reduction in
scores of a standard test that measures disability.
They also had an overall 87 percent improvement in scores on a
composite test that measures physical and mental function.
However, Kerr has warned that that the `reboot' phenomenon didn't
work in all the patients.
Two years after treatment, MRI images showed that the disease had
reactivated in about half the study participants, suggesting that
their renewed ability may not be permanent.
Kerr's colleague Adam Kaplin, M. D., Ph. D., assistant professor of
psychiatry and neurology at the Johns Hopkins School of Medicine, is
leading efforts to improve HiCy therapy with a blood test in
development that could predict which patients would benefit the most
from HiCy treatment.
Also, since immune cells that regrow after HiCy treatment may contain
the same defect that leads to MS, Kaplin and his colleagues are
working on a way to regrow only healthy immune cells.
The study is published in the June 9 Archives of Neurology. (ANI)
http://www.topnews.
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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