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Cyclophosphamide | ||
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This article,
although written about lupus, helps to describe the action of cyclophosphamide
in altering the autoimmune system.
An
intense blast of a cancer drug could help patients with the potentially fatal
disease lupus, researchers suggest. Lupus is a disease where
the immune system turns on the body's organs and tissues, continually damaging
them. It affects around 40 to 50
people per 100,000, most commonly affecting women aged around 30. But US researchers have
been able to successfully treat the condition with a high-dose
intensive treatment using the anti-cancer drug cyclophosphamide.
The
treatment helps "reprogramme" the immune system by
"blasting" the lupus to wipe out the abnormal immune system. The
body can then relearn to function normally. Lower doses of the drug
are already used in lupus treatment, but only 25% of patients fully respond
after six months. Organ failure
Researchers from Johns
Hopkins Lupus Center and Kimmel Cancer Center gave 14 patients a four-day
high-dose of cyclophosphamide directly into their veins. All had failed to respond
to standard treatments and suffered significant organ failure. They were then followed up
for over two-and-a-half years. Three have remained
completely disease-free after stopping the treatment, six saw some improvements
and take lower doses of immune suppressing drugs, which had previously failed to
work. Two did not respond to the
therapy, and one had some response but then developed a new renal condition. Previous
research had suggested high-dose cyclophosphamide treatment could control
autoimmune diseases including lupus and aplastic anaemia. In the traditional
low-dose treatment, patients given cyclophosphamide over a long period of time
could experience side effects such as ovarian failure, severe osteoporosis and
high blood pressure. In this latest study, no
patients had premature ovarian failure, and all 11 pre-menopausal patients
continued to have periods. However, some side-effects
such as hair loss, nausea and temporary low blood cell counts were seen. Stem cells
Michelle Petri, a
professor of rheumatology who led the study, said: "Living with long-term
severe lupus is devastating as the body's immune system attacks itself. "Lupus has
permanently damaged one or more organ systems in about half of all our patients,
in spite of currently available therapies." She added: "The
idea with this treatment is to blast the lupus once and wipe out the abnormal
immune system." Other researchers are
using the high-dose cyclophosphamide treatment alongside bone marrow or stem
cell transplants because the drug can be toxic to the immune system. But the Johns Hopkins team
said this was not necessary. Robert
Brodsky, associate professor of oncology and medicine, said: "Stem cells,
the bone marrow cells that give rise to all immune cells, are resistant to
high-dose cyclophosphamide. "The
malfunctioning immune cells are destroyed by the cyclophosphamide, while the
stem cells withstand the therapy and continue to rebuild a new, hopefully
disease-free immune system." Larger scale trials of the
treatment are now planned. A spokeswoman for Lupus UK
told BBC News Online: "Cyclophosphomide has been used to treat lupus
patients for many years and is therefore not a breakthrough." But she said: "We are
aware that this drug has been helpful to lupus patients who have not responded
to other types of medication." The research is published
in the journal Arthritis and Rheumatism. |
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