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 Monday, 13 January, 2003, 00:00 GMT
Cancer drug offers lupus hope
Laboratory work
Research into lupus treatment is ongoing
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 idea with this treatment is to blast the lupus once and wipe out the abnormal immune system

Professor Michelle Petri, Johns Hopkins Lupus Center
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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