Drug Fights Lupus-Linked Kidney Disease

WEDNESDAY, Nov. 23, 2005 (HealthDay News) -- An immunosuppressant drug normally used to prevent organ rejection shows promise in patients with lupus nephritis, otherwise known as lupus kidney disease.

The drug is mycophenolate mofetil (MMF), marketed as CellCept by drug maker Roche. If approved by the U.S. Food and Drug Administration, it would be the first drug approved for lupus in more than 30 years.

"This is very exciting," said Sandra C. Raymond, president and CEO of the Lupus Foundation of America. "It's been almost 40 years since the FDA approved a new therapy for lupus. Having a drug that provides a young individual with a better quality of life is a great thing."

The study appearing in the Nov. 24 issue of the New England Journal of Medicine expands on data first presented in October 2003 at the meeting of the American College of Rheumatology.

"After the presentation in 2003, there was an increase in the use of CellCept and more comfort on the part of physicians in using it, but I expect and hope that will become even greater with publication of this paper," said study lead author Dr. Ellen Ginzler, professor of medicine and chief of rheumatology at the State University of New York (SUNY) Downstate Medical Center in New York City.

Lupus is an autoimmune disease primarily affecting women between the ages of 15 and 44. The illness causes the immune system to attack the body's own tissue and organs, including the joints, kidneys, heart, lungs, brain, blood or skin. It may eventually cause tissue damage, organ failure, disability and even death.

An estimated 1.5 million Americans have some form of lupus. Black women are three times more likely to get the disease than white women. Hispanic and Asian women are also at higher risk.

Lupus nephritis is the most severe complication of the disease and, according to an accompanying editorial, affects 25 to 50 percent of patients with lupus.

Deaths from lupus surged 44 percent in the United States from 1998 to 2001, and hospitalizations soared 128 percent.

Currently, the standard of care for severe lupus is intravenous cyclophosphamide (Cytoxan), a chemotherapy drug which has debilitating side effects including hair loss, nausea, vomiting and infertility.

"It's the 20-something, 30-something young women whose hair is falling out and who are vomiting," Raymond said. "Every time they have another flare-up, they have to go back for another course. It's dreaded."

CellCept, which is FDA-approved for use in kidney, heart and liver transplant patients, has also been used off-label for lupus patients who failed Cytoxan, Ginzler said.

"In anecdotal studies, patients who were the sickest and least likely to respond to something were tried on CellCept and, surprisingly, maybe 80 to 90 percent of them had a response," Ginzler said.

In the current, 24-week trial (funded by the FDA and Roche), 140 patients were randomly assigned to receive CellCept or Cytoxan. More than half (56 percent) of the participants were black.

In the CellCept group, 22.5 percent experienced a complete remission versus 5.8 percent in the Cytoxan group. Partial remission occurred in 29.6 percent of the CellCept group and 24.6 percent of the Cytoxan group.

Three individuals died while taking Cytoxan. Another person died after declining Cytoxan therapy. Participants taking CellCept had fewer severe infections and hospitalizations, but more diarrhea.

CellCept is also easier to take as it comes in pill form.

More and more insurance plans are already covering CellCept for lupus, even though it is not yet approved for that indication, Ginzler said. And the FDA has asked for two confirmatory studies in order to consider the drug for approval.

Raymond feels there may also be potential for the drug to help people with general lupus.

As for lupus kidney disease, "I think CellCept could be considered a new standard of care," she said. She stressed that some patients may still only get better with Cytoxan, but CellCept "is a fabulous alternative."

More information

To learn more about lupus, visit the Lupus Foundation of America.