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|  | OCTOBER 21, 2004
By Catherine Arnst Old Drugs, New Impact For rare illnesses that drugmakers tend to ignore, patients' best hope may lie in scientists finding new uses for existing medications It isn't unusual in medical science for something old to be new again. Quite often, a drug that has been on the market for years for one disease turns out to be effective against something else altogether. This may become even more true as researchers discover that rogue cells or genes may be implicated in a number of different diseases, so a drug for one indication may well work for another. An interesting example of this is emerging in lupus, an incurable, sometimes fatal disease of the immune system. A team of Mayo Clinic researchers reported on Oct. 17 that Cellcept, a drug introduced nine years ago to prevent rejection of organ transplants, can effectively manage symptoms of the most common form of lupus, with far fewer side effects than other treatments. RARE TREATMENTS. The report is a pat on the back for a tiny company in Victoria, British Columbia, Aspreva Pharmaceuticals, founded specifically to find new uses for old drugs. A year ago, Aspreva convinced pharmaceutical giant Roche, which owns Cellcept, to allow it to develop the drug for use against lupus. Lupus has too few patients to make it worth Roche's while to develop the drug for just that disease. But if the effort is successful, Roche will throw its marketing might behind the drug. In an unusual twist for the pharmaceutical industry, however, Aspreva isn't trying to come up with billion-dollar drugs for common conditions such as high cholesterol or heartburn. Founded by doctors, the privately held outfit says its mission is to develop drugs for patients suffering from rare diseases "for which there's currently a lack of adequate treatment options and few, if any, treatments in development." Lupus certainly qualifies. At least 1.5 million Americans suffer from the disease, but no new treatments have been developed in some three decades. The most promising new candidate was a drug named Riquent, developed by La Jolla Pharmaceutical in San Diego to treat very sick lupus patients suffering from kidney damage. But on Oct. 14, the Food & Drug Administration ruled that Riquent may be "approvable" -- but only after the outfit conducts another clinical trial, which could take four years and millions of dollars that La Jolla, a startup, doesn't have. ORGAN DAMAGE. Here lies another another economic hurdle. Big Pharma isn't interested in lupus because it's a particularly hard disease to conquer -- or even diagnose. It has no known cause and was first identified in the 13th century (it was given the Latin name for wolf because a rash that often appears on the faces of victims can give a wolf-like appearance). Lupus is an autoimmune disease, so named because the immune system goes haywire and attacks the body's tissues and organs. The symptoms, such as extreme fatigue, achy joints, skin rashes, and anemia, can resemble those of other illnesses, so patients are often misdiagnosed. Lupus almost always strikes young adults, and is 10 to 15 times more common in women then men. It's also two to three times more common in African Americans, Hispanics, and Asians than in Caucasians. The most common type of lupus, affecting 70% of victims, is known as systemic lupus erythematosus (SLE). About half of these patients will suffer damage to a major organ, usually the kidneys. The main treatments are a highly toxic chemotherapy drug, cyclophosphamide, which can cause infertility and boost cancer risk, and steroids, which can weaken bones. FEW SIDE EFFECTS. Cellcept attracted the interest of Aspreva because it blocks the activity of certain powerful components of the immune system called T and B cells. "These are the bad actors in lupus," says Dr. Reinhard Baildon, head of clinical and regulatory affairs for Aspreva. "Cellcept interferes with their immune action and lowers the whole immune response." Just as important: It has few side effects, and 10 years of use has proven its long-term safety, an important issue for any medication taken for a chronic disease. The Mayo Clinic's Cellcept study, presented recently at the American College of Rheumatology annual meeting in San Antonio, found that 12 of 18 patients with early stages of SLE showed significant improvement over the six months of the study. Dr. Kevin Moder, a Mayo Clinic rheumatologist and one of the trial's investigators, says the drug "would be a nice addition to medications we can use for these patients, an alternative and widely applicable." Aspreva is now planning to set up two large clinical trials of Cellcept early next year. Baildon says the outfit is discussing the best way to proceed with the FDA. "The agency is very keen to work with us because lupus doesn't have adequate treatment, and it's very complicated to do a proper trial." BROADENING SEARCH. Researchers are also looking for other existing drugs that might work against the disease. One of the most prominent candidates is Rituxan, an antibody introduced by Genentech (DNA ) and Biogen Idec (BIIB ) in 1997 for the treatment of lymphoma, a deadly cancer of the lymph nodes. Rituxan works by blocking B cells, culprits in lymphoma. A team of doctors from the University of Rochester reported in August that just one injection of the drug eased symptoms of several patients for a year or more. Genentech is currently planning clinical trials to test Rituxan against lupus. For such a rare affliction, adapting such existing treatments may offer the best hope for sufferers. And as scientists learn more about the specific triggers behind other diseases, they're likely to come up with even more uses for drugs that target those triggers -- no matter what illness the medication was originally meant to treat.  Arnst is a senior writer for BusinessWeek in New York
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