Drug Targets Lupus by Tricking Immune System

A new approach shows early promise in fighting the devastating disease.

MIT Technology Review—June 21, 2010

Scientists developing drugs to treat lupus face a daunting set of challenges. The disease, which affects 1.5 million Americans, results when the immune system mistakenly recognizes healthy tissues as dangerous and attacks them, touching off a range of responses. Some patients get arthritis and rashes, others develop heart disease, and some suffer kidney damage that can endanger their lives. And it still isn't clear what causes all these symptoms.

Now two companies are working together to attack the disease with an experimental drug that tricks the immune system into behaving more normally. Results from phase II trials of the drug, called Lupuzor, showed a 53% improvement in symptoms among patients on the drug compared to a 36% improvement in those on placebo. That was enough to generate a tremendous amount of excitement leading up to the 9th International Congress on Systemic Lupus Erythematosus in Vancouver, British Columbia, which begins June 24.

Many drugs commonly used to treat autoimmune diseases, such as chemotherapy drugs, are known as immunosuppressants because they effectively shut down the entire immune system. Lupuzor, by contrast, is an immunomodulator--a drug that targets the specific immune cells involved in the disease. That's important because disabling the immune system entirely can cause unwanted side effects, such as dangerous infections.

Cephalon has yet to announce the timing of its phase III program or details about how it will measure patient response to Lupuzor in those trials. Still, the fact that there are any products at all in late-stage testing is a welcome relief to physicians like Kyriakos Kirou, M.D., a rheumatologist at the Mary Kirkland Center for Lupus Care at Hospital for Special Surgery in New York. "The drugs we have now are nonspecific and not very potent," Kirou says. "We need to strike the right balance in controlling the immune system, and hopefully these new medicines will do a better job of that. It's a very exciting time in lupus."

This article originally appeared at technologyreview.com.