Southern novelist Flannery O'Connor died at the age of 39 from complications of lupus, an inherited disease that had disabled her father and his father before him. Television journalist Charles Kuralt died from complications, as did British labor leader Hugh Gaitskell and Philippines President Ferdinand Marcos. Celebrities Anna Nicole Smith and Michael Jackson are thought to have suffered from it. R&B singer Seal, British model Sophie Howard, singer Elaine Paige, Oakland A's outfielder Tim Raines and "American Idol" finalist Leslie Hunt all suffer from it.
Lupus is a complex spectrum of autoimmune diseases that afflicts an estimated 1.5 million Americans and 5 million people worldwide, the vast majority of them women between age 15 and 45. It gets its name from the Latin word for wolf because, in 1851, a doctor observed a patient with red marks on her face that resembled wolf bites.
The disease is virtually a running joke on the Fox TV medical series "House," with doctors often speculating that a patient with mysterious ailments has lupus, only to find that it is something else. But that illustrates the great difficulty of diagnosing the disease, which is often called one of the "great imitators" because its symptoms often appear to be those of other diseases.
The news today that Human Genome Sciences has achieved success with an experimental drug called Benlysta has sparked tremendous excitement in the lupus community because no new treatments have been brought to market since the Eisenhower administration and because existing treatments have severe side effects. "People have been waiting an awfully long time for good news" like this, said Sandra Raymond, president of the Lupus Foundation of America.
Indeed, recent years have been marked by a wide variety of failures by well-known drug companies, including Roche and Biogen Idec (Rituxan), La Jolla Pharmaceutical (Riquent), BristolMyersSquibb (Orencia), Zymogenetics and Merck Serono (atacicept), Genelabs Technologies (Prestara), Teva Pharmaceutical Industries (edratide) and Aspreva Pharmaceuticals and Roche (CellCept). The outlook has been so bleak that market analysts have argued that HGS was throwing money down a rathole, and its stock fell from about $100 per share last year to $3.32 last Friday. Buoyed by today's news, the shares rose to $12.51.
The company reported today -- although the results have not been published yet -- that patients receiving the drug showed a statistically significant improvement in an index of symptoms that correlated with overall health and quality of life. Perhaps most important, according to Dr. Kenneth Kanunian of UC San Diego, the drug allowed many patients to reduce their use of steroids, which have debilitating side effects that can be nearly as overwhelming as the disease itself.
A second clinical trial is also under way, and if its results confirm today's findings, the Food and Drug Administration seems likely to approve Benlysta. In fact, HGS worked closely with the FDA to develop the index of symptoms that was used to measure the drug's effectiveness.
But Benlysta is not for everyone. Because it takes at least six months of injections for its effects to take hold, it is not useful for patients hospitalized with severe flareups of lupus. It is also not meant for people in the mild early stages of the disease, said Dr. Daniel Wallace of UCLA's David Geffen School of Medicine. It is targeted primarily at people who have an active form of the disease with perhaps one organ already involved. The goal is to prevent the disease from getting worse and to prevent other organs from being affected.
The biggest drawback is likely to be price. HGS has not said how much it will charge for Benlysta, but the drug is a monoclonal antibody, a protein that must be produced under exacting conditions. It must also be given once a month by infusion. Other monoclonal antibodies now in use for treating a variety of diseases cost thousands of dollars per dose, and Benlysta is unlikely to be any different.
-- Thomas H. Maugh II