New hope for lupus patients

First drug in 40 years specific to the disease is tested for approval

February 13, 2007|By Tricia Bishop | Tricia Bishop,Sun reporter

Doctors first gave Karen Banfield Evans a year of chemotherapy, even though she doesn't have cancer. Then, they switched her to a drug that prevents organ rejection, though she has never had a transplant.

Add into the mix several blood pressure reducers, a steroid, various nutritional supplements along with a multivitamin, and the count of pills Evans takes per day comes up to 10 - none of which was designed to treat her disease.

Evans has lupus, an often debilitating illness in which the immune system attacks the body, destroying organs, tissue, joints and quality of life. Five million people worldwide, including 1.5 million Americans, suffer in varying ways, yet it has been at least 40 years since a drug was created and approved to treat it. Doctors and patients have had to experiment with therapies meant for other ailments, some of which have severe side effects, and insurance companies typically won't cover such "off-label" uses.

Today, however, Human Genome Sciences Inc. plans to announce that it has given its experimental lupus treatment, LymphoStat-B, to the first patient as part of a late-stage clinical trial that it must pass to get U.S. Food and Drug Administration approval.

Human Genome is one of more than two dozen drugmakers - many from the biotechnology sector - that are using new science and data to develop treatments designed for lupus that could also treat other autoimmune diseases such as rheumatoid arthritis.

It's a big deal for the lupus community as well as the 14-year-old HGS, of Rockville, which has yet to bring a drug to market.

"Our fervent hope is that one or more or all of these companies will prove their drugs to be effective, because this is a situation that is intolerable," said Sandra C. Raymond, chief executive of the Lupus Foundation of America, which is based in Washington.

"People are angry. They feel they've waited too long [for a treatment] and, frankly, I don't blame them."

More than 90 percent of those afflicted with lupus are women, and most are minorities - two groups that have been historically underrepresented in health care research, medical professionals said. But that has begun to change as people of color collectively move toward a majority and women have become a focus in medicine.

The disease can affect nearly any organ in the body, making diagnosis extremely difficult. Physicians mistake symptoms for the underlying problem and put out fires as they arise.

Lupus patients generally live with the disease, which typically comes on between the ages of 15 and 45, for an average of four years before getting an accurate diagnosis, according to Raymond's group. And diagnosis usually only comes after the patient visits at least three doctors.

For those reasons, the patient population has traditionally been significantly underestimated, making it difficult for drugmakers to identify the demand, lupus specialists said. But new understanding of the disease and breakthroughs in treating other autoimmune disorders have led to new numbers and more interest in developing drugs.

"In the last several years, we have had a significant increase in the number of pharmaceutical and biotech companies entering the field, and I think that's because they think there's a market there," said Raymond, whose foundation accepts donations from many of the companies developing treatments, including Human Genome Sciences.

"That's why companies invest: They're not only interested in helping patients, they need to [make money]."

Three treatments are currently FDA-approved for lupus, though none was specifically developed for the disease.

In mild cases, aspirin is approved and used to dampen pain and low-grade fever. The steroid prednisone is often prescribed to control inflammation, though it also can cause weight gain, heart disease and avascular necrosis, a condition that kills bone tissue. And Plaquenil, an anti-malaria drug, has been used to ease skin lesions and reduce immune system performance, which lessens attacks but also reduces the body's ability to fight off outside infections.

Using chemotherapy over the past couple of decades to treat severe forms of lupus, such as those that lead to kidney transplants, has been considered a giant step forward, generally able to extend a patient's life by decades. But it comes with a price. Chemotherapy kills the lupus antibodies that attack a patient's body, but it also kills healthy tissue. In the short term, side effects include nausea and hair loss. In the long term, chemotherapy can eventually lead to heart disease, osteoporosis and diabetes.

That's why many are putting their hopes into the drugs being developed by the biotechnology sector using manufactured "monoclonal antibodies," which are thought to be able to focus on specific sites in the body.

"[Such] biologics just target parts of the immune system, they won't go around killing normal tissues like ovaries," said Dr. Michelle Petri, a professor of medicine and director of the Johns Hopkins Lupus Center.

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