New technologies are giving patients reason for hope, experts say
FRIDAY, Sept. 28 (HealthDay News) -- High-tech research into better, safer ways to treat the autoimmune disease lupus is building up steam, a group of scientists report.
Striking an upbeat tone, researchers from across the United States spoke at a teleconference Tuesday hosted by the Lupus Research Institute (LRI) of the improving outlook for patients with systemic lupus erythematosus.
"Today, we do have therapies that work," conference moderator Dr. Lee Simon, an associate clinical professor of medicine at Harvard Medical School in Boston, said. "So, while in the 1950s, 95 percent of people were dead within five years of diagnosis, today, 95 percent of patients survive five years out. But the treatments we have carry a heavy toxic burden. So, we need new drugs that work as well, if not better, with less harsh side effects."
"Today, I'm incredibly excited," added Simon, who has also served as a division director for the U.S. Food and Drug Administration. "We are now in a period of time when the development of molecular technology has given us the tools to dissect and tease out some aspects of the disease and identify biomarkers. That means that where previously we tended to treat the symptoms of patients, now we're trying to treat what actually drives the disease," he said.
Other researchers included in the teleconference hailed from the University of California, Los Angeles, the University of Chicago, Rush-Presbyterian-St. Luke's Medical Center (RPSLMC), and the Salk Institute of Biological Studies.
Lupus is a chronic and sometimes fatal autoimmune disease that affects upwards of 1.5 million Americans, according to LRI figures.
The disease usually strikes between the ages of 15 and 44, with 90 percent of patients being women. Hispanic, Asian and Native American women appear to have a higher risk of developing the disease, while blacks seem to face both a higher risk and a more severe prognosis once diagnosed.
The incurable and often-disabling condition strikes different patients in differing ways, making it difficult to screen and diagnosis. Lupus involves a hyperactive immune system that assaults otherwise healthy organs, such as the kidney, brain, heart, lungs and skin, as well as joints and blood.
According to Simon, patients typically experience fatigue and a general malaise, while further plagued by skin rashes and sensitivity to sunlight. Arthritis affects 90 percent of lupus patients, while 50 percent face serious kidney disease. Alongside respiratory complications, as many as one-third of patients develop cardiovascular complications.
The current standard of treatment typically involves nonsteroidal anti-inflammatory drugs (NSAIDs), anti-malarials and steroids.
Immunosuppressive medications -- such as azathioprine and cyclosporine -- are also used to dampen an immune system gone haywire. However, such regimens, while effective, can provoke severe side effects.
Conference attendees said that, until very recently, doctors and patients have been stuck in a treatment rut that has left them with few alternative prospects by way of treatment development.
"There hasn't been a new therapy approved for the treatment of lupus in over 40 years," Dr. Robert S. Katz, a rheumatologist with RPSLMC, said during the teleconference.
The problem, noted Simon, is that for many years, lupus was handled like an "orphan" disease, with the pharmaceutical industry largely ignoring it because of its relative rarity and the fear of not making a profitable return on drug-development costs.
This, despite the fact that more Americans are diagnosed with lupus than with cerebral palsy, multiple sclerosis, sickle-cell anemia, cystic fibrosis, or AIDS, LRI statistics indicate.
But the lupus research scene has undergone a renaissance, the experts said.
"It's a horse race," Katz said. "There are now 27 drug companies that are working on new lupus medicine. And the new therapies are more targeted therapies. They're not immune tranquilizers."
In fact, a new lupus information Web site -- established by the LRI this past May -- highlights 15 research trials that are currently under way. Many of the studies are for effective drugs with fewer side effects.
"I think one of the reasons that there is a measure of excitement in lupus and in other complex diseases that we've been confronted with for a long time is the advent of a whole series of new molecular tools that allow us to address basic issues," Salk molecular neurobiology professor Greg E. Lemke told reporters.
He pointed especially to the sequencing of the human genome and the use of genetically altered animal models as innovations that are boosting lupus research.
In one innovation, researchers led by Philip Low, of Purdue University in West Lafayette, Ind., say they've developed what could be a vaccine against lupus. Reporting in the Sept./Oct. issue of Molecular Pharmaceutics, the team said the shot reduced disease, cut down on kidney damage and extended the survival of laboratory mice with the illness.
This and other new research is raising expectations, the researchers said.
"Our goal should be trying to get remission in patients with lupus," UCLA's Dr. Bevra Hahn, chief of rheumatology in the School of Medicine, noted during the teleconference. "Patients all tend to accept what I call 'simmering' disease. There is fatigue, the patient never feels good, but they're not in danger, and we accept that as OK. I think it's time for both people who have the disease and physicians who treat them to do better."
Everyone agreed that heeding this advice will require an enormous public support --both in terms of continued financial investment and in enrolling patients in clinical trials.
The optimism is there, however.
"I'm a rheumatologist by training, and I've been seeing patients since 1978," Simon said. "And I'm much, much more encouraged than in the past about where we're heading. It's a very exciting time. The confluence of science, technology, and patient advocacy will allow all this to move forward."
For additional information on lupus research, visit the Lupus Research Institute.
SOURCES: Sept. 25, 2007, teleconference, Lupus Research Institute, with Lee Simon, M.D., associate clinical professor, medicine, Harvard Medical School, Boston; Bevra Hahn, M.D., chief of rheumatology, David Geffen School of Medicine, University of California, Los Angeles; Greg E. Lemke, M.D., professor, molecular neurobiology, Salk Institute of Biological Studies, La Jolla, Calif.; Robert S. Katz, M.D., rheumatologist, Rush-Presbyterian-St. Luke's Medical Center, Chicago; Sept. 26, 2007, news release, American Chemical Society
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