In a recent announcement, the U.S. Food & Drug Administration has approved the Investigational New Drug application for LymphoStat-B(TM), a potential new treatment for autoimmune diseases that is set to begin clinical trials.
The drug’s manufacturer, Human Genome Sciences will now proceed with a Phase 1 clinical trial in patients with systemic lupus erythematosus. The trial will be a multi-center, dose-escalation study to determine the safety and pharmacology of the drug in adult patients who are receiving standard therapies. In the future, LymphoStat-B may also be tested in patients with other autoimmune diseases, such as rheumatoid arthritis, immune thrombocytopenic purpura, and Sjogren’s syndrome
LymphoStat-B is a fully human monoclonal antibody that acts by binding to, and inactivating a natural immune stimulator, the B Lymphocyte Stimulator (BLyS(TM)). Many patients that suffer from systemic lupus erythematosus and rheumatoid arthritis have elevated levels of BLyS in their blood or joint fluid. Laboratory studies show that LymphoStat-B can reverse the immune stimulatory effects of BLyS.
“Studies in laboratory models of lupus, as well as in human lupus patients, suggest that elevated levels of BLyS may have a significant role in systemic lupus erythematosus,” said William Stohl, M.D., Ph.D., Professor of Medicine, Division of Rheumatology, University of Southern California, “Furthermore, laboratory studies of rheumatoid arthritis and studies in human rheumatoid arthritis patients raise the possibility that elevated BLyS levels may contribute to this disease as well.”
“LymphoStat-B, by virtue of its ability to neutralize BLyS activity, may prove to be an effective treatment approach for patients suffering from systemic lupus, rheumatoid arthritis and possibly other autoimmune diseases.”
Robert P. Kimberly, M.D., Professor of Medicine and Microbiology, and Director, Division of Clinical Immunology and Rheumatology, The University of Alabama at Birmingham, said, “Autoimmune diseases represent the third-greatest clinical burden after cardiovascular disease and cancer, and currently have no cure. These diseases are complex and often devastating chronic illnesses. Patients with systemic lupus, in particular, can experience any number of symptoms that can flare unexpectedly on average two to three times a year. For patients with severe symptoms, typically involving their internal organs, this disease can sometimes be fatal. There is great need for further research into the causes of these diseases, as well as for new treatment options that can help control disease progression and improve patients’ quality of life.”
David C. Stump, M.D., Senior Vice President, Drug Development, Human Genome Sciences, Inc., said, “LymphoStat-B has the potential to treat a family of serious autoimmune diseases, including systemic lupus erythematosus and rheumatoid arthritis. If the initial Phase 1 trials of the drug are successful, we hope to be able to add new indications for additional trials for patients with rheumatoid arthritis and other autoimmune diseases, such as immune thrombocytopenic purpura, and Sjogren’s syndrome. These studies should also shed much light on the role of hyperactive B-cell immunity, and of BLyS in particular, in the origin of a broad family of autoimmune diseases.”
William A. Haseltine, Ph.D., Chairman and Chief Executive Officer, Human Genome Sciences, Inc., said, “I am delighted that we can now begin trials of LymphoStat-B for the treatment of systemic lupus erythematosus and other autoimmune diseases. As a family, these diseases cause immense suffering to millions of patients worldwide. LymphoStat-B provides a new, fresh, rational, mechanism-based approach for the treatment of these diseases.”
Systemic lupus erythematosus is a serious, life-threatening disease. It can lead to arthritis, kidney failure, heart and lung inflammation, central nervous system abnormalities, inflammation of the blood vessels and blood disorders.Between 200,000 and 500,000 people are diagnosed with systemic lupus each year in the United States alone. The disease affects between eight and ten times as many women as men. It may occur at any age, but appears mostly in young people between the ages of fifteen and forty-five. Symptoms may include extreme fatigue, painful and swollen joints, unexplained fever and kidney problems.