Advances in the Lupus Treatment and Clinical Research Opportunities for Patients with Lupus
From the 2012 Lupus Education Series
Advances in the Treatment of SLE, Richard Furie, MD
Presenter Richard Furie, MD noted that like snowflakes, no two cases of lupus are alike, making it difficult to treat and research.
Lupus therapy should control disease activity, prevent damage, and prevent flares.
- The focus of treatment should be on active lupus because you can’t get rid of damage that has already been done.
- The least toxic dose should be used to treat the manifestation of greatest concern.
- Medications typically used to treat the various manifestations of lupus and the drugs toxicity include. He said the current feeling is that everyone who could be, should be, on Plaquenil (hydroxychloroquine). He gave an example of six disease manifestations that a patient may be suffering from and explained that while the patient may be most concerned about swollen fingers and joints, the physician will be most interested in treating the low platelet count (thrombocytopenia) and least interested in treating irreversible damage.
When conventional therapy fails there are some other options such as pulse steroids (1200 mg a day for 3 days by IV, usually for kidney disease), bone marrow transplant which is experimental and has not been that successful or safe, TNF inhibitors that are used to treat rheumatoid arthritis but can aggravate lupus, biologics such as belimumab, and off-label biologics such as rituximab or abatacept.
Drug Development and Clinical Trials
There is a need for more efficacious and safer therapies for lupus. However, the wide range of lupus manifestations makes trial design very difficult. Issues include what endpoints to measure and the use of background medications. There are different pathways in lupus that can be targeted for therapy. Dr. Furie pointed to the only drug approved just for lupus, belimumab (Benlysta®) as targeting one of these pathways, BLyS, which overstimulates B cells. By reducing BLyS in one’s system, the B cell is no longer stimulated to make antibodies.
He explained that Phase I studies are small, 50 to 60 patients, and designed to make sure the drug targets what it is supposed to and is safe. Phase II looks at effectiveness and compares patients taking the drug to those taking a placebo. And in Phase III, they continue to look at safety and effectiveness but also look at different subsets of patients.
In recent clinical trials for lupus there have been surprising failures, such as rituximab, and successful failures. The Food and Drug Administration requires a trial to show a drug is superior to an approved drug. Mycophenolate mofetil (MMF or cellcept) is an example of this. The trial failed to show that MMF was superior to cyclophosphamide (cytoxan) but it did show it was equivalent. As a result, the FDA did not approve MMF for lupus but because it is less toxic than cyclophosphamide it is often used.
There are currently 12-13 drugs being developed for lupus. Clinical trials have gone worldwide. A lot of patients will be needed to participate to determine the efficacy of these drugs.
Richard Furie, MD
Professional Title: Chief, Division of Rheumatology and Allergy-Clinical Immunology, North Shore LIJ Health System; Professor of Medicine, Hofstra North Shore-LIJ School of Medicine
Bio: Dr. Richard Furie, Chief of the Division of Rheumatology and Allergy-Clinical Immunology at the North Shore LIJ Health System, is a rheumatologist whose activities for the last several decades have focused on patient care, physician education, and clinical research in the area of anti-rheumatic drug development. He directs the Hospital’s SLE and Autoimmune Disease Treatment Center, internationally recognized for its role in developing new therapies for SLE. Dr. Furie is on the Medical and Scientific Advisory Board of the SLE Foundation as well as the Alliance for Lupus Research Scientific Advisory Board. He has served on many committees of the American College of Rheumatology, and has recently been appointed to the College’s Board of Directors.
Clinical Research Opportunities for Patients with SLE, Laura Donohue, CCRC
Presenter Laura Donohue, CCRC described what a clinical trial is, what a trial participant can expect, risks, and different types of trials.
- Safety of the patients is very important and patients are required to sign a consent form that explains known associated risks. She also explained that trials are very carefully monitored and patients can withdraw from a trial at any time. It usually does not cost anything to participate in a trial and travel expenses are often reimbursed.
- Different types of trials include interventional studies, blood draw studies, and observational trials.
- Interventional studies are drug studies and currently the Program in Novel Therapeutics at North Shore-LIJ is conducting studies in lupus nephritis, discoid lupus, and non-renal active lupus.
- Blood draw studies typically involve a patient giving blood once. The blood is then studied looking a genes and biomarkers.
- Observational studies entail watching how people respond to various treatments but there are no special treatments or procedures.
- She also emphasized that if your doctor is not affiliated with a study site, you can still participate in a study and the study site will communicate with your doctor. People with lupus who are interested in participating in a trial can contact Laura at (516) 708-2558 or firstname.lastname@example.org.
Laura Donohue, CCRC
Laura Donohue, CCRC is the senior research coordinator at the Division of Rheumatology and Allergy Clinical Immunology at the North Shore LIJ Health System. She has been working with Dr. Furie on research studies for over 11 years and has coordinated over 20 lupus trials.