Rays of Fortitude:  Awareness, Education & Support of Lupus - "Strengthening The Mind To Endure With Courage"
The Alliance for Lupus Research (ALR)
 
Lupus Research Update: 2013 Volume 1
 
Making the Connection Between Cardiovascular Disease and Lupus
 
Developing from a single cell, the human body becomes a wondrous and complex organism comprised of various systems that drive specific functions. In lupus, one of these systems — the immune system — malfunctions and can cause damage to the heart and other major organs.
With her ALR grant, Barbara Vilen, PhD, Associate Professor at the University of North Carolina, Chapel Hill, is directing a novel investigation that is looking at connections between lupus and cardiovascular disease — specifically atherosclerosis, or hardening of the arteries.
Why look at cardiovascular disease? Statistics reveal that the incidence of the disease is increased in lupus patients by 5-6 fold compared to the general population. Dr. Vilen explains the challenge: "While the numbers suggest that the immune system plays a role in atherosclerosis, the molecular basis underlying the immune trigger remains unknown."
Corroborations of the connection between lupus and cardiovascular disease came by Dr. Vilen's end-stage renal failure investigation in murine models. "Lights went on and we thought there is a connection between the immune complexes inducing BAFF (B-cell activating factor), BAFF driving kidney disease and BAFF driving lipid formation."
In her work, Dr. Vilen has observed an important clue: Immune complexes — which are formed from the integral binding of auto antibody to apoptotic debris — accumulated on the surface of immune cells.
Another piece of the puzzle is that cells undergoing apoptosis — the process of cell death naturally occurring in the body — generate internal debris that can also trigger the immune system in people with lupus. "Normally the body is highly efficient in removing these cells," said Dr. Vilen. "But in lupus patients, the system is broken."
Dr. Vilen suggests that part of the defect affects lipid biosynthesis, which is essential for the body's internal environment to remain stable. "Defects in lipid biosynthesis contribute to a variety of diseases... lead to the increased production of lipids, fatty cells, and foam cell formation — where immune cells absorb large amounts of fatty substance. These factors greatly contribute to atherosclerosis," said Dr. Vilen.
Thus, a person with lupus could be getting massive amounts of lipid biosynthesis from several different pathways.
In this complex framework, Dr. Vilen is looking to define if heightened BAFF dysregulates the production of lipocytes and fat cells, while promoting atherosclerosis. BAFF is a cytokine, which is known to be important to B-cell survival — and the overexpression of B-cells leads to lupus manifestations.
While many questions remain unanswered, Dr. Vilen's work is opening up new pathways for anti-BAFF treatment.
Dr. Vilen is vocal in her appreciation of the ALR for allowing her work to move forward: "The ALR enables scientists like me to approach lupus in different ways. This is an especially tough time for scientific funding, so I am all the more grateful to the ALR and its donors for making these types of investigations possible."
 
 
 
 
 

About Lupus
Lupus is an unpredictable and potentially fatal autoimmune disease in which the immune system is out of balance, causing inflammation and tissue damage to any organ system in the body.  The health effects of lupus include heart attacks, strokes, seizures, and organ failure.  An estimated 1.5 million Americans and at least five million people worldwide have a form of lupus.  For more information, visit www.lupus.org.
Lupus Foundation of America Applauds FDA's Decision to Approve Benlysta®
First new treatment for lupus in more than 52 years
Watch a message from Sandra C. Raymond, LFA President and CEO.

Watch Gary S. Gilkeson, MD, Chair of LFA Medical-Scientific Advisory Council, and Joan T. Merril, MD, LFA Medical Director, speak about what a new treatment may mean for the future of lupus patients.

Watch Kelly Jean Drury speak about what a new treatment means to her as someone with lupus.
(Washington, DC, March 9, 2011) Today, the U.S. Food and Drug Administration (FDA) approved the drug, BENLYSTA®, for the treatment of lupus, an autoimmune disease.

Sandra C. Raymond, President and Chief Executive Officer of the Lupus Foundation of America (LFA), has issued the following statement regarding the FDA’s decision:

“This is a historic day for the millions of people with lupus and their families around the world who have waited more than 52 years for a treatment breakthrough for lupus. We at the LFA applaud the FDA’s decision to approve BENLYSTA®. BENLYSTA is the first drug ever to be specifically developed to treat lupus, and is a significant first step toward reaching our goal of developing an arsenal of new, safe, effective, and tolerable treatments. Today marks the beginning of a new era of improved diagnosis, prevention, and treatment for the disease.

“The LFA wishes to thank the physicians, researchers, industry leaders, and the many study volunteers who made this day possible. We also extend a special thank you to BENLYSTA®’s developers, the staff of Human Genome Sciences and GlaxoSmithKline, who have long been committed to the research and development process. These efforts will go a long way in elevating the profile of this disease that remains a significant national public health problem.

“There are a number of pioneering biotechnology and pharmaceutical companies, involved in the research and development of new treatments for lupus, and our hope is that today’s decision will further stimulate additional companies to invest in new therapies for lupus. To build on this momentum and encourage the development of new treatments, the LFA has launched new initiatives that help to strengthen clinical trials. These programs include the launch of a Web-based program designed to train clinical investigators on the instruments used in trials. As well, the LFA recently implemented the LFA Lupus Research Registry which enables individuals to be notified about new clinical trials in their geographic area. The Registry is part of the LFA’s Center for Clinical Trials Education.

“The LFA also is partnering with key stakeholders from industry, government, and the scientific community to evaluate data from previous lupus clinical trials with the goal to improve the design of future studies."
Developing from a single cell, the human body becomes a wondrous and complex organism comprised of various systems that drive specific functions. In lupus, one of these systems — the immune system — malfunctions and can cause damage to the heart and other major organs.
With her ALR grant, Barbara Vilen, PhD, Associate Professor at the University of North Carolina, Chapel Hill, is directing a novel investigation that is looking at connections between lupus and cardiovascular disease — specifically atherosclerosis, or hardening of the arteries.
Why look at cardiovascular disease? Statistics reveal that the incidence of the disease is increased in lupus patients by 5-6 fold compared to the general population. Dr. Vilen explains the challenge: "While the numbers suggest that the immune system plays a role in atherosclerosis, the molecular basis underlying the immune trigger remains unknown."
Corroborations of the connection between lupus and cardiovascular disease came by Dr. Vilen's end-stage renal failure investigation in murine models. "Lights went on and we thought there is a connection between the immune complexes inducing BAFF (B-cell activating factor), BAFF driving kidney disease and BAFF driving lipid formation."
In her work, Dr. Vilen has observed an important clue: Immune complexes — which are formed from the integral binding of auto antibody to apoptotic debris — accumulated on the surface of immune cells.
Another piece of the puzzle is that cells undergoing apoptosis — the process of cell death naturally occurring in the body — generate internal debris that can also trigger the immune system in people with lupus. "Normally the body is highly efficient in removing these cells," said Dr. Vilen. "But in lupus patients, the system is broken."
Dr. Vilen suggests that part of the defect affects lipid biosynthesis, which is essential for the body's internal environment to remain stable. "Defects in lipid biosynthesis contribute to a variety of diseases... lead to the increased production of lipids, fatty cells, and foam cell formation — where immune cells absorb large amounts of fatty substance. These factors greatly contribute to atherosclerosis," said Dr. Vilen.
Thus, a person with lupus could be getting massive amounts of lipid biosynthesis from several different pathways.
In this complex framework, Dr. Vilen is looking to define if heightened BAFF dysregulates the production of lipocytes and fat cells, while promoting atherosclerosis. BAFF is a cytokine, which is known to be important to B-cell survival — and the overexpression of B-cells leads to lupus manifestations.
While many questions remain unanswered, Dr. Vilen's work is opening up new pathways for anti-BAFF treatment.
Dr. Vilen is vocal in her appreciation of the ALR for allowing her work to move forward: "The ALR enables scientists like me to approach lupus in different ways. This is an especially tough time for scientific funding, so I am all the more grateful to the ALR and its donors for making these types of investigations possible."
 
LFA answers to Frequently Asked Questions about BENLYSTA®
Updated: March 09, 2011
1. What is BENLYSTA?
BENLYSTA is a human monoclonal antibody that was approved for the treatment of lupus by the U.S. Food and Drug Administration (FDA) on March 9, 2011. A monoclonal antibody is a type of protein made in the laboratory that is developed to find and attach to only one type of substance in the body.

2. How does BENLYSTA work?
BENLYSTA is a human monoclonal antibody that specifically recognizes and blocks the biological activity of B-lymphocyte stimulator, or BLyS® (pronounced bliss), a naturally occurring protein which was discovered by scientists at Human Genome Sciences (HGS). Elevated levels of BLyS prolong the survival of B cells which can contribute to the production of autoantibodies – antibodies that target the body’s own tissues. Studies have shown that BENLYSTA can reduce autoantibody levels and help control autoimmune disease activity.

3. Who developed BENLYSTA?
BENLYSTA was co-developed by Human Genome Sciences (HGS) and GlaxoSmithKline (GSK).

4. What clinical research has been conducted on BENLYSTA?
HGS conducted two large scale Phase III clinical trials, BLISS-52 and BLISS 76. Both trials had positive top-line results and met their primary endpoints. The design of the two trials was similar, but the durations of therapy in the two trials were different, 52 weeks for BLISS-52, and 76 weeks for BLISS-76. The BLISS-52 study was conducted primarily in Asia, South America, and Eastern Europe, and BLISS-76 was conducted mostly in North America and Europe.

5. What does an FDA approval of BENLYSTA mean for people with lupus?
BENLYSTA represents a breakthrough in the treatment of lupus. BENLYSTA is the first drug approved to treat lupus in more than 50 years AND is the first drug developed specifically for lupus since the disease was discovered more than a century ago! Successful treatment of lupus will require an arsenal of safe, effective, and tolerable treatments. The approval of BENLYSTA is a significant first step toward reaching that goal.

6. What makes BENLYSTA different from other lupus treatments?
BENLYSTA is the FIRST FDA-approved medication specifically designed for the treatment of lupus. BENLYSTA targets specific immune cells, rather than the blanket approach of other therapies which suppress the entire immune system. Currently approved medications for lupus are borrowed from other diseases and conditions; other treatments are used off-label, which means they were never approved by the FDA for lupus. Many of these treatments have serious and devastating side effects.

These drugs include high doses of steroids, antimalarial medications, immunosupressive drugs, and organ-rejection drugs.

7. When will BENLYSTA be available for patients?
According to Human Genome Sciences, BENLYSTA will be available to physicians and patients before the end of March 2011.  It is best to talk to your doctor about the availability of BENLYSTA and if it is right for you.

8. Who should take BENLYSTA? Will it work for everyone?
Each person with lupus is unique, and BENLYSTA will not be an option for everyone.  You will need to discuss with your doctor if BENLYSTA may be an appropriate treatment option for you.

BENLYSTA is approved for the treatment of adult patients with active, autoantibody-positive systemic lupus erythematosus (SLE) who are receiving standard therapy.

The label for BENLYSTA includes the following limitations of use: The efficacy of BENLYSTA has not been evaluated in patients with severe active lupus nephritis or severe active central nervous system lupus, and has not been studied in combination with other biologics or intravenous cyclophosphamide. Use of BENLYSTA is therefore not recommended in these situations.

9.       Is BENLSYTA approved for use in children with lupus?
No. Additional studies are required before BENLYSTA can be approved for use in children.

10.   Will there be any further clinical trials on BENLYSTA?
Yes, additional trials are being planned. To learn more about clinical trials, visit lupus.org/clinicaltrials. On the Website you can also join our Lupus Research Registry to stay informed about lupus trials in your community.

11. What side effects have been found with BENLYSTA?
The most commonly reported adverse reactions with BENLYSTA were nausea, diarrhea, pyrexia, nasopharyngitis, bronchitis, insomnia, pain in extremity, depression, migraine, and pharyngitis.

12. How is BENLYSTA administered? 
BENLYSTA is administered through an IV (intravenous) infusion directly into the vein.

13. How much does BENLYSTA cost? 
We understand that there are going to be many questions, particularly around the affordability and accessibly of BENLYSTA, and the LFA will continue to address these important issues, and provide information as it is available.

14. Will there be any patient assistance programs available for BENLYSTA?
We believe that there will be a patient assistance program under the guidance and administration of HGS and GSK. We will continue to track the availability of assistance programs, and will update our website when more information becomes available.

15. Are there other treatments being researched for lupus?
There are a number of pioneering biotechnology and pharmaceutical companies involved in the research and development of new therapies for lupus, and there are several promising treatments in the near-term pipeline. The historic decision by the FDA will likely stimulate further investment in additional clinical trials in lupus. We can’t make new treatments a reality without your support. You can help by learning more about clinical trials and volunteering to participate in a clinical trial. To learn more, visit the LFA’s Center for Clinical Trial Education at www.lupus.org/clinicaltrials

16. Why has it taken so long to find a treatment for lupus?
Lupus is a complex disease. It can affect multiple organ systems and symptoms can range in severity from one day to the next. Also, lupus affects each person differently, with varying responses to treatment. The complexity and heterogeneity of the disease presents challenges in evaluating potential new therapies. With each research study, regardless of the outcome, there are new discoveries that help pave the way for new therapies.
17. What are the research barriers to developing new treatments for lupus?
The unique biology of this disease has made it difficult to assemble uniform patient groups to test new treatments, and the various background medications taken by patients have affected clinical trial results.  The pharmaceutical and biotechnology industries have lacked a clearly defined pathway for measuring outcomes in lupus clinical trials as required by the FDA. In addition, the instruments used by investigators to assess disease activity are also extremely complex.

18. What is the LFA doing to further the development of new treatments for lupus?
As part of the LFA’s National Research Program: Bringing Down the Barriers™, the LFA has recently launched new initiatives that will standardize and improve clinical trial design, allowing future studies to be completed successfully. These initiatives include a new worldwide Lupus Research Registry through the LFA’s Center for Clinical Trials Education, and a Web-based service to standardize research training on instruments used to assess disease activity in clinical studies and practice. The LFA also is partnering with key stakeholders from industry, government, and the scientific community to evaluate data from previous lupus clinical trials with the goal to improve the design of future studies. These initiatives, along with the approval of BENLYSTA, will help provide a pathway toward additional treatments specifically developed for lupus.
 
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