New Year Rings In New Hope for People Affected by Lupus
By Dr. Susan Manzi, Lupus Foundation of America Medical Director and Dr. Karen Costenbader, Lupus Foundation of America Medical-Scientific Advisory Council Chair
Singer Selena Gomez brought lupus awareness to new heights in early 2017 when she shared her compelling story about having a kidney transplant after lupus had ravaged her kidneys. The sharing of her experiences helps give people a greater understanding about the life-threatening impact of lupus and boosts public demands for greater funding to support critical lupus research studies.
As we reflect back on the past year, one thing is clear – there has been significant progress, not only in awareness but also in lupus research and advocacy efforts to secure additional research funding for lupus. We are encouraged by the advances of 2017 and look forward to reporting on more successes during 2018 and beyond, with great hope of a better quality of life for all people affected by lupus!
Great Progress in the Development of an Arsenal of New Treatments
- Results of several clinical trials provided good reasons for people with lupus to remain hopeful that more effective and tolerable treatments are right around the corner.
- Voclosporin - A phase IIb clinical study of voclosporin for treating lupus nephritis (lupus affecting the kidneys) showed nearly half of the participants who received a low dose of voclosporin, along with standard of care, achieved complete remission after 48 weeks of treatment. The research is continuing and a phase III trial, Aurora, began in late May of 2017.
- Anifrolumab - A phase II trial of anifrolumab demonstrated that it significantly reduced disease activity and improved symptoms, such as rash, and joint pain and swelling among those with moderate to severe lupus. Two phase III studies of anifrolumab for moderate to severe lupus have begun and are expected to wrap up in the second half of 2018.
- Ustekinumab - A phase II trial of ustekinumab (UST) found that a greater portion of people who received UST showed improvements in disease activity, compared to those who received a placebo. UST is already approved for use in treating other diseases under the brand name Stelara®. A Phase III trial of UST for use in lupus is planned to begin later this year.
- Atacicept - A phase II trial of atacicept showed that it reduced overall disease activity and severe flares, particularly among people with high levels of lupus disease activity. . A Phase III trial is being planned.
- CC-220 - A phase II trial of a compound named CC-220 demonstrated positive results among people with lupus who had skin involvement, while decreasing overall lupus activity.
New Self-Administered Formulation of Benlysta® - The U.S. Food and Drug Administration (FDA) approved a new formulation of Benlysta® (belimumab) to treat subcutaneous (SC) lupus (lupus that affects the skin). This new formulation provides a less invasive method for administering Benlysta, allowing people with lupus to administer the biologic themselves at home as a once-per-week self-injection, rather than traveling to a medical facility for monthly infusions.
New Technique May Help Assess Skin and Kidney Damage - A large, multicenter National Institutes of Health- and industry-sponsored research project called “Accelerating Medicines Partnership” (AMP) has produced remarkable preliminary results showing that a new technique, called single cell RNA sequencing, using tissue from kidney and skin biopsies, may provide new insights into what causes lupus nephritis and how it develops, and perhaps providing a better way assess damage in skin and kidney tissue.
Gut bacteria and lupus disease activity - Researchers discovered that people with high lupus disease activity had more of a certain bacterium in their guts than those with low disease activity. These bacteria can alter the immune response suggesting that the gut may play an important role in developing lupus and in tracking lupus flares.
Efforts to Identify People at Risk for Lupus and Prevent Damage - Researchers have found a strong link between trauma and posttraumatic stress disorder (PTSD) and the onset of lupus. With this information, doctors may be able to better identify who might be at risk for lupus and prevent flares by reducing stress.
Stem Cell Therapy Trial Moves Forward - In December, the Lupus Foundation of America announced it will provide $3.8 million in funding to support the first clinical trial in the United States to evaluate mesenchymal stem cells (MSC) as a treatment for lupus. The study will evaluate whether this stem cell therapy can decrease lupus disease activity, reduce the need for medications like steroids, which have harmful side effects, and prevent further damage to vital organs.
Congressional Appropriations for Lupus Research and Education Top $13 Million – During 2017, together with our national network of lupus advocates, we stimulated funding for lupus research and education programs within the US Centers for Disease Control and Prevention, the Office on Minority Health and the Department of Defense (DoD). The DoD funding led to creating a dedicated lupus research program. This new program provides lupus investigators an additional funding source to support their important studies. Additionally, the Foundation successfully advocated Congress to provide $5 million for this new program in 2018 that will focus on why the impact of lupus is so different in each person living with the disease.
Giving People with Lupus a Stronger Voice
- National Public Policy Summit on Lupus – We brought Congress face-to-face with lupus when more than 200 lupus advocates from across the nation went to Capitol Hill to advocate for more funding for lupus research and education programs and for better access to healthcare. People with lupus and their families educated members of Congress on the physical, economic and emotional burdens of lupus and urged lawmakers to support legislative priorities important to the lupus community.
- Patient-Focused Drug Development Meeting - Top decision makers at the FDA heard from more than 550 people impacted by lupus during the FDA’s externally-led Patient-Focused Drug Development (PFDD) Initiative. This ongoing FDA effort enables regulators to better understand the perspectives of people with diseases such as lupus so they can more thoroughly assess the risks and benefits of drugs under review. The meeting was organized by the Lupus Foundation of America, the Lupus and Allied Diseases Association and the Lupus Research Alliance.