What treatments are being studied for lupus?
Many drugs, both old and new, are now being studied in people with lupus. The drugs described below represent only the first generation of potential new lupus therapies. This is not a complete list of all the drugs currently in development for lupus, and they may not all end up as approved medications. However, the variety of scientific approaches reflects the growing interest in lupus at all levels of the biomedical and pharmaceutical industry.
The interest in lupus among the biomedical and pharmaceutical industry offers realistic hope for additional treatments for lupus in the not-too-distant future.
Before any drug can be approved for use as a medical treatment, it must undergo a series of research studies, called clinical trials, to determine if it is safe and effective. Clinical trials are done in several phases, and this period of testing can take as many as 15 years. Generally, as part of the clinical trial design, some of the volunteers receive a placebo (an inactive substance, sometimes referred to as a “sugar pill”). This is done to better understand the effects of the study medicine.
There will be no new drugs for lupus without clinical trial volunteers! While participation in research studies is not for everyone, we encourage people with lupus, as well as healthy individuals, to participate in clinical trials.
There is no guarantee that a drug being studied will provide any benefit, and of course there are always risks with all drugs, whether investigational or FDA-approved.
We have listed these investigational drugs in alphabetical order. Some have already been approved by the FDA for treating other conditions, and are now being studied in lupus.
Abatacept (Orencia®) targets the T cells, a type of white blood cell that plays an important role in the immune response. Levels of these T cells are higher than normal in lupus and other autoimmune disorders. Stopping the overproduction of these T cells may reduce disease activity in lupus. Orencia has been approved by the FDA to treat rheumatoid arthritis (RA). This is in the biologics class of drugs.
Anti-BAFF monoclonal antibody is designed, like belimumab (Benlysta®), to block signals that stimulate B cells. This is in the biologics class of drugs.
Anti-CD154 was designed to attach to a surface protein called CD40L that appears on T cells, which are involved in the immune response. This action interferes with the activity of certain white blood cells called lymphocytes. This is in the biologics class of drugs.
Anti-interferon-alpha antibody targets interferon-alpha (INF-α), a chemical the body makes to fight viruses. Too much IFN-α can lead to immune problems, and levels of INF-α are higher than normal in lupus and other autoimmune disorders. Stopping the overproduction of INF-α may reduce disease activity in lupus. This is in the biologics class of drugs.
Anti-TNF-alpha drugs are developed to interfere with the action of tumor-necrosis factor-alpha (TNF-α). TNF-α is a particular type of protein molecule, called a cytokine, that plays key roles in acute and chronic inflammation, anti-tumor responses, and infections. Enbrel®, Humira®, and Remicade® are anti-TNF-α therapies approved for RA Although these drugs have been found to cause drug-induced lupus in some people (reversible when the medicine is stopped), they may be effective in treating the arthritis that can occur in lupus. These are in the biologics class of drugs.
Atacicept (TACI-Ig) was designed to block signals from a protein called immunoglobulin (Ig) that stimulates B cells. This is in the biologics class of drugs.
Cyclosporine (cyclosporin A, Neoral®, Sandimmune®) was the first drug that allowed the body to regulate T cell activity without causing too much damage to the immune system. Cyclosporine is also used alone or with methotrexate to treat the symptoms of RA and to treat psoriasis (a skin disease) in people who have not been helped by other treatments. This is in the organ transplant anti-rejection class of drugs.
Eculizumab (anti-C5a) is an antibody developed to stop the overactivity of complement C5. Complement refers to a group of proteins that can become overactive or misdirected in a disease like lupus. This is in the biologics class of drugs.
Epratuzumab is an antibody designed to attach to a protein called CD22 that appears on B cells in order to control active lupus by interfering with B cell function. This is in the biologics class of drugs.
Leflunomide (Arava®), approved for the treatment of RA, also appears to help with the arthritis caused by lupus. It works by stopping the overproduction of immune cells, which leads to swelling, inflammation, stiffness, and joint pain. This is in the immunosuppressives (immune modulators) class of drugs.
Lequinimod appears to change how the body responds to inflammation, and has previously been studied for multiple sclerosis. This is in the immunosuppressives (immune modulators) class of drugs.
Lupuzor™ was developed to change the behavior of a specific type of T cell involved in lupus disease activity. This is in the biologics class of drugs.
Prograf® works by interfering with T cell function. This is in the organ transplant anti-rejection class of drugs.
Rituximab (Rituxan®, anti-CD20) targets a specific protein known as CD20 that appears on the surface of B cells. Rituxan attaches to CD20 and is believed to work with the body’s own immune system to attack and kill B cells. This is in the biologics class of drugs.
Tacrolimus (Protopic™) and pimecrolimus (Elidel™) are creams that have been shown to suppress the activity of the immune system in the skin, including the “butterfly” rash, subacute cutaneous lupus erythematosus, and possibly even discoid lupus skin lesions. These are in the topical immunomodulators class of drugs.
Thalidomide (Thalomid®) has been shown to greatly improve symptoms of cutaneous lupus (affecting the skin) in people who have not responded to other treatments. This is in the immunosuppressives (immune modulators) class of drugs.
Tocilizumab blocks the activity of a cytokine called interleukin-6. It is approved for the treatment of RA. This is in the biologics class of drugs.
Triptorelin, a type of hormonal therapy developed to treat advanced prostate cancer, is being studied for the protection of the ovaries in women who are receiving cyclophosphamide therapy for lupus. This is in the hormones class of drugs.
Sirolimus (rapamycin, Rapamune®), used in combination with other medications to prevent rejection of kidney transplants, works by suppressing the body’s immune system. This is in the organ transplant anti-rejection class of drugs.
Stem cell transplantation is studied in people with severe, life-threatening autoimmune conditions who have not responded to any other treatments. Stem cells are the most basic type of cell, and are able to become different types of cells; they also have the ability to self-renew to produce more stem cells. The procedure works by eliminating the defective immune system and replacing it with a new, healthy system. Stem cell transplants use bone marrow cells from self (autologous), or stem cells from donors (allogenic). Both self and donor stem cell transplantation have been under investigation for many years in Europe and in the U.S.
The use of mesenchymal stromal cells (MSCs) in stem cell transplantation has also been studied in China and Europe. MSCs are unique adult stem cells with immunosuppressive properties and also may play a key role in preventing autoimmunity. When successful, disease symptoms become more easily controlled, and the need for medications reduced. However, some people may experience a recurrence of their symptoms over time, and there is a risk of death from the procedure.
Medically reviewed on August 08, 2013