- An estimated 1.5 million people in the United States have the autoimmune disease lupus.
- There is no known cure and few effective treatments are available for the disease.
- Researchers report that a new CAR-T cell immunotherapy showed promise in a recent study.
- Experts said the study showed promise, but more research is needed.
Five patients with lupus treated with a form of immunotherapy known as CAR-T cell therapy reportedly achieved complete remission within months of their treatment.
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“These data suggest that CD19 CAR T cell transfer in SLE is feasible, tolerable and highly effective,” the study authors write.
“However, longer follow-up in larger cohorts of patients will be needed to confirm the persistent lack of autoimmunity and resolution of inflammation in patients with SLE who have received CAR T-cell therapy,” they added.
CAR-T cell therapy is a form of immunotherapy typically used in cancer treatment.
During CAR-T cell therapy, a person’s blood is drawn to collect T cells, a type of cell in the immune system. These cells work by migrating throughout the body to destroy defective cells, such as cancer cells.
In CAR-T cell therapy, some of these cells are taken and modified in the laboratory so that they can attack a new target. They are then given back to a person via an IV.
In lupus, immune cells called B cells make autoantibodies that attack healthy tissue. In the new study, T cells were modified so that when reinfused into the patient’s body, they would target a protein called CD19, which is present on the B cells that attack healthy tissue.
The researchers reported that CAR-T cell therapy was highly effective in destroying B cells that had previously attacked healthy tissue, with B cells disappearing completely on the second day after CAR T-cell therapy.
Study participants had previously failed to respond to a variety of other immunosuppressive therapies.
However, with CAR-T cell therapy, they experienced improvement in a number of severe symptoms and were able to stop their lupus medication.
Even when the B cells reappeared after treatment, the participants remained disease-free with no lupus flare-ups, the researchers reported. The researchers reported that the re-emerging B cells were naïve, meaning they were not yet specific for an antigen (target) like the previous B cells were.
dr Chris Wincup, a consultant rheumatologist at King’s College Hospital London and a clinical research fellow at University College London, says while further studies are needed on a larger cohort, the results are significant.
“The fact that this treatment worked, first of all, is very interesting because these were patients who were already on fairly strong and conventional treatment,” he told Healthline. “The fact that it actually put her into remission is really quite special. So is the fact that they were able to achieve a complete remission within three months after not responding to so many of the potent and conventional treatments we use.”
“It really raises the possibility that this could potentially be a new therapeutic option for patients with lupus with very severe and refractory disease,” Wincup added.
It is estimated that 1.5 million people in the United States have lupus, 90% of them women. Systemic lupus erythematosus (SLE) is the most common form of lupus.
Lupus causes the immune system to attack tissues in the body, leading to inflammation and tissue damage in organs. Blood vessels, joints, lungs, kidneys, brain and skin can all be affected by lupus.
There is no cure for lupus and experts say more treatment options are needed.
“There is a huge unmet need for better treatments for people with lupus,” said Dr. Sean O’Neill, Associate Professor of Rheumatology at the Royal North Shore Hospital and the University of Sydney in Australia, told Healthline. “Current options in standard of care include drugs like prednisone, which are steroid drugs that come with a lot of side effects and people usually take them for a very long time. So they get a lot of problems like osteoporosis and high blood sugar and cardiovascular disease related to their steroids.”
“While they can be very effective for some people, there are many, many patients with lupus with active disease despite these treatments, or with mildly active disease and toxicity from their treatments,” O’Neill added.
The researchers said more studies with larger cohorts of participants and longer follow-up periods are needed to ensure the effectiveness and safety of CAR T-cell therapy for people with lupus.
The experts who spoke to Healthline say that while the study represents an exciting new avenue of research, it will be some time before something like CAR T-cell therapy becomes a standard treatment for people with lupus.
Both noted that the cost of CAR-T cell therapy is likely to be significant, which could limit the population for whom the treatment is appropriate.
“One of the difficult things about CAR-T cell therapy is that it’s extremely expensive,” Wincup said. “So if we have that widely available, if we have other cheaper drugs that can be effective in many cases, that means they should only be used in more severe patients who have not responded to some of the treatments that are already available.”
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