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- An estimated 1.5 million people in the United States have the autoimmune disorder 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 is promising, but more research is needed.
Five patients with lupus who were 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 is feasible, tolerable, and highly effective in SLE,” the study authors wrote.
“However, longer follow-ups in larger cohorts of patients will be necessary to confirm sustained absence 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, blood is taken from a person to collect T cells, a type of immune system cell. These cells work by traveling around the body to destroy cells that are defective, 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 put back into a person via an infusion.
In lupus, immune cells called B cells make autoantibodies that attack healthy tissues. In the new study, T cells were modified so that when they were re-infused into the patient’s body they targeted a protein called CD19, which is present on the B cells that were attacking healthy tissues.
The researchers reported that CAR T-cell therapy was highly effective at destroying the B cells that were previously attacking healthy tissues, with the B cells disappearing entirely on the second day following CAR T-cell therapy.
The participants in the study had previously not responded to a number of other immunosuppressive therapies.
However, with the CAR T-cell therapy, they experienced an improvement in a number of severe symptoms and were able to stop their lupus medications.
Even when B cells reappeared after treatment, the participants continued to be disease free with no lupus flares, the researchers reported. The researchers reported that the reappearing B cells were naive, meaning that they weren’t yet specific for an antigen (target) like the previous B cells were.
Dr. Chris Wincup, a consulting rheumatologist at King’s College Hospital London and a Clinical Research Fellow at University College London, says although further study on a larger cohort is needed, the results are significant.
“The fact that this treatment worked, firstly, is very interesting, as these were patients who have had pretty strong and conventional treatment already,” he told Healthline. “The fact that it did actually bring them into remission, is really quite something. So is the fact that they were able to get in complete remission within three months having been refractory to so many of the strong and conventional treatments we use.”
“It really does show 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, with 90% of them being women. System lupus erythematosus (SLE) is the most common form of lupus.
Lupus causes the immune system to attack tissues in the body, resulting in inflammation and damage to tissues in organs. The 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’s a huge unmet need for better treatments for people with lupus,” Dr. Sean O’Neill, an associate professor of rheumatology at Royal North Shore Hospital and the University of Sydney in Australia, told Healthline. “The current options when it comes to standard care include medications like prednisone, steroid medications that bring a lot of side effects, and people are typically on 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 those treatments, or with some mildly active disease and toxicity from their treatments,” O’Neill added.
The researchers said that further study is required with larger cohorts of participants and longer follow-up periods to ensure the efficacy and safety of CAR T-cell therapy for people with lupus.
The experts who spoke with Healthline say while the study represents an exciting new avenue of exploration, it will take time for something like CAR T-cell therapy to become a standard treatment for people with lupus.
Both noted that the expense of CAR T-cell therapy is likely to be significant, which may limit the pool of people for whom the treatment is appropriate.
“One of the tricky things about CAR T-cell therapy is it’s extremely expensive,” said Wincup. “So, to have that available widely when we have other cheaper drugs that may be effective in many cases means that this may only be used in the more severe patients who have not responded to some of the treatments we already have available.”
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