Engineered immune cells, known as CAR T cells, have demonstrated potential beyond cancer treatment, showing promise in treating the autoimmune disease lupus. A recent study provides encouraging early results for CAR T-cell therapy in a small group of patients with lupus, suggesting a new avenue for treating this challenging condition.
CAR T-Cell Therapy for Lupus
The study, published in Nature Medicine, details the outcomes of five young adults with lupus who had not responded to conventional treatments. These patients received CAR T-cell therapy, which involves modifying a patient's own T cells to target and eliminate specific cells in the body. In the case of lupus, the CAR T-cells were designed to target B cells, which play a key role in the disease's pathology.
Remarkable Remission
The results of the study were remarkable. All five patients went into remission and were able to stop taking their lupus drugs within three months of receiving a single, relatively small dose of CAR T-cell therapy. Unlike cancer patients who often require ongoing antibody infusions, the lupus patients experienced a return of their B cells, naturally replenished from blood stem cells in bone marrow.
Implications and Future Research
According to researchers, lupus is a logical target for CAR T-cell therapy because it is driven by B cells. In lupus, these B cells attack the patient's own organs and tissues. The experimental CAR T-cell therapies employed existing anti-B-cell designs.
While the study's findings are promising, researchers emphasize the need for confirmation through larger studies and longer-term follow-up. However, early indications suggest that lupus may be a more straightforward CAR T-cell target than B-cell cancers.
"Disease-driving B cells are much less numerous in lupus," explained Daniel Baker, a doctoral student at the University of Pennsylvania. "Thus, effective CAR T treatment of this autoimmune disease may require a much lower dose that greatly reduces the problem of immunological side-effects."
CAR T-Cell Therapy Background
CAR T-cell therapy involves collecting a patient's T cells, genetically engineering them to target specific cells, and then re-infusing them back into the patient. The first CAR T-cell therapy, Kymriah, was developed at Penn Medicine and approved by the FDA in 2017. There are now six FDA-approved CAR T-cell therapies in the United States, used to treat various B-cell leukemias, lymphomas, and other blood cancers.
