Scientists are testing new ways to treat autoimmune diseases by reprogramming malfunctioning immune systems rather than suppressing them. The approach, which includes adapting CAR-T therapy autoimmune techniques used in cancer care, aims to help patients with rheumatoid arthritis, multiple sclerosis, lupus, and other chronic conditions. Researchers say the work is in early stages but may offer a path for longer-lasting control of the disease.
Autoimmune conditions develop when the immune system attacks healthy tissue. Standard treatments reduce inflammation but do not correct the underlying dysfunction. Many patients require lifelong drugs, infusions or injections, which can have serious side effects and do not always prevent flare-ups.
Dr. Maximilian Konig, a rheumatologist at Johns Hopkins University, said, “We’re entering a new era,” adding that CAR-T therapy autoimmune approaches could “control disease in a way we’ve never seen before.”
How CAR-T works
CAR-T therapy was originally designed for leukemias and lymphomas that arise from B cells, a type of immune cell. Some autoimmune diseases also involve harmful B cells; researchers are adapting CAR-T therapy autoimmune for these conditions. T cells from a patient’s blood, engineered in a lab to attack B cells, and then infused back after chemotherapy clears some existing immune cells.
Studies in Germany led by Dr. Georg Schett at the University of Erlangen-Nuremberg showed promising early results. A woman with severe lupus who received the therapy in 2021 has remained in remission without other medication. Schett’s team has since treated several dozen patients with lupus, myositis, and scleroderma, reporting few relapses.
Those findings helped spur a wave of clinical trials in the U.S. and abroad. Konig said the early outcomes were “shocking” and suggested a possible reset of the immune system. The therapy depletes both faulty and healthy B cells, allowing new cells to regenerate without harmful activity.
Patients who have undergone the treatment describe significant improvements. Mileydy Gonzalez, 35, of New York, received CAR-T therapy at NYU Langone Health after her lupus worsened and attacked her lungs and kidneys. “I’m going to trust you,” she recalled telling her doctor. Months later, she said she regained strength and no longer needed daily medications.
Exploring new cell-based tools
CAR-T therapy autoimmune is intensive, expensive, and customized for each patient, with cancer versions costing up to $500,000. Companies are now exploring off-the-shelf options using donor cells. Other researchers are adapting “regulatory T cells,” which naturally calm immune responses, by engineering them to counter harmful inflammation in diseases such as rheumatoid arthritis.
Scientists are also testing drugs called T cell engagers, which redirect existing immune cells to eliminate problematic B cells without personalized manufacturing. In one study, Dr. Ricardo Grieshaber-Bouyer in Erlangen gave the drug teclistamab to 10 patients with varied autoimmune diseases. All but one improved, and six entered remission without other treatment.
At Johns Hopkins, Konig’s lab is developing methods to target only the B cells that produce faulty antibodies. The goal, he said, is to remove “that very small population of rogue cells that really causes the damage” while leaving the rest of the immune system intact.
Other teams are using messenger RNA (mRNA) to send instructions directly to immune cells. Biomedical engineer Jordan Green at Johns Hopkins is testing nanoparticles that deliver mRNA to specific immune “generals,” prompting them to curb harmful T cells and expand protective ones. Studies in people may begin in several years.
Future prevention efforts
Researchers also hope to delay or prevent autoimmune diseases before symptoms begin. Dr. Kevin Deane at the University of Colorado Anschutz studies early markers of rheumatoid arthritis and tracks people who carry antibodies that indicate a higher risk. Similar work in Type 1 diabetes led to a drug, teplizumab, that can delay the onset of the disease by modifying the immune response.
Despite encouraging early results, scientists caution that long-term safety, cost and durability remain uncertain. CAR-T therapy autoimmune is furthest along but still carries risks.
For patients like Allie Rubin, 60, of Boca Raton, Florida, the gains have been life-changing. After decades of lupus, she qualified for CAR-T during lymphoma treatment. Nearly two years later, she said she remains free of both diseases.
Konig said the field is rapidly evolving. “I think the next 10 years will dramatically change our field forever,” he said.
Visit more of our news! The Lifesciences Magazine
