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After receiving treatment using bioengineered and CRISPR-modified immune cells, two men and one woman with severe autoimmune diseases have gone into remission. The three Chinese patients are the first to receive treatment for autoimmune illnesses using modified immune cells made from donor cells as opposed to autologous cells. This development marks the beginning of the mass manufacturing of these treatments.
Mr. Gong, a 57-year-old Shanghai resident, is one of the recipients. He suffers from systemic sclerosis, a disease that damages connective tissue and can cause organ damage and hardening of the skin. He claims that three days after starting the therapy, he could once again move his fingers and open his mouth and felt his skin relax. He went back to work at his office two weeks later. He states, more than a year after the treatment, "I feel very good."
Half a dozen products containing engineered immune cells, known as chimeric antigen receptor (CAR)-T cells, are approved in the U.S. These cells have shown great promise in treating blood cancers and may also be used to treat autoimmune diseases like multiple sclerosis and lupus, where rogue immune cells release autoantibodies that attack the body's own tissue. However, because the therapy is personalized and usually depends on an individual's immune cells, it is costly and time-consuming.
The experiment is the first to publish findings for autoimmune disorders, and it is being headed by Xu Huji, a rheumatologist at Shanghai's Naval Medical University. Last month, the results were published in Cell. The patients stayed in remission for almost six months following treatment. According to Xu, another two dozen people have had the donor-derived treatment along with a slightly altered product. He claims that most of the outcomes have been favorable.
"The clinical outcomes are phenomenal," says Lin Xin, an immunologist at Tsinghua University who is leading a separate trial using donor-derived CAR-T cells to treat lupus.
T cells, which are immune cells, are usually taken from the patient undergoing CAR-T-cell therapy. The recipient's body receives the cells back after they have been enhanced with CAR proteins that specifically target B cells.
The CAR-T cells began to function after being injected into the hosts. They proliferated, attacked and eliminated every B cell, including harmful cells connected to autoimmune diseases. The bioengineered T cells persisted for weeks in the recipients before mainly disappearing. New, healthy B cells eventually proliferated again, but no pathogenic ones did. People with autoimmune diseases who received CAR-T cells made from their own cells have shown a similar reaction.
According to Nature, a generic CAR-T product, if successful, would allow pharmaceutical companies to scale up production, potentially significantly reducing costs and production times in the future, and be able to meet the needs of large-scale patients. In the meantime, however, it remains to be seen whether patients with autoimmune diseases treated with donor-derived CAR-T cells will face other risks.