Shanghai, China — In a groundbreaking medical advancement, three Chinese patients suffering from severe autoimmune diseases have gone into remission after receiving treatment with bioengineered and CRISPR-modified immune cells derived from donors. This pioneering therapy marks the first successful use of donor-derived chimeric antigen receptor (CAR)-T cells to treat autoimmune disorders, potentially heralding a new era in mass-produced immunotherapies.
Among the recipients is Mr. Gong, a 57-year-old resident of Shanghai diagnosed with systemic sclerosis—a debilitating disease that damages connective tissues, leading to organ impairment and skin hardening. “Three days after starting the therapy, I could move my fingers and open my mouth again,” Mr. Gong shared. “I felt my skin relax.” Remarkably, he returned to his office job just two weeks post-treatment and reports feeling “very good” more than a year later.
CAR-T cell therapy has revolutionized treatment for certain blood cancers in recent years, with about half a dozen products approved in the United States. These therapies typically involve engineering a patient’s own T cells to target and eliminate malignant B cells. However, this personalized approach is both costly and time-consuming, limiting its accessibility.
The new approach, led by rheumatologist Dr. Xu Huji of Shanghai’s Naval Medical University, utilizes donor-derived CAR-T cells, potentially streamlining production and reducing costs. The results, published last month in the journal Cell, revealed that patients remained in remission for nearly six months following treatment. Dr. Xu reports that an additional two dozen patients have undergone the donor-derived therapy with similarly promising outcomes.
“The clinical outcomes are phenomenal,” noted Dr. Lin Xin, an immunologist at Tsinghua University conducting a separate trial using donor-derived CAR-T cells to treat lupus. “This could be a game-changer for patients with autoimmune diseases.”
The therapy works by engineering donor T cells with CAR proteins that specifically target B cells—the immune cells responsible for producing antibodies, including the autoantibodies that attack the body’s own tissues in autoimmune diseases. Upon injection, these modified T cells proliferate, seeking out and destroying all B cells, including the harmful ones. Over time, the bioengineered T cells diminish, allowing new, healthy B cells to regenerate without the pathogenic variants.
The potential to mass-produce a generic CAR-T product could enable pharmaceutical companies to scale up production significantly, reducing costs and production times. This advancement could meet the needs of a vast number of patients suffering from autoimmune conditions. However, questions remain about long-term efficacy and potential risks associated with donor-derived therapies.
As research continues, the success of these initial treatments offers hope for millions battling autoimmune diseases worldwide.
Reference(s):
First therapy using donor cells puts autoimmune disorders in remission
cgtn.com
