World first: Therapy with donor cells leads to remission of autoimmune diseases
World first: Therapy with donor cells leads to remission of autoimmune diseases
A woman and two men with serious autoimmune diseases have experienced remission after treatment with bio-enemy and crispr-modified immune cells. Data track category = "References"> 1 . These three people from China are the first people with autoimmune diseases treated with immune cells made from donor cells instead of removing cells from their own body. This progress is the first step towards mass production of such therapies.
A recipient, Mr. Gong, a 57-year-old man from Shanghai with systemic sclerosis, who affects the connective tissue and can lead to skin stiffening and organ damage, reports that three days after the therapy, he noticed how his skin could relax and that he could start moving again and open his mouth. Two weeks later he returned to his workplace. "I feel very good," he says more than a year after the treatment.
engineering immune cells, known as Chimeric antigen receptor (car) T cells, have in the treatment of blood cancer Great Hope shown-a handful of products are in the United States Lupus and Multiple sclerosis , in which deviating immune cells produce that attack the body's own tissue. However, the therapy is usually based on a person's own immune cells, which makes them expensive and time -consuming.
Therefore, researchers have started to develop car T therapies from donated immune cells. If this is successful, pharmaceutical companies could scale production, which probably means the Costs and production times are significantly reduced. Instead of producing treatment for one person, therapies could be produced for more than a hundred people from the cells of a donor, according to Lin Xin, an immunologist at Tsinghua University in Beijing. Car T cells from donor cells have already been used to treat cancer patients, but so far with limited success 2 .
autoimmune diseases
The study, which was directed by Xu Huji, a rheumatologist from Naval Medical University in Shanghai, is the first to report results for autoimmune diseases. These were published in the magazine Cell last month. More than six months after the treatment, the recipients remained in remission. According to XU, another two dozen people have now received donor cell treatment and a slightly modified product. The results were mostly positive, he says.
"The clinical results are phenomenal," says Lin, who leads a separate study with car T cells from donor cells for the treatment of lupus.
The success and safety of therapy look promising, but have to be proven in many other people before the researchers can use conclusions about their broad application, says Christina Bergmann, a rheumatologist at the University Hospital in Germany.
However, it should be successful in more people over a longer period of time, it could be "paradigm alternating", said Daniel Baker, an immunologist at the University of Pennsylvania in Philadelphia. Over 80 autoimmune diseases are related to faulty immune cells.healthy donor
Car-t cell therapy usually includes the removal of immune cells, known as T cells, from the treated person. These cells are enriched with car proteins, attack the B cells, and then infused into the body of the person.
The process for the production of car T cells from donated immune cells is similar. XU and his colleagues take T cells from a 21-year-old woman and provide them with Cars, the CD19, a receptor that is found on the surface of B cells. They used the CRISPR-CAS9 genetic engineering tool to switch off five genes in the T cells in order to prevent the transplanted cells from attacking the landlord's body and avoiding the immune system of the host.
The first person who received the treatment was a 42-year-old woman in May 2023 with a kind of autoimmunmyopathy who attacks the skeletal muscle tissue and leads to weakness and fatigue. Mr. Gong and another 45-year-old man had an aggressive form of sclerosis. Her treatments started in June and August 2023.
once injected into the host, the car T cells began to work. They reproduced each other and aimed at all B cells-including pathogenic cells that are linked to the autoimmune diseases. The bio-engineered T cells survived in the recipients for weeks before they mostly disappeared. After all, new healthy B cells returned, while no pathogenic cells remained. A similar reaction was made in people with Autoimmune Diseases observed that received car T cells that come from their own cells 3 .
"complete remission"
Two months after the treatment, the researchers report that the woman achieved complete remission and maintained this status after six months in her follow -up examination. Baker notes that although the woman showed clear clinical improvements, he would be more careful to call this as a complete remission because the valuation time frame was short. The woman's autoantibodies had fallen on unexplained values, and their muscle strength and mobility had improved dramatically.
The two men also recorded significant improvements in their symptoms - including the regression of scar tissue - and a decline in the autoantibodies.
None of the people experienced an extreme inflammatory reaction, which is known as cytokine release syndrome and was observed in some cancer patients, received car-t therapy, and there were no evidence that the graft attacked the host. However, the researchers continue to try to find out whether the landlord repels the graft over time.
A central security concerns that were found in some people who received car-t cell therapies for the treatment of cancer is the appearance of new tumors , although the researchers continue to investigate whether they are related to the therapy. Baker emphasizes that it is too early to know whether people with autoimmune diseases treated with donor-derived car T cells are exposed to this risk. "Only time will show."
The central question is, according to Baker, whether the same approach works for more people and how durable the effects will be. "Will these patients remain free of symptoms for years?"
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Wang, X. et al. Cell 187, 4890–4904 (2024).
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Chiesa, R. et al. N. Engl. J. Med. 389, 899–910 (2023).
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Müller, F. et al. N. Engl. J. med. 390, 687–700 (2024).