Chinese scientists demonstrated the effectiveness of CAR‑T therapy in autoimmune neurological diseases.
- Pilot trials of CAR-T therapy were conducted in China.
- All 16 patients demonstrated clinical improvement.
- The therapy was well tolerated, with minimal side effects.
- The results were published in The New England Journal of Medicine.
In a recent study published in the journal The New England Journal of Medicine, Chinese scientists presented the results of the first pilot clinical trial of CAR-T lymphocytes obtained directly in the patient's body (in vivo). The application of this technology was aimed at treating autoimmune neurological diseases, which are traditionally difficult to treat.
What is CAR-T therapy
CAR-T (Chimeric Antigen Receptor T-cell) is genetically modified T-lymphocytes capable of recognizing and destroying cells that carry a specific antigen. In traditional approaches, cells are modified ex vivo, that is, in the laboratory, and then reintroduced into the patient. In the study in question, cells were modified directly in the body, which potentially simplifies the process and reduces the risk of complications associated with transporting and culturing cells.
For the trial, 16 patients with various forms of autoimmune neurological diseases, including multiple sclerosis and myasthenia, were selected. All participants received a single infusion of CAR-T lymphocytes targeted against specific autoantigens involved in the pathogenesis of their diseases.
The results were unequivocally positive: all 16 patients showed both clinical improvement (reduction of symptoms, improvement of nervous system functions) and biological improvement (decrease in autoantibody levels and inflammation markers). At the same time, the side effects were mild and short-lived, most of them limited to mild fever and fatigue, indicating good tolerability of the therapy.
Researchers emphasize that such results open new prospects for the treatment of autoimmune diseases, where traditional immunosuppressive drugs often prove to be insufficiently effective or cause serious complications. The CAR-T approach, unlike broad immunosuppressants, can selectively suppress pathogenic immune cells while leaving overall immune protection almost intact.
However, scientists warn that the current data has been obtained from a small pilot study and needs to be confirmed in larger randomized clinical trials. There are plans to expand the patient cohort to include more diverse forms of autoimmune neurological diseases and to assess the duration of the observed effect.
In the context of the global development of biotechnology, this study highlights the growing influence of Asian scientific centers, particularly China, in the field of advanced immunotherapies. The results could be an important step towards creating personalized treatment methods capable of changing the approach to chronic autoimmune pathologies.
So far, the therapy remains experimental; however, its successes have already attracted the attention of international regulators and investors willing to support further development of the technology. If subsequent studies confirm its effectiveness and safety, CAR‑T could become a new line of treatment for patients for whom traditional medications are no longer effective.
Source: N+1
