The first case in China! Tongji Hospital's CAR-T immunotherapy allows myasthenia gravis patients to become mothers

Jingchu.com (Hubei Daily Online) (Reporter Mao Yiyuan, Correspondent Tian Juan) Ms. Xie, a patient with relapsed and refractory myasthenia gravis, has achieved lasting improvement with the support of CAR-T therapy adopted by Professor Wang Wei's team from the Department of Neurology of Tongji Hospital Affiliated to Huazhong University of Science and Technology. During the 18-month follow-up, she has achieved a healthy and normal life without medication and successfully gave birth to a baby. This is the first time in the country that CAR-T cells (CT103A) targeting mature B cell antigen (BCMA) have been used to treat the disease. On February 26, the research paper was published online in the international authoritative academic journal "Journal of the European Molecular Biology Association".

Ms. Xie (pseudonym), 33, was diagnosed with myasthenia gravis in the Department of Neurology of Tongji Hospital four years ago. Despite trying various drug therapies, her condition has not been controlled. When the disease breaks out, she can't even do simple actions such as getting up and picking up things. At the worst, she was "unable" to breathe and had to be intubated and supported by a ventilator.

Myasthenia gravis is an autoimmune disease with acquired neuromuscular junction transmission disorder mediated by autoantibodies. At present, the treatment of myasthenia gravis is still mainly based on conventional immunotherapy such as cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulin, plasma exchange, etc. However, myasthenia gravis has a long course, is difficult to cure, and is prone to relapse. Although the current traditional drug treatment can improve myasthenia symptoms to a certain extent, it is insufficient in disease control and long-term safety.

On April 14, 2022, Professor Wang Wei's team conducted a detailed assessment of her condition and finally decided to use CAR-T therapy to treat myasthenia gravis. Chimeric antigen receptor (CAR) T cell therapy is an emerging treatment method that has been used to control the condition of a variety of blood system tumors for a long time. The therapy extracts the patient's own T lymphocytes, performs gene editing in vitro, and adds CAR targeting specific antigens as a guide to transform them into CAR-T cells with targeted killing effects. When the CAR-T cells are infused back into the patient's body, they expand on their own, act on target cells that secrete abnormal antibodies, and eliminate them, thereby alleviating the progression of the disease.

After the treatment, Ms. Xie stopped taking various immunotherapy treatments and successfully achieved a healthy and normal life without medication. In December 2023, she conceived naturally and gave birth to a healthy baby boy.

Wang Wei said that the refractory myasthenia gravis patients who were treated at the same time as Ms. Xie had also received multiple immunosuppressive treatments and did not achieve clinical remission. After receiving CAR-T therapy, the body's limb strength and lung capacity were significantly improved, and clinical symptoms continued to improve. The study also found that after the CAR-T cells were reinfused, they expanded well in the patient's body and lasted for a short time. The myasthenia gravis-related autoimmune antibodies of the two patients decreased rapidly after reinfusion and remained at an extremely low level for a long time. At the same time, the B cell phenotype was reconstructed, the B cells that secreted abnormal antibodies disappeared, the normal B cells reappeared, and the state of the immune environment was improved.

Autoimmune diseases often have complex mechanisms, limited treatment options, are difficult to treat, and recur repeatedly, which can bring great physical and psychological burdens to patients and their families. Professor Wang Wei's successful breakthrough in using CAR-T cells to treat a variety of relapsed and refractory autoimmune diseases has brought new treatment options to such patients.