For the first time, universal CAR-T therapy has been used to treat autoimmune diseases, and a Chinese scientist has been named one of Nature’s Top Ten People of the Year.

On December 9, 2024, Nature announced its list of the top ten people of the year, which included two scientists from China: Xu Huji, the chief physician of the Department of Rheumatology and Immunology at the Second Affiliated Hospital of Naval Medical University (Shanghai Changzheng Hospital), and Li Chunlai, a researcher at the National Astronomical Observatories of the Chinese Academy of Sciences and the deputy chief designer of the Chang’e 6 mission.

The purpose of this list is to highlight ten individuals who have made significant contributions to major scientific events over the year. Nature praised Xu Huji as a doctor and researcher who bravely attempted a revolutionary treatment for autoimmune diseases.

According to previous reports by The Paper, on July 16, 2024, Xu Huji’s team published original clinical research results online in Cell, introducing a revolutionary CAR-T cell therapy for treating autoimmune diseases, marking the first international revelation of using allogeneic universal CAR-T cells to treat rheumatic and autoimmune diseases.

Rheumatic diseases refer to a large category of systemic autoimmune diseases, including common conditions such as rheumatoid arthritis, ankylosing spondylitis, and systemic lupus erythematosus. The first patients in Xu Huji’s study were diagnosed with necrotizing myopathy and systemic sclerosis, both of which are rheumatic autoimmune diseases.

Xu Huji’s research team utilized T cells derived from healthy donors, which were genetically engineered to create a universal CAR-T cell drug targeting CD19 on B lymphocytes (TyU19). After the first treatment of the three initial patients, Xu expressed his anxiety and sleeplessness, as these cases involved severely ill patients.

Two weeks after treatment, the first patient, a woman with immune-mediated necrotizing myopathy (IMNM), informed the nurse that she had regained the strength to lift her arms and comb her hair. The other two male patients, suffering from diffuse systemic sclerosis, also began to experience a reduction in symptoms. More than six months later, all three patients achieved long-term remission of their conditions. “We finally relaxed a bit,” Xu said.

Over the years, CAR-T cell therapy has shown tremendous potential in treating hematologic malignancies and autoimmune diseases. Since 2017, the U.S. FDA has approved six CAR-T cell therapies for the treatment of blood cancers such as leukemia and lymphoma. Recently, CAR-T cell therapy has also demonstrated great promise in treating autoimmune diseases like systemic lupus erythematosus and multiple sclerosis.

However, this therapy typically relies on the patient’s own immune cells, and all currently available CAR-T cell therapies use genetically engineered immune cells derived from the patients themselves, making the treatment extremely expensive and time-consuming. For instance, in 2023, a team in Germany reported that they had successfully treated at least 15 patients with various autoimmune diseases using CAR-T cell therapy.

In contrast, Xu Huji’s team used CAR-T cell therapy derived from donor cells of healthy individuals (rather than the patients’ own cells) to treat autoimmune disease patients. This universal CAR-T cell product represents a crucial first step toward the mass production of CAR-T cell therapies for autoimmune diseases, potentially significantly increasing the accessibility of CAR-T cell therapy.

In their paper, Xu Huji’s team reported on the treatment outcomes of the first three patients, who continued to show symptom improvement during a six-month follow-up after treatment. Clinical observations and laboratory tests confirmed that the universal CAR-T cell therapy targeting CD19 was safe.

In October 2024, Xu Huji revealed in an interview with The Paper that they had treated 24 patients with different types of allogeneic universal CAR-T cells, achieving the expected therapeutic effects. He stated that the research demonstrated the significant potential of allogeneic universal CAR-T cell therapy in terms of efficacy and safety, marking a new phase in the treatment of immune diseases. With further research and expansion of clinical trials, there is reason to expect that this therapy will bring benefits to more patients.