CD19 CAR-T Therapy for SLE+APS+TBIRS (Rare Disease), "Killing Three Birds with One Stone"

——Self-Developed Retroviral Vector CAR-T Technology Opens a New Path for Autoimmune Disease Treatment

The First Affiliated Hospital of China Medical University and Our Company

Collaborative Clinical Research Results Published in an International Journal

The First Affiliated Hospital of China Medical University and Shenzhen Cell Valley Biopharmaceutical Co., Ltd. recently published significant clinical research findings in the authoritative immunology journal Frontiers in Immunology (Q1, IF=5.9) (http://doi.org/10.3389/fimmu.2025.1645304). This marks the first reported case of successful treatment using anti-CD19 CAR-T cell therapy for a complex and rare condition involving systemic lupus erythematosus (SLE), antiphospholipid syndrome (APS), and type B insulin resistance syndrome (TBIRS). This achievement provides a novel approach for treating multi-system autoimmune diseases.

Rare Case Challenges the Limits of Traditional Treatment

The patient was diagnosed with SLE combined with TBIRS in 2020. Over four years, they underwent various treatments, including hormone pulse therapy, immunosuppressants, and biologics, but the condition relapsed and worsened, eventually progressing to APS and life-threatening complications such as retinal hemorrhage. Traditional treatments, which long relied on hormones and anticoagulants, not only had significant side effects but also failed to control disease progression effectively.

Innovative CAR-T Therapy Achieves "Killing Three Birds with One Stone" Efficacy

The research team at Shenzhen Cell Valley utilized retroviral vector-constructed anti-CD19 CAR-T cells (FMC63 scFv+CD28 costimulation + CD3ζ activation domain), which were produced under GMP standards and infused into the patient. The following remarkable outcomes were observed post-treatment:

• Comprehensive Relief of SLE Symptoms: Anti-dsDNA antibodies turned negative, creatinine levels normalized, and urinary protein decreased from ++++ to +;

• Effective Control of APS: Anti-β2GPI antibodies continued to decline, and coagulation function returned to normal;

• Rare Cure of TBIRS: Blood glucose stabilized, glycated hemoglobin normalized, and no further hypoglycemic therapy was required;

The patient recovered within one week post-treatment without severe adverse reactions such as cytokine release syndrome (CRS). They discontinued hormones, anticoagulants, and hypoglycemic drugs, retaining only a low-dose immunomodulator.

Breakthrough and Significance

This marks the first global report of CAR-T therapy successfully treating TBIRS syndrome, and it is also one of the very few cases that simultaneously improve SLE and APS. Compared to traditional B-cell depletion therapies, this study represents the first international use of a retroviral vector (RVV) to prepare CAR-T cells for SLE treatment. The RVV offers advantages such as high transduction efficiency, excellent cellular stability, and low plasmid requirements, providing a new therapeutic paradigm for multi-system autoimmune diseases.

Future Prospects

As a leading domestic one-stop solution provider focused on clinical translation and application, Shenzhen Cell Valley will collaborate with more clinical centers in the future to deepen clinical research and translational applications of cell therapy, delivering high-quality cell preparation services. In fields like autoimmune diseases, Cell Valley will continuously validate the safety and efficacy of therapies, offering new treatment hopes for more patients with refractory conditions.