A pioneering study led by Professor Xu Huji from the Second Affiliated Hospital of Naval Medical University (Shanghai Changzheng Hospital) has demonstrated for the first time that off-the-shelf CAR-T cell therapy can effectively treat severe autoimmune diseases. The groundbreaking findings, published online in Cell on July 15, 2024, mark a major step forward in cell-based immunotherapy beyond cancer treatment.
A New Era for Autoimmune Disease Treatment
CAR-T cell therapy—originally developed for treating blood cancers—involves engineering a patient’s T cells to express a Chimeric Antigen Receptor (CAR) that targets disease-causing cells. Most existing CAR-T therapies are autologous, meaning they use the patient’s own immune cells. However, this method is complex, time-consuming, and costly, especially for patients with compromised immune systems.
The newly tested off-the-shelf (allogeneic) CAR-T approach uses T cells from healthy donors, genetically modified and mass-produced for immediate use in multiple patients. This allows faster deployment and significantly reduced treatment costs.
Clinical Success in Three Patients
In the investigator-initiated trial (IIT), three patients with severe, treatment-resistant autoimmune conditions—immune-mediated necrotizing myopathy (IMNM) and systemic sclerosis (SSc)—were treated with the new therapy. All experienced significant improvement:
- Patient 1 (IMNM): Previously unable to stand without assistance, the patient regained full muscle strength within one month. Lab tests showed normalized muscle enzyme levels, and autoimmune antibodies were eliminated.
- Patients 2 and 3 (SSc): Both had extremely hardened skin, which softened after treatment, improving mobility and injection accessibility. Biopsies confirmed reduced inflammation and regeneration of skin structures. Imaging revealed reversal of heart and lung fibrosis, sustained for over six months.
Professor Xu noted, “We used to believe fibrosis was irreversible. Now, imaging and pathology show it can be reversed—this opens a new scientific frontier.”
Massive Potential for Widespread Disease
Autoimmune diseases affect over 8% of the global population, with more than 100 million patients in China alone, many facing disability and high treatment costs. Current therapies—including steroids and biologics—are often inadequate, especially for severe cases.
“This new therapy could become a powerful option for patients with limited choices,” said Xu.
Although this trial involved only three patients, 12 more have since been treated, and broader clinical trials are anticipated to fully validate the therapy’s safety and efficacy.
This study represents a landmark achievement in the treatment of autoimmune diseases and a major step toward more accessible, affordable CAR-T therapies. If confirmed in larger studies, off-the-shelf CAR-T could transform care for millions of patients with severe immune disorders.
