World's First! Fudan Eye & ENT Hospital Team Treats Advanced INI1-Deficient Head and Neck Tumor with CAR-T

Background

Advanced INI1-deficient sinonasal malignant tumors are rare and highly aggressive cancers. These tumors are characterized by loss of INI1 (SMARCB1) expression and classified as SWI/SNF-deficient carcinomas. They frequently invade local structures such as the eyeball and can metastasize, resulting in severe symptoms including significant pain. Standard treatments often include repeated endoscopic surgical resections, radiotherapy, chemotherapy, targeted therapy and immunotherapy.

What was achieved

The team from the Eye & ENT Hospital of Fudan University consisting of Yu Hongmeng, Wu Haitao and Chen Jian, in collaboration with a domestic biopharmaceutical technology company, completed the world's first use of Trop2-targeted CAR-T cell therapy for an advanced recurrent INI1-deficient sinonasal malignant tumor. The CAR-T cells were infused in August 2025 after ethics committee approval. This built upon the hospital's cell therapy research team's long-term basic research on CAR-T.

The patient, an elderly male diagnosed in 2024, had previously received multiple nasal endoscopic tumor resections, more than 50 sessions of radio-chemotherapy combined with targeted and immunotherapy, with conventional options exhausted. One week after infusion, he reported significant relief from right eye cancer pain. Pain resolved completely by two weeks. Magnetic resonance imaging showed obvious lesion regression, with previously compressed blood vessels reappearing clearly, indicating tumor shrinkage. The multidisciplinary team confirmed significant remission. The patient remained stable with follow-up exceeding 245 days as of the April 2026 report.

Yu Hongmeng, vice president of the hospital, stated that the successful treatment resulted from collaboration between the hospital MDT team and the innovative enterprise. The hospital intends to promote clinical translation of more innovative therapies. The outcome demonstrates the potential of Trop2-targeted CAR-T cells in solid tumor treatment.

How it works

CAR-T therapy is an immunotherapy that uses a patient's own immune cells. T cells are collected from the blood. In the laboratory, these cells are genetically modified to express a chimeric antigen receptor (CAR) on their surface. The CAR is designed to recognize a specific protein called Trop2 that is expressed on the tumor cells in this case. The modified cells are then expanded in number and infused back into the patient.

Once returned to the body, the CAR-T cells seek out and bind to Trop2 on the cancer cells, activating to destroy them. This next-generation CAR-T approach was applied to a solid tumor after standard therapies failed. Solid tumors present additional challenges compared to blood cancers, including barriers to cell infiltration and an immunosuppressive environment. Treatment in this case was coordinated via multidisciplinary team oversight following ethics approval for the individual patient whose tumor expressed the target.

What it means for international patients

This case represents an effort to apply CAR-T cell therapy, which has shown success in certain blood cancers, to advanced refractory solid tumors in the head and neck region. It highlights research activity at specialized Chinese centers into innovative cellular therapies for tumors with limited conventional options.

The Eye & ENT Hospital of Fudan University provides services accessible to international patients. Individuals with advanced INI1-deficient or similar refractory head and neck tumors interested in such therapies must contact the hospital directly to confirm eligibility, current protocols, availability and any regulatory requirements. This reported treatment was performed on an individualized basis under ethics approval, and suitability depends on specific tumor characteristics such as Trop2 expression and the patient's clinical status.

About the hospital

The Eye & ENT Hospital of Fudan University is a Grade 3A specialized tertiary hospital located in Shanghai. It is affiliated with Fudan University and focuses on ophthalmology, otolaryngology, head and neck surgery and related conditions. The hospital conducts clinical care, education and research across these specialties, including work on gene therapy and cell therapy. It has supported establishment of a rare disease gene editing and cell treatment key laboratory.

The institution operates multiple campuses and has a history of advancing treatments for complex eye, ear, nose and throat disorders, including skull base and head and neck tumor management.