A groundbreaking clinical study led by Renji Hospital (Shanghai Jiao Tong University School of Medicine) has achieved a world-first: demonstrating the potential of CAR-T cell therapy in treating advanced hepatocellular carcinoma (HCC)—the most common type of liver cancer.
Published in the prestigious journal Clinical Cancer Research (Impact Factor: 8.9), the Phase I clinical trial focused on the safety and efficacy of CAR-GPC3 T cells, a novel therapy targeting Glypican-3 (GPC3)—a tumor-specific antigen highly expressed in 70–80% of HCC cases.
A Landmark Case: Tumor-Free Survival for Nearly 5 Years
Among the 13 patients treated in the trial, one individual with advanced liver cancer has remained tumor-free and in good health for nearly five years. This patient, who enrolled in July 2015, showed no active lesions during follow-ups and maintained normal tumor marker levels, setting a remarkable precedent for long-term remission in late-stage HCC.
Promising Safety and Early Efficacy Results
The trial included 13 patients with GPC3-positive advanced liver cancer. Findings showed that:
- Most patients tolerated the therapy well.
- Common side effects included mild cytokine release syndrome (CRS) and temporary lymphopenia.
- No severe toxicity was observed.
- Two patients experienced partial remission (PR).
- Median overall survival (OS) was 278 days, with 6-month, 1-year, and 3-year survival rates at 50.3%, 42.0%, and 10.5%, respectively.
One of the longest-surviving patients had extensive metastasis, including cancer thrombi in the inferior vena cava and right atrium. After treatment, their AFP (alpha-fetoprotein) levels normalized and remained stable.
A New Chapter for Solid Tumor Treatment
CAR-T therapy has revolutionized treatment for blood cancers, but solid tumors have posed greater challenges. This study marks the first clinical report globally of CAR-T therapy targeting GPC3 in liver cancer, providing valuable data and hope for expanding CAR-T applications beyond hematologic malignancies.
Professor Bo Zhai, lead investigator and director of Interventional Oncology at Renji Hospital, emphasized the need for continued innovation, especially for patients diagnosed at late stages, where surgery is often no longer an option. He noted that while this study is just the beginning, it lays essential groundwork for broader applications of CAR-T in solid tumors.
Professor Zonghai Li, inventor of the CAR-GPC3 T cell and founder of CARsgen Therapeutics, highlighted GPC3’s potential as an ideal target due to its tumor-specific expression and its absence in normal tissues. CARsgen plans to further develop this therapy and expand clinical research, aiming to bring new hope to patients with liver cancer worldwide.
CAR T Cells China continues to monitor and report on major breakthroughs in CAR-T therapy across China. For more information on clinical trials or treatment access, please contact us.
