On January 15, 2024, #CARsgen Pharmaceuticals announced that its autologous #CARTcell candidate product #CT011, targeting Glypican-3 (GPC3), has been approved for clinical use in China. It is intended for the treatment of GPC3-positive stage IIIa hepatocellular carcinoma patients at risk of recurrence after surgical resection.

Glypican-3 (GPC3)

    Glypican-3 (GPC3)plays a crucial role in regulating cell growth and differentiation and is closely associated with the occurrence and progression of liver cancer. GPC3 is expressed in reproductive system tumors such as hepatocellular carcinoma, ovarian clear cell carcinoma, and yolk sac tumors. Its expression rate in hepatocellular carcinoma reaches 74.8%, while it is virtually absent in normal liver tissue, making it an ideal new target for #livercancer #CARTtherapy.

Promising Target

Due to its tumor specificity, GPC3 is considered a promising target for cancer immunotherapy. Higher expression of GPC3 in hepatocellular carcinoma is associated with poorer prognosis.

Frontiers in Immunology

In August 2022, a Chinese medical team published a long-term survival case report of treating advanced hepatocellular carcinoma with CT011 in the journal “#FrontiersinImmunology.” The study reported complete remission (#CR) and long-term survival in a patient with advanced hepatocellular carcinoma after receiving GPC3 CAR-T cell therapy in combination with the multi-target kinase inhibitor sorafenib.

Results showed good tolerance to CT011 combined with sorafenib treatment. The patient achieved partial remission (PR) from the third month onwards and attained complete remission at the 12th month after the first CT011 infusion. The tumor did not progress for over 36 months, maintaining complete remission status for over 24 months after the first infusion.

Cancer Communications

On October 12, 2023, CARsgen Pharmaceuticals announced the clinical efficacy of its independently developed CAR-T cell therapy targeting GPC3 in two patients with advanced hepatocellular carcinoma (HCC). The clinical trial results were published in “#CancerCommunications.” Both patients had inferior prognostic outcomes upon enrollment, with one having inferior vena cava tumor thrombus and the other having retroperitoneal lymph node metastasis.

In the clinical trial, one patient received local treatment for intrahepatic recurrent tumors and inferior vena cava tumor thrombus (microwave ablation and gamma knife) before receiving CAR-T cell therapy targeting GPC3. Six months later, AFP (alpha-fetoprotein) levels normalized, and imaging revealed no active tumors. The other patient underwent local treatment (microwave ablation and gamma knife) for controlling intrahepatic tumors, inferior vena cava tumor thrombus, and peritoneal lymph node metastases while receiving CAR-T cell therapy.

Encouragingly, both patients showed significant efficacy after combined local treatment and CAR-T cell infusion, maintaining tumor-free status during long-term follow-up, with both individuals being tumor-free for over 7 years. Throughout the follow-up period, both patients only received oral antiviral therapy for hepatitis B and did not undergo any other cancer treatment.

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