Autologous Anti-GFRa4 CAR-TCR-zeta-4-1BB-expressing T-cells

**Semantic type:** Cell|Pharmacologic Substance

**Definition:** A preparation of autologous T-lymphocytes that have been transduced with a lentiviral vector to express a chimeric antigen receptor (CAR) consisting of an anti-glial-derived neurotrophic factor (GDNF) family receptor alpha 4 (GFRa4) single chain variable fragment (scFv), coupled to the zeta chain of the human T-cell receptor (TCR/CD3zeta) and the co-stimulatory molecule 4-1BB (CD137), with potential immunostimulating and antineoplastic activities. Upon re-introduction into the patient, the autologous anti-GFRa4 CAR-TCR-zeta-4-1BB-expressing T-cells target and bind to GFRa4-expressing tumor cells. This results in a cytotoxic T-lymphocyte (CTL) response against GFRa4-expressing tumor cells, resulting in tumor cell lysis. GFRa4 is highly expressed in metastatic medullary thyroid cancer (MTC) and also normally expressed in parafollicular cells within the thyroid and thymus.

**Synonyms:** - Autologous Anti-GFRa4 CAR-CD3zeta-4-1BB-transduced T-lymphocytes - Autologous Anti-GFRa4 CAR-T Cells - Autologous GDNF Family Receptor Alpha 4-targeted CAR-TCR-zeta-4-1BB-expressing T-lymphocytes - CART-GFRa4 Cells