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.