A multi-site clinical trial is in progress for lentiviral-vector-based CD33 CAR T-cell treatments for pediatric Acute Myeloid Leukemia (AML). Six sites were approved for this trial sponsored by the National Marrow Donor Program. NCI’s Division of Cancer Treatment and Diagnosis (DCTD) is funding the Biopharmaceutical Development Program to serve as the central manufacturing facility for this autologous T-cell product for patients in this trial. The program receives frozen patient apheresis on a set schedule and ships back frozen autologous CAR-T product after approximately three weeks. As of June 2023, the BDP has successfully manufactured CD33 CART cell products for 30 patients.
NCI/DCTD is also supporting BDP in manufacturing autologous GD2-targeted CAR T-cells for pediatric sarcoma and glioblastoma patients. This is a multi-site clinical trial sponsored by the NCI Cancer Therapy Evaluation Program (CTEP) Pediatric Early Phase Clinical Trials Network (PEP-CTM). Manufacturing for the retroviral-vector-based GD2 CART product was developed at Stanford University and the technology was transferred to the BDP. The production protocol incorporates the drug dasatinib which purportedly inhibits T-cell exhaustion. The BDP manufactured cell product for the first patient on February 15, 2022. As of May 2023, GD2 CAR T-cell clinical production has been conducted for 6 patients