FDA Authorizes First Human Clinical Trial for a CAR-T Therapy for Neuroblastoma

The Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for PHOX2B PC-CAR T (Myrio Therapeutics, Melbourne, Australia), a novel chimeric antigen receptor T-cell (CAR-T) therapy under investigation for the potential treatment of neuroblastoma. The decision enables the therapy to enter first-in-human clinical trials for the treatment of relapsed neuroblastoma, a high-mortality pediatric cancer of the sympathetic nervous system.

PHOX2B PC-CAR-T was developed to recognize and target the PHOX2B protein present in neuroblastoma cells. According to a statement from Myrio Therapeutics, the therapy’s design using a proprietary technology includes a highly specific binder that targets PHOX2B peptide-major histocompatibility complex (p-HLA), enabling recognition across multiple human leukocyte antigen (HLA) allotypes. Other CAR-T approaches can target only a single HLA allotype by contrast.

The therapy was developed in collaboration with the Children’s Hospital of Philadelphia (CHOP).  In a statement from John M. Maris, MD, Pediatric Oncology, CHOP: “Neuroblastoma is the most common tumor of the sympathetic nervous system, the most common malignancy of infancy, and accounts for 15% of pediatric cancer-related deaths. The current treatment options for patients with high-risk neuroblastoma are associated with low response rates and significant toxicities and the development of new treatment options is desperately needed. This investigational immunotherapy has the potential to be a major advance for patients suffering from this devastating disease.”

Myrio Therapeutics announced that a phase 1 clinical trial will be conducted by a team led by Dr. Maris. The first patient is expected to be enrolled mid 2025, and the trial will be titled “PHOX2B Peptide-Centric Chimeric Antigen Receptor Autologous T cells (PHOX2B PC-CAR T).”