GD2-CART01 / Bambino Gesu Hospital - LARVOL DELTA

CAR T Cell Therapies for Pediatric Solid Tumors (ICBMT 2025) - "At the Bambino Gesù Children's Hospital (OPBG) we developed a third-generation (CD28-4.1bb), GD2-directed CAR construct (GD2-CART01), incorporating the safety switch inducible caspase 9 (iC9)...Lastly, the experience of the Center for Cell and Gene Therapy, Baylor College of Medicine and Texas Children's Cancer Center group in Houston, with the use of armoured CAR T cells directed towards GPC3 and the remarkable results obtained will be reviewed. All these experiences highlight that a new era for the treatment of solid tumors with CAR T cells has just begun and many more promising results are expected in the near future."

CAR T-Cell Therapy • Clinical • Brain Cancer • Diffuse Midline Glioma • Gene Therapies • Glioma • Hematological Malignancies • Neuroblastoma • Oncology • Pediatrics • Solid Tumor • GPC3

GD2-targeting CAR T cells in high-risk neuroblastoma: a phase 1/phase 2 trial. (PubMed, Nat Med) - P1/2 | "Grade 3 immune effector cell-associated neurotoxicity syndrome was diagnosed in four children and rapidly controlled with the activation of the inducible caspase-9 suicide gene by rimiducid. These results confirm that GD2-CART01 can induce durable remissions in children with high-risk metastatic, relapsed, or refractory neuroblastoma. ClinicalTrials.gov identifier: NCT03373097 ."

Journal • P1 data • P2 data • Neuroblastoma • Oncology • Solid Tumor

Anti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk And/or Relapsed/refractory Neuroblastoma or Other GD2-positive Solid Tumors (clinicaltrials.gov) - P1/2 | N=42 | Active, not recruiting | Sponsor: Bambino Gesù Hospital and Research Institute | Trial primary completion date: Dec 2024 ➔ Dec 2026 | Recruiting ➔ Active, not recruiting

Enrollment closed • Trial primary completion date • CNS Tumor • Ewing Sarcoma • Neuroblastoma • Oncology • Osteosarcoma • Pediatrics • Sarcoma • Solid Tumor

Donor-derived GD2-specific CAR T cells in relapsed or refractory neuroblastoma. (PubMed, Nat Med) - "In comparison to autologous CAR T cells, ALLO_GD2-CAR T cells were characterized by pathways associated with T cell proliferation, immune-synapse formation and cell chemotaxis. The safety and efficacy of ALLO_GD2-CART01 in children with r/r HR-NB deserve further investigation in a prospective trial."

Journal • Acute Graft versus Host Disease • Bone Marrow Transplantation • CNS Tumor • Graft versus Host Disease • Immunology • Neuroblastoma • Oncology • Solid Tumor • Transplantation

GD2-CAR T Cells for Pediatric Brain Tumours (clinicaltrials.gov) - P1 | N=54 | Recruiting | Sponsor: Bambino Gesù Hospital and Research Institute

CAR T-Cell Therapy • Trial completion date • Trial primary completion date • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Embryonal Tumor • Glioma • Medulloblastoma • Oncology • Pediatrics • Solid Tumor

GD2-CAR T Cells for Pediatric Brain Tumours (clinicaltrials.gov) - P1 | N=54 | Recruiting | Sponsor: Bambino Gesù Hospital and Research Institute | Not yet recruiting ➔ Recruiting

CAR T-Cell Therapy • Enrollment open • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Embryonal Tumor • Glioma • Medulloblastoma • Oncology • Pediatrics • Solid Tumor

GD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma. Reply. (PubMed, N Engl J Med) - No abstract available

Journal • CNS Tumor • Neuroblastoma • Neuroendocrine Tumor • Oncology • Solid Tumor

GD2-CART01 Elicits Clinical Activity, Tolerability in Pediatric R/R Neuroblastoma (OncLive) - P1/2 | N=42 | NCT03373097 | "The investigational CAR T-cell therapy GD2-CART01 showed preliminary antitumor activity and safety in pediatric patients with relapsed or refractory high-risk neuroblastoma, according to data from a phase 1/2 trial...Three-year results from the study showed that patients who received GD2-CART01 (n = 27) experienced an overall response rate of 63%, with 9 patients achieving a complete response (CR). Additionally, patients who received the recommended dose had a 3-year overall survival (OS) rate of 60% and a 3-year event-free survival (EFS) rate of 36%."

P1/2 data • CNS Tumor • Neuroblastoma • Oncology • Solid Tumor

GD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma. (PubMed, N Engl J Med) - P1/2 | "The use of GD2-CART01 was feasible and safe in treating high-risk neuroblastoma. Treatment-related toxic effects developed, and the activation of the suicide gene controlled side effects. GD2-CART01 may have a sustained antitumor effect. (Funded by the Italian Medicines Agency and others; ClinicalTrials.gov number, NCT03373097.)."

Journal • CNS Tumor • Neuroblastoma • Neuroendocrine Tumor • Oncology • Solid Tumor

GD2-CAR T Cells for Pediatric Brain Tumours (clinicaltrials.gov) - P1 | N=54 | Not yet recruiting | Sponsor: Bambino Gesù Hospital and Research Institute

CAR T-Cell Therapy • New P1 trial • Brain Cancer • CNS Tumor • Diffuse Intrinsic Pontine Glioma • Diffuse Midline Glioma • Embryonal Tumor • Glioma • Medulloblastoma • Oncology • Pediatrics • Solid Tumor