Cell Medica, a leader in next-generation cellular immunotherapies for the treatment of cancer, today announced that its collaborators from Baylor College of Medicine and Texas Children’s Hospital presented the latest positive progress in the GINAKIT2 trial for children with R/R high risk neuroblastoma at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Washington, D.C.*

Patients were infused with an autologous CAR-NKT therapy, engineered to target GD2+ neuroblastoma cells. Early data from the first two patients at the lowest dose level (3 x 106 CAR-NKT cells/m2) showed substantial in vivo expansion of CAR-NKT cells and subsequent infiltration of CAR-NKT cells into both the solid tumor mass and bone marrow. There was also strong radiological evidence of extensive tumor regression at 4 weeks post infusion in one patient, with further regression observed at 8 weeks. No significant treatment associated toxicities, including cytokine release syndrome or neurotoxicity, were observed.

The CAR construct used in the GINAKIT2 study was also engineered to induce expression of the cytokine, IL-15. Data from in vivo mouse models, also presented at ASGCT, showed enhanced persistence of the CAR-NKT cells, which is thought to be important for long-term efficacy.

Dr. Andras Heczey, Principal Investigator, Assistant Professor, Pediatrics-Oncology at Baylor College of Medicine and Physician-Scientist, Texas Children’s Cancer Center commented: “Although it is early in the study, we are pleased to see evidence supporting the activity of CAR-NKT cells. We are looking forward to bringing additional patients onto the trial and treating at higher dose levels.”

Dr. Leonid Metelitsa, Professor of Pediatrics, Hematology-Oncology, Baylor College of Medicine and Co-Director, Neuroblastoma Program, Texas Children’s Cancer Center added: “These are early data but, nevertheless, they support the fundamental premise of CAR-NKT cells to effectively home to the site of disease in solid tumors and kill tumor cells. We hope these properties of CAR-NKT cells can be exploited to treat other malignancies and not only using patient-derived cells, but also in the allogeneic (off-the-shelf) setting.”

Chris Nowers, Cell Medica’s CEO, said: “Whilst these data are preliminary, it is exciting to see the first clinical evidence of a CAR therapy based on natural killer T cells reducing a solid tumor in a neuroblastoma patient, especially at the lowest dose level. This study will continue to recruit patients and build a clearer picture of the potential of CAR-NKT therapy. Concurrently we are also finalizing additional studies that will explore the potential of our innovative CAR-NKT platform in an off-the-shelf setting.”