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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Cell Medica’s CAR-NKT cell therapy holds promise in treating neuroblastoma

Early data from Cell Medica’s chimeric antigen receptor-natural killer T (CAR-NKT) cell therapy trial, the first ever human trial of a genetically engineered NKT therapy, provides preliminary evidence for the efficacy of this treatment in children with high risk neuroblastoma.

Interim results of the trial, called as GINAKIT2, were presented by Cell Medica’s collaborators at Baylor College of Medicine and Texas Children’s Hospital at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Washington.

GINAKIT2 is a dose escalation study evaluating CMD-501 in children with high risk neuroblastoma. In the trial, patients are infused with an autologous CAR-NKT therapy, in which NKT cells are genetically engineered with a CAR targeting GD2.

Neuroblastoma, one of the deadliest types of childhood cancer, is the most common solid tumor in children that occurs outside of the brain and makes up 8% of the total number of children’s cancers. GD2 is a molecule expressed on the surface of most neuroblastoma cells and it’s these molecules that Cell Medica’s CMD-501 targets.

8-week data obtained from the first two patients who received the lowest dose of the product showed significant expansion of CAR-NKT cells and subsequent infiltration of CAR-NKT cells into both the solid tumor mass and bone marrow. There was also evidence of tumor regression at 4 weeks in one patient, with further regression reported at 8 weeks. No significant treatment associated toxicities, including cytokine release syndrome or neurotoxicity, were observed.

One highlight of this GD2-specific CAR construct is that it is additionally engineered to secrete the cytokine IL-15, which has been shown in preclinical studies to increase the persistence of CAR-NKT cells and improve their efficacy within the tumor microenvironment.

The study will now focus on testing these cells on additional patients at higher dose levels. The company is also working with Baylor College of Medicine to develop off-the-shelf CAR-NKT cell therapy, where cells from a healthy donor is used to generate CAR-NKT cells. The NKT cells that are genetically engineered with a CAR targeting CD19 will be tested for its efficacy in hematological malignancies and solid tumors.

Chris Nowers, Cell Medica’s CEO commented: “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.”

Source: Cell Medica Collaborators, Baylor College of Medicine and Texas Children’s Hospital, Present Positive Early Patient Data From CAR-NKT Neuroblastoma Trial at American Society Of Gene & Cell Therapy 22ndAnnual Meeting; Press Release

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