Gene therapy scores big wins against blood cancers
In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients' blood cells into soldiers that seek and destroy cancer.
Novartis pipeline CTL019 (CART-19) Phase I/II
Chimeric antigen receptor-transduced T cells targeted to CD19
[*]CD19 is widely expressed on B cells, starting from the earliest precursor cells through all stages of maturation.
[*]CTL019 therapy involves adoptive transfer of autologous T cells that have been modified to express chimeric antigen receptors designed to recognize and kill CD19+ cancer cells.
[*]The chimeric antigen receptor consists of an intracellular T-cell receptor CD3-zeta chain signaling domain that induces T-cell activation, a costimulatory 4-1BB domain that enhances T-cell mediated responses and anti-CD19 antibody fragments that bind to CD19.
[*]T cells harvested from a patient are transduced with a lentiviral vector encoding the anti-CD19 chimeric antigen receptor. The resulting CTL019 cells are expanded ex vivo prior to infusion into the patient, who has undergone lymphocyte-depleting therapy. The CTL019 cells destroy tumor cells expressing CD19 and remain persistent in the body to guard against residual or recurring disease.
[*]Ongoing pilot studies are investigating the activity and safety of CTL019 therapy in patients with resistant or refractory CD19+ hematologic malignancies. Preliminary data from 3 patients with CLL have been published.
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