JAMA: FDA Approval of Tisagenlecleucel: Promise and Complexities of a $475 000 Cancer Drug Unlike most cancer therapies that are identical from patient to patient, CAR-T therapies are made by removing the T cells of a patient, genetically modifying them to respond to certain targets expressed on the patient’s cancer cells, and then reinfusing the cells.
When the T cells come into contact with the relevant target (for instance, CD19 in the case of ALL), they proliferate while secreting a number of programmed substances including inflammatory cytokines that destroy the cancerous cells.
Targeted killing of tumor cells by lymphocytes was first suggested by the graft-vs-leukemia effect in bone marrow transplantation, but that effect and the infusion of donor T cells more generally has no effect on solid tumor malignancies or most hematologic cancers.
The innovation underlying CAR-T involved exploiting the specificity of antibody-mediated recognition of tumor antigens, and then engineering CAR-T cells to have the relevant antibody fragment fused to the T-cell receptor.
Oh, there are sometimes controversies, but normal presidents avoid conflicts of interest, follow the guidelines of ethics officials and other best practices, and generally act as if they care about the appearance, at least, of playing by the rules.
Have something to add to this story? Share it in the comments.