David L. Porter, MD
Saying that the treatment is “poised to transform the outlook for children and adults with certain otherwise incurable cancers,” ASCO has selected CAR T-cell therapy as its Advance of the Year.
“The patients who participated in the early clinical trials of this therapy at Penn starting nearly 8 years ago are pioneers, whose stories are now part of medical history. Our experiences developing this therapy and caring for these patients have revolutionized our belief about what is possible in oncology, and galvanized us as we move into adapting CAR T-cell therapies for the treatment of solid tumors,” added Porter.
Tisagenlecleucel won approval based on results from the phase II, single-arm, international ELIANA trial of 63 patients who received a single dose of tisagenlecleucel. The overall remission rate was 82.5% (95% CI, 70.9-91.0) in treated patients. Forty patients (63%) had complete remission (CR) and 12 (19%) had complete remission with incomplete hematologic recovery (CRi). All patients who had CR or CRi also had negative minimal residual disease status in the bone marrow.
In the updated safety results, 48% of patients experienced grade 3/4 cytokine release syndrome (CRS). There were no deaths associated with CRS.
The FDA relied on results from the single-arm ZUMA-1 study in approving axi-cel.2
Those with DLBCL had an ORR of 82% and a CR rate of 49%. After 8.7 months of follow-up, the ORR in the DLBCL group was 36%, which included a CR rate of 31%. In the PMBCL/TFL group, the ORR was 83% and the CR rate was 71%. The 8.7-month ORR rate was 67%, with a CR rate of 63%.
In ZUMA-1, patients were enrolled into 2 cohorts consisting of those with DLBCL (n = 77) and those with PMBCL or TFL (n = 24). Axi-cel was administered as a single infusion of modified autologous T cells at a target dose of 2 x 106
CAR-positive T cells/kg.
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