Related Experiment Video
Updated: May 13, 2026
A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells
Published on: November 12, 2019
Chimeric antigen receptor-modified T cells for acute lymphoid leukemia
Stephan A Grupp1, Michael Kalos1, David Barrett1
1Children's Hospital of Philadelphia (S.A.G., D.B., R.A., S.R.R., D.T.T., B.H., J.F.W.); the Department of Pediatrics (S.A.G., D.B., R.A., S.R.R., D.T.T.), Abramson Cancer Center (S.A.G., M.K., R.A., D.L.P., S.R.R., D.T.T., M.C.M., B.L.L., C.H.J.); and the Departments of Pathology and Laboratory Medicine (M.K., A.C., B.H., J.F.W., M.C.M., B.L.L., C.H.J.) and Medicine (D.L.P.), University of Pennsylvania - all in Philadelphia.
Chimeric antigen receptor T-cell therapy shows promise for acute lymphoblastic leukemia (ALL). This treatment led to complete remission in two pediatric patients, though one relapsed due to CD19-negative cells, highlighting the need for broader targeting strategies.
Area of Science:
- Immunotherapy
- Oncology
- Cellular Therapy
Background:
- Chimeric antigen receptor (CAR) T-cell therapy targeting CD19 has shown efficacy in chronic lymphocytic leukemia (CLL).
- The clinical activity of CAR T-cells in acute lymphoblastic leukemia (ALL) requires further investigation.
- CAR T-cells offer a potential new avenue for treating relapsed and refractory hematologic malignancies.
Observation:
- Two pediatric patients with relapsed/refractory B-cell ALL received CTL019 CAR T-cell therapy.
- CAR T-cells demonstrated significant expansion in vivo, reaching over 1000 times the initial engraftment level.
- CAR T-cells were detected in bone marrow and cerebrospinal fluid, persisting for at least six months.
Findings:
- Both patients achieved complete remission, with one ongoing at 11 months.
- Adverse events included cytokine-release syndrome and B-cell aplasia; cytokine blockade was effective.
- One patient relapsed due to the emergence of CD19-negative leukemia cells.
Implications:
- CAR T-cell therapy is capable of eradicating aggressive, treatment-refractory ALL.
- The development of antigen-negative relapse underscores the need for alternative or combination targeting strategies in ALL treatment.
- Further research into novel CAR T-cell targets is crucial for durable responses in ALL patients.
