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Published on: December 17, 2019
Chimeric antigen receptor T cells for sustained remissions in leukemia
Shannon L Maude1, Noelle Frey, Pamela A Shaw
1From the Division of Oncology, Children's Hospital of Philadelphia (S.L.M., R.A., D.M.B., N.J.B., S.R.R., D.T.T., S.A.G.), the Departments of Pediatrics (S.L.M., R.A., D.M.B., N.J.B., S.R.R., D.T.T., S.A.G.), Biostatistics and Epidemiology (P.A.S., R.A.), and Pathology and Laboratory Medicine (J.J.M., B.L.L., C.H.J., S.A.G.), the Division of Hematology-Oncology (N.F., D.L.P.), and Abramson Cancer Center (N.F., A.C., V.E.G., Z.Z., S.F.L., Y.D.M., J.J.M., B.L.L., C.H.J., D.L.P., S.A.G.), Perelman School of Medicine, University of Pennsylvania - all in Philadelphia; and Novartis Pharmaceuticals, East Hanover, NJ (A.S.).
Chimeric antigen receptor (CAR) T-cell therapy targeting CD19 effectively treats relapsed acute lymphoblastic leukemia (ALL). This CAR T-cell therapy achieved high remission rates, even in patients with refractory disease or after stem-cell transplantation.
Area of Science:
- Oncology
- Immunotherapy
- Cellular Therapy
Background:
- Relapsed acute lymphoblastic leukemia (ALL) presents significant treatment challenges.
- Conventional therapies often fall short for patients with refractory ALL.
- Chimeric antigen receptor (CAR)-modified T cells targeting CD19 offer a promising therapeutic strategy.
Purpose of the Study:
- To evaluate the efficacy and safety of CD19-directed CAR T-cell therapy (CTL019) in patients with relapsed or refractory ALL.
- To assess treatment response, toxic effects, and CAR T-cell persistence in treated patients.
Main Methods:
- Infusion of autologous T cells engineered with a CD19-specific CAR (CTL019) via lentiviral vector.
- Administration of CTL019 at doses ranging from 0.76×10^6 to 20.6×10^6 cells/kg.
- Monitoring of patients for treatment response, adverse events, and CTL019 cell expansion and persistence.
Main Results:
- Complete remission achieved in 90% (27/30) of patients, including those with blinatumomab-refractory disease and post-stem-cell transplant.
- CTL019 cells demonstrated in vivo proliferation and were detectable in blood, bone marrow, and cerebrospinal fluid.
- Six-month event-free survival was 67% and overall survival was 78%; sustained remissions up to 24 months were observed.
- Cytokine-release syndrome occurred in all patients, with severe cases (27%) managed effectively with tocilizumab.
Conclusions:
- CD19-directed CAR T-cell therapy (CTL019) is effective for relapsed and refractory ALL.
- The therapy demonstrates high remission rates and durable responses, even in challenging patient populations.
- CTL019 offers a viable treatment option for patients who have failed previous therapies, including stem-cell transplantation.
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