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Autologous chimeric antigen receptor (CAR) T cells — genetically modified and expanded ex vivo to target the CD19 antigen expressed on most B-cell malignancies — have been shown to be a highly promising cellular immunotherapy for relapsed acute lymphoblastic leukemia (ALL; NEJM JW Oncol Hematol Oct 15 2014).
Now, investigators have conducted a phase I trial to assess CAR-T-cell dosing, response, and toxicity in 20 patients (age range, 1–30 years) with relapsed or refractory B-precursor ALL (B-ALL). Of these patients, six had primary refractory B-ALL, and eight had relapsed after prior allogeneic stem-cell transplantation (SCT).
Results were as follows:
2 patients had unsuccessful CAR-T-cell generation and did not respond to therapy.
14 patients…