Refining CAR-T Cell Therapy for Acute Lymphoblastic Leukemia

A novel, gene-edited “off-the-shelf” CAR-T cell product showed feasibility and clinical responses in relapsed or refractory B-cell ALL.

Chimeric antigen receptor T-cells (CAR-T) generated from autologous T-cells of patients with B-cell malignancies can induce durable remission, but this therapy may not be available in settings of rapid disease progression or inability to collect and reprogram sufficient cells for reinfusion. Investigators report pooled results from two phase 1 clinical studies of allogeneic CAR-T therapy using healthy donor T-cells targeted to the B-cell antigen CD19 plus inactivation of the normal T-cell receptor and CD52 surface antigen. The resulting “UCART19” was infused into 7 children (ages 9 months–16 years) and 14 adults (ages 18–62 years) with heavily pretreated, relapsed or refractory B-cell acute lymphoblastic leukemia, following intensive immuno…

Reviewing Author

Michael E. Williams, MD, ScM

Disclosures

Consultant/Advisory BoardAbbVie; AstraZeneca; Beigene; Janssen; Pharmacyclics

Grant/Research SupportJanssen

Editorial BoardsAmerican Board of Internal Medicine (ABIM) Hematology Longitudinal Knowledge Approval Committee

Michael E. Williams, MD, ScM

Disclosures

Consultant/Advisory BoardAbbVie; AstraZeneca; Beigene; Janssen; Pharmacyclics

Grant/Research SupportJanssen

Editorial BoardsAmerican Board of Internal Medicine (ABIM) Hematology Longitudinal Knowledge Approval Committee

Refining CAR-T Cell Therapy for Acute Lymphoblastic Leukemia

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Reviewing Author