Two infants with advanced acute lymphoblastic leukemia (ALL) achieved remission after treatment with genetically engineered (chimeric) antigen receptor (CAR) immune T cells, researchers reported in Science Translational Medicine.

Both infants had relapsed, treatment-refractory CD19+ B-cell ALL. Previous efforts have involved administering patients genetically engineered versions of their own immune cells, but the infants were administered “universal” autologous CAR-T cells that had been modified using the TALEN gene-editing platform. The CAR-T cells were infused into the patients’ blood, where they recognized and eradicated leukemic B cells.

Both experienced complete remissions.

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“Molecular remissions were achieved within 28 days in both infants,” reported lead study author Waseem Qasim, MD, PhD, from University College London, United Kingdom, and colleagues. “This bridge-to-transplantation strategy demonstrates the therapeutic potential of gene-editing technology.”

The approach is more practical than older allogenic approaches because the premanufactured CAR-T cells can be used “off the shelf”; they need not be obtained from each patient for genetic engineering and infusion.

“This therapeutic application of TALEN-engineered cells highlights the feasibility and potency of gene-editing strategies for the delivery of antitumor immunity,” the authors concluded.


1. Qasim W, Zhan H, Samarasinghe S, et al. Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells. Sci Transl Med. 2017;9:eaaj2013. doi: 10.1126/scitranslmed.aaj2013