Two Infants Achieve Leukemia Remission After Gene Therapy

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CAR-T cells infused in the infants' blood eradicated leukemic B cells and helped them achieve remission.
CAR-T cells infused in the infants' blood eradicated leukemic B cells and helped them achieve remission.

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.

"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.

Reference

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

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