Persistence and efficacy of second generation CAR T cell against the LeY antigen in acute myeloid leukemia

Mol Ther. 2013 Nov;21(11):2122-9. doi: 10.1038/mt.2013.154. Epub 2013 Jul 8.


In a phase I study of autologous chimeric antigen receptor (CAR) anti-LeY T-cell therapy of acute myeloid leukemia (AML), we examined the safety and postinfusion persistence of adoptively transferred T cells. Following fludarabine-containing preconditioning, four patients received up to 1.3 × 109 total T cells, of which 14-38% expressed the CAR. Grade 3 or 4 toxicity was not observed. One patient achieved a cytogenetic remission whereas another with active leukemia had a reduction in peripheral blood (PB) blasts and a third showed a protracted remission. Using an aliquot of In111-labeled CAR T cells, we demonstrated trafficking to the bone marrow (BM) in those patients with the greatest clinical benefit. Furthermore, in a patient with leukemia cutis, CAR T cells infiltrated proven sites of disease. Serial PCR of PB and BM for the LeY transgene demonstrated that infused CAR T cells persisted for up to 10 months. Our study supports the feasibility and safety of CAR-T-cell therapy in high-risk AML, and demonstrates durable in vivo persistence.

Publication types

  • Clinical Trial, Phase I
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Bone Marrow / immunology
  • Female
  • Humans
  • Immunotherapy, Adoptive*
  • Leukemia, Myeloid, Acute / immunology
  • Leukemia, Myeloid, Acute / therapy*
  • Lewis Blood Group Antigens / immunology*
  • Male
  • Middle Aged
  • Receptors, Antigen, T-Cell / immunology*
  • Remission Induction
  • T-Lymphocytes / immunology*
  • Transplantation Conditioning
  • Vidarabine / analogs & derivatives
  • Vidarabine / therapeutic use


  • Lewis Blood Group Antigens
  • Lewis Y antigen
  • Receptors, Antigen, T-Cell
  • Vidarabine
  • fludarabine