PI3K orchestration of the in vivo persistence of chimeric antigen receptor-modified T cells

Leukemia. 2018 May;32(5):1157-1167. doi: 10.1038/s41375-017-0008-6. Epub 2018 Feb 2.

Abstract

In vivo persistence of chimeric antigen receptor (CAR)-modified T cells correlates with therapeutic efficacy, yet CAR-specific factors that support persistence are not well resolved. Using a CD33-specific CAR in an acute myeloid leukemia (AML) model, we show how CAR expression alters T cell differentiation in a ligand independent manner. Ex vivo expanded CAR-T cells demonstrated decreased naïve and stem memory populations and increased effector subsets relative to vector-transduced control cells. This was associated with reduced in vivo persistence. Decreased persistence was not due to specificity or tumor presence, but to pre-transfer tonic signaling through the CAR CD3ζ ITAMs. We identified activation of the PI3K pathway in CD33 CAR-T cells as responsible. Treatment with a PI3K inhibitor modulated the differentiation program of CAR-T cells, preserved a less differentiated state without affecting T cell expansion, and improved in vivo persistence and reduced tumor burden. These results resolve mechanisms by which tonic signaling of CAR-T cells modulates their fate, and identifies a novel pharmacologic approach to enhance the durability of CAR-T cells for immunotherapy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Differentiation / drug effects
  • Cell Line, Tumor
  • Humans
  • Immunotherapy, Adoptive / methods*
  • Leukemia, Myeloid, Acute / therapy*
  • Lymphocyte Activation / drug effects
  • Phosphatidylinositol 3-Kinases / metabolism*
  • Phosphoinositide-3 Kinase Inhibitors
  • Receptors, Chimeric Antigen / therapeutic use*
  • Sialic Acid Binding Ig-like Lectin 3 / pharmacology
  • Sialic Acid Binding Ig-like Lectin 3 / therapeutic use
  • T-Lymphocytes
  • Tumor Burden / drug effects

Substances

  • CD33 protein, human
  • Phosphoinositide-3 Kinase Inhibitors
  • Receptors, Chimeric Antigen
  • Sialic Acid Binding Ig-like Lectin 3